Trial Outcomes & Findings for A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome (NCT NCT03936777)
NCT ID: NCT03936777
Last Updated: 2025-11-17
Results Overview
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. An AE can, therefore, be any unfavorable and unintended sign (including an abnormal, clinically significant laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not considered related to the medicinal (investigational) product. TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment-emergent. The percentage of participants was rounded to one decimal place.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
412 participants
Primary outcome timeframe
From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
Results posted on
2025-11-17
Participant Flow
The study started to enroll participants in April 2019 and concluded in May 2025.
The Participant Flow refers to the Safety analysis set.
Participant milestones
| Measure |
Any ZX008 Open Label Dose
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Overall Study
STARTED
|
412
|
|
Overall Study
COMPLETED
|
360
|
|
Overall Study
NOT COMPLETED
|
52
|
Reasons for withdrawal
| Measure |
Any ZX008 Open Label Dose
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Death
|
3
|
|
Overall Study
Lack of Efficacy
|
24
|
|
Overall Study
Lost to Follow-up
|
3
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Withdrawal by Subject
|
11
|
|
Overall Study
Subjected Weaned of ZX008
|
1
|
|
Overall Study
Missing
|
6
|
Baseline Characteristics
A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome
Baseline characteristics by cohort
| Measure |
Any ZX008 Open Label Dose
n=412 Participants
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Age, Continuous
|
14.1 Years
STANDARD_DEVIATION 6.8 • n=202 Participants
|
|
Age, Customized
2 - <12 years
|
168 Participants
n=202 Participants
|
|
Age, Customized
12 - <18 years
|
117 Participants
n=202 Participants
|
|
Age, Customized
18 - <65 years
|
127 Participants
n=202 Participants
|
|
Sex: Female, Male
Female
|
191 Participants
n=202 Participants
|
|
Sex: Female, Male
Male
|
221 Participants
n=202 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
4 Participants
n=202 Participants
|
|
Race/Ethnicity, Customized
Asian
|
19 Participants
n=202 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
11 Participants
n=202 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=202 Participants
|
|
Race/Ethnicity, Customized
White
|
327 Participants
n=202 Participants
|
|
Race/Ethnicity, Customized
Other or Mixed
|
4 Participants
n=202 Participants
|
|
Race/Ethnicity, Customized
Not Reported
|
42 Participants
n=202 Participants
|
|
Race/Ethnicity, Customized
Unknown
|
7 Participants
n=202 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
|
60 Participants
n=202 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
303 Participants
n=202 Participants
|
PRIMARY outcome
Timeframe: From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)Population: Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. An AE can, therefore, be any unfavorable and unintended sign (including an abnormal, clinically significant laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not considered related to the medicinal (investigational) product. TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment-emergent. The percentage of participants was rounded to one decimal place.
Outcome measures
| Measure |
Any ZX008 Open Label Dose
n=412 Participants
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
|
75.5 percentage of participants
|
SECONDARY outcome
Timeframe: Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)Population: The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points.
The Clinical Global Impression-Improvement (CGI-I) scale is used to assess the severity of illness and global improvement in patients. Global function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The global CGI-I assessment was conducted by the Parent/Caregiver. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Outcome measures
| Measure |
Any ZX008 Open Label Dose
n=401 Participants
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 6: Very much improved
|
39 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 6: Much improved
|
88 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 6: Minimally improved
|
87 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 6: No change
|
124 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 6: Minimally worse
|
23 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 6: Much worse
|
7 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 6: Very much worse
|
3 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 12: Very much improved
|
42 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 12: Much improved
|
65 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 12: Minimally improved
|
56 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 12: No change
|
77 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 12: Minimally worse
|
17 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 12: Much worse
|
9 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 12: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 18: Very much improved
|
34 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 18: Much improved
|
43 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 18: Minimally improved
|
60 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 18: No change
|
67 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 18: Minimally worse
|
16 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 18: Much worse
|
2 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 18: Very much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 24: Very much improved
|
33 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 24: Much improved
|
46 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 24: Minimally improved
|
41 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 24: No change
|
59 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 24: Minimally worse
|
8 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 24: Much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 24: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 30: Very much improved
|
16 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 30: Much improved
|
41 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 30: Minimally improved
|
34 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 30: No change
|
47 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 30: Minimally worse
|
9 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 30: Much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 30: Very much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 36: Very much improved
|
16 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 36: Much improved
|
31 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 36: Minimally improved
|
29 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 36: No change
|
39 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 36: Minimally worse
|
10 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 36: Much worse
|
3 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Month 36: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Last On-Treatment Visit: Very much improved
|
71 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Last On-Treatment Visit: Much improved
|
109 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Last On-Treatment Visit: Minimally improved
|
85 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Last On-Treatment Visit: No change
|
108 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Last On-Treatment Visit: Minimally worse
|
21 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Last On-Treatment Visit: Much worse
|
7 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver
Last On-Treatment Visit: Very much worse
|
0 Participants
|
SECONDARY outcome
Timeframe: Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)Population: The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points.
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Global function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The global CGI-I assessment was conducted by the Investigator. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Outcome measures
| Measure |
Any ZX008 Open Label Dose
n=401 Participants
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 6: Very much improved
|
23 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 6: Much improved
|
86 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 6: Minimally improved
|
85 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 6: No change
|
141 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 6: Minimally worse
|
26 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 6: Much worse
|
4 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 6: Very much worse
|
2 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 12: Very much improved
|
28 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 12: Much improved
|
63 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 12: Minimally improved
|
66 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 12: No change
|
87 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 12: Minimally worse
|
17 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 12: Much worse
|
3 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 12: Very much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 18: Very much improved
|
23 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 18: Much improved
|
45 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 18: Minimally improved
|
58 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 18: No change
|
86 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 18: Minimally worse
|
12 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 18: Much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 18: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 24: Very much improved
|
20 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 24: Much improved
|
54 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 24: Minimally improved
|
37 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 24: No change
|
72 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 24: Minimally worse
|
5 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 24: Much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 24: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 30: Very much improved
|
11 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 30: Much improved
|
44 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 30: Minimally improved
|
34 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 30: No change
|
56 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 30: Minimally worse
|
3 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 30: Much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 30: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 36: Very much improved
|
7 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 36: Much improved
|
33 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 36: Minimally improved
|
25 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 36: No change
|
52 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 36: Minimally worse
|
5 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 36: Much worse
|
2 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Month 36: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Last On-Treatment Visit: Very much improved
|
33 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Last On-Treatment Visit: Much improved
|
108 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Last On-Treatment Visit: Minimally improved
|
101 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Last On-Treatment Visit: No change
|
134 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Last On-Treatment Visit: Minimally worse
|
19 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Last On-Treatment Visit: Much worse
|
5 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator
Last On-Treatment Visit: Very much worse
|
1 Participants
|
SECONDARY outcome
Timeframe: Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)Population: The modified Intent-to-Treat (mITT) analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the open-label extension (OLE) in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points.
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Cognitive function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of cognition was conducted by the Parent/Caregiver. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Outcome measures
| Measure |
Any ZX008 Open Label Dose
n=401 Participants
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 36: No change
|
49 Participants
Interval 29.8 to 47.3
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 36: Very much improved
|
6 Participants
Interval 1.7 to 9.9
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 36: Much improved
|
32 Participants
Interval 17.8 to 33.4
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 36: Minimally improved
|
29 Participants
Interval 15.7 to 30.9
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 36: Minimally worse
|
11 Participants
Interval 4.4 to 14.9
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 36: Much worse
|
1 Participants
Interval 0.0 to 4.3
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 24: Much improved
|
35 Participants
Interval 13.3 to 24.9
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 24: Minimally worse
|
6 Participants
Interval 1.2 to 6.8
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 18: Much improved
|
43 Participants
Interval 14.3 to 25.0
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 18: Minimally improved
|
69 Participants
Interval 24.8 to 37.3
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 18: No change
|
86 Participants
Interval 32.0 to 45.1
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 18: Minimally worse
|
6 Participants
Interval 1.0 to 5.7
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 18: Much worse
|
2 Participants
Interval 0.1 to 3.2
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 18: Very much worse
|
0 Participants
Interval 0.0 to 1.6
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 6: Very much improved
|
26 Participants
Interval 4.6 to 10.1
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 6: Much improved
|
75 Participants
Interval 16.3 to 24.7
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 6: Minimally improved
|
87 Participants
Interval 19.3 to 28.2
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 6: No change
|
161 Participants
Interval 38.4 to 48.7
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 6: Minimally worse
|
15 Participants
Interval 2.3 to 6.6
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 6: Much worse
|
4 Participants
Interval 0.3 to 2.7
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 6: Very much worse
|
2 Participants
Interval 0.1 to 1.9
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 12: Very much improved
|
17 Participants
Interval 3.8 to 10.1
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 12: Much improved
|
54 Participants
Interval 15.7 to 25.7
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 12: Minimally improved
|
78 Participants
Interval 24.0 to 35.3
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 12: No change
|
108 Participants
Interval 34.8 to 46.9
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 12: Minimally worse
|
5 Participants
Interval 0.6 to 4.3
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 12: Much worse
|
3 Participants
Interval 0.2 to 3.3
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 12: Very much worse
|
0 Participants
Interval 0.0 to 1.4
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 18: Very much improved
|
18 Participants
Interval 4.8 to 12.4
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 24: Very much improved
|
14 Participants
Interval 4.1 to 12.2
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 24: Minimally improved
|
59 Participants
Interval 24.8 to 38.5
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 24: No change
|
73 Participants
Interval 31.8 to 46.2
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 24: Much worse
|
1 Participants
Interval 0.0 to 2.9
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 24: Very much worse
|
0 Participants
Interval 0.0 to 1.9
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 30: Very much improved
|
7 Participants
Interval 1.9 to 9.4
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 30: Much improved
|
31 Participants
Interval 14.6 to 28.2
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 30: Minimally improved
|
41 Participants
Interval 20.5 to 35.4
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 30: No change
|
62 Participants
Interval 33.6 to 50.0
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 30: Minimally worse
|
6 Participants
Interval 1.5 to 8.6
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 30: Much worse
|
1 Participants
Interval 0.0 to 3.7
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 30: Very much worse
|
1 Participants
Interval 0.0 to 3.7
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Month 36: Very much worse
|
0 Participants
Interval 0.0 to 2.8
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Last On-Treatment Visit: Very much improved
|
33 Participants
Interval 5.7 to 11.4
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Last On-Treatment Visit: Much improved
|
98 Participants
Interval 20.3 to 28.9
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Last On-Treatment Visit: Minimally improved
|
101 Participants
Interval 21.0 to 29.7
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Last On-Treatment Visit: No change
|
148 Participants
Interval 32.2 to 41.8
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Last On-Treatment Visit: Minimally worse
|
16 Participants
Interval 2.3 to 6.4
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Last On-Treatment Visit: Much worse
|
5 Participants
Interval 0.4 to 2.9
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver
Last On-Treatment Visit: Very much worse
|
0 Participants
Interval 0.0 to 0.9
|
SECONDARY outcome
Timeframe: Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)Population: The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points.
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Behavior is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of behavior was conducted by the Parent/Caregiver. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Outcome measures
| Measure |
Any ZX008 Open Label Dose
n=401 Participants
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 6: Much worse
|
12 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 6: Very much worse
|
3 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 12: Very much improved
|
9 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 12: Much improved
|
46 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 12: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 18: Very much improved
|
13 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 30: Very much improved
|
8 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 30: Much improved
|
26 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 30: Minimally worse
|
15 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 6: Minimally improved
|
75 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 6: No change
|
174 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 12: Much worse
|
11 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 6: Minimally worse
|
35 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 24: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 12: Minimally improved
|
51 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 12: No change
|
120 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 12: Minimally worse
|
28 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 18: Much improved
|
37 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 18: Minimally improved
|
42 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 18: No change
|
100 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 18: Minimally worse
|
27 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 18: Much worse
|
4 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 18: Very much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 24: Very much improved
|
13 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 24: Much improved
|
31 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 24: Minimally worse
|
16 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 24: Much worse
|
9 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 30: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 36: Minimally improved
|
28 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 36: No change
|
58 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 36: Minimally worse
|
10 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 36: Very much improved
|
7 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 6: Much improved
|
51 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 6: Very much improved
|
20 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 36: Much improved
|
18 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 30: Much worse
|
4 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 24: Minimally improved
|
36 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 24: No change
|
83 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 30: Minimally improved
|
27 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 30: No change
|
69 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 36: Much worse
|
6 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Month 36: Very much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Last On-Treatment Visit: Very much improved
|
24 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Last On-Treatment Visit: Much improved
|
73 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Last On-Treatment Visit: Minimally improved
|
83 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Last On-Treatment Visit: No change
|
169 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Last On-Treatment Visit: Minimally worse
|
36 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Last On-Treatment Visit: Much worse
|
15 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver
Last On-Treatment Visit: Very much worse
|
1 Participants
|
SECONDARY outcome
Timeframe: Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)Population: The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points.
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Motor abilities function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of motor abilities was conducted by the Parent/Caregiver. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Outcome measures
| Measure |
Any ZX008 Open Label Dose
n=401 Participants
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 6: Very much improved
|
17 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 6: Much improved
|
45 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 6: Minimally improved
|
86 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 6: No change
|
190 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 6: Minimally worse
|
26 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 6: Much worse
|
4 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 6: Very much worse
|
2 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 12: Very much improved
|
14 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 12: Much improved
|
31 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 12: Minimally improved
|
65 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 12: No change
|
134 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 12: Minimally worse
|
14 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 12: Much worse
|
6 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 12: Very much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 18: Very much improved
|
10 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 18: Much improved
|
31 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 18: Minimally improved
|
48 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 18: No change
|
114 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 18: Minimally worse
|
16 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 18: Much worse
|
4 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 18: Very much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 24: Very much improved
|
16 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 24: Much improved
|
19 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 24: Minimally improved
|
42 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 24: No change
|
97 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 24: Minimally worse
|
13 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 24: Much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 24: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 30: Very much improved
|
7 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 30: Much improved
|
22 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 30: Minimally improved
|
27 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 30: No change
|
77 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 30: Minimally worse
|
13 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 30: Much worse
|
2 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 30: Very much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 36: Very much improved
|
6 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 36: Much improved
|
16 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 36: Minimally improved
|
22 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 36: No change
|
67 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 36: Minimally worse
|
9 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 36: Much worse
|
7 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Month 36: Very much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Last On-Treatment Visit: Very much improved
|
24 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Last On-Treatment Visit: Much improved
|
55 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Last On-Treatment Visit: Minimally improved
|
94 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Last On-Treatment Visit: No change
|
191 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Last On-Treatment Visit: Minimally worse
|
25 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Last On-Treatment Visit: Much worse
|
12 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver
Last On-Treatment Visit: Very much worse
|
0 Participants
|
SECONDARY outcome
Timeframe: Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)Population: The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points.
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Cognitive function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of cognition was conducted by the Investigator. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Outcome measures
| Measure |
Any ZX008 Open Label Dose
n=399 Participants
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 6: Very much improved
|
13 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 6: Much improved
|
54 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 6: Minimally improved
|
101 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 6: No change
|
184 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 6: Minimally worse
|
9 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 6: Much worse
|
2 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 6: Very much worse
|
2 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 12: Very much improved
|
13 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 12: Much improved
|
39 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 12: Minimally improved
|
79 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 12: No change
|
128 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 12: Minimally worse
|
4 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 12: Much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 12: Very much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 18: Very much improved
|
13 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 18: Much improved
|
32 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 18: Minimally improved
|
53 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 18: No change
|
118 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 18: Minimally worse
|
10 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 18: Much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 18: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 24: Very much improved
|
9 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 24: Much improved
|
33 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 24: Minimally improved
|
55 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 24: No change
|
88 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 24: Minimally worse
|
3 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 24: Much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 24: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 30: Very much improved
|
4 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 30: Much improved
|
25 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 30: Minimally improved
|
36 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 30: No change
|
81 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 30: Minimally worse
|
3 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 30: Much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 30: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 36: Very much improved
|
4 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 36: Much improved
|
14 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 36: Minimally improved
|
34 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 36: No change
|
65 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 36: Minimally worse
|
7 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 36: Much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Month 36: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Last On-Treatment Visit: Very much improved
|
16 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Last On-Treatment Visit: Much improved
|
64 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Last On-Treatment Visit: Minimally improved
|
103 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Last On-Treatment Visit: No change
|
201 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Last On-Treatment Visit: Minimally worse
|
12 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Last On-Treatment Visit: Much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator
Last On-Treatment Visit: Very much worse
|
2 Participants
|
SECONDARY outcome
Timeframe: Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)Population: The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points.
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Behavior is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of behavior was conducted by the Investigator. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Outcome measures
| Measure |
Any ZX008 Open Label Dose
n=399 Participants
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 6: Very much improved
|
10 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 6: Much improved
|
42 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 6: Minimally improved
|
80 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 6: No change
|
200 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 6: Minimally worse
|
27 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 6: Much worse
|
5 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 6: Very much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 12: Very much improved
|
11 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 12: Much improved
|
35 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 12: Minimally improved
|
54 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 12: No change
|
144 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 12: Minimally worse
|
17 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 12: Much worse
|
3 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 12: Very much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 18: Very much improved
|
7 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 18: Much improved
|
32 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 18: Minimally improved
|
41 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 18: No change
|
122 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 18: Minimally worse
|
20 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 18: Much worse
|
3 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 18: Very much worse
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 24: Very much improved
|
11 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 24: Much improved
|
28 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 24: Minimally improved
|
36 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 24: No change
|
95 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 24: Minimally worse
|
17 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 24: Much worse
|
2 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 24: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 30: Very much improved
|
2 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 30: Much improved
|
28 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 30: Minimally improved
|
25 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 30: No change
|
77 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 30: Minimally worse
|
15 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 30: Much worse
|
2 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 30: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 36: Very much improved
|
1 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 36: Much improved
|
19 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 36: Minimally improved
|
28 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 36: No change
|
63 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 36: Minimally worse
|
11 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 36: Much worse
|
2 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Month 36: Very much worse
|
0 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Last On-Treatment Visit: Very much improved
|
14 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Last On-Treatment Visit: Much improved
|
57 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Last On-Treatment Visit: Minimally improved
|
74 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Last On-Treatment Visit: No change
|
229 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Last On-Treatment Visit: Minimally worse
|
20 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Last On-Treatment Visit: Much worse
|
3 Participants
|
|
Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator
Last On-Treatment Visit: Very much worse
|
2 Participants
|
SECONDARY outcome
Timeframe: Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)Population: The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points.
The CGI-I scale is used to assess the severity of illness and global improvement in patients. Motor abilities is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of motor abilities was conducted by the Investigator. The number of participants in each of the 7 categories of the CGI-I for each visit is reported.
Outcome measures
| Measure |
Any ZX008 Open Label Dose
n=399 Participants
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 6: Very much improved
|
10 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 6: Much improved
|
33 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 6: Minimally improved
|
81 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 6: No change
|
221 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 6: Minimally worse
|
17 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 6: Much worse
|
1 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 6: Very much worse
|
2 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 12: Very much improved
|
13 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 12: Much improved
|
29 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 12: Minimally improved
|
52 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 12: No change
|
159 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 12: Minimally worse
|
10 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 12: Much worse
|
0 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 12: Very much worse
|
2 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 18: Very much improved
|
9 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 18: Much improved
|
19 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 18: Minimally improved
|
42 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 18: No change
|
133 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 18: Minimally worse
|
21 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 18: Much worse
|
2 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 18: Very much worse
|
0 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 24: Very much improved
|
7 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 24: Much improved
|
24 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 24: Minimally improved
|
43 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 24: No change
|
99 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 24: Minimally worse
|
13 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 24: Much worse
|
3 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 24: Very much worse
|
0 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 30: Very much improved
|
4 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 30: Much improved
|
19 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 30: Minimally improved
|
29 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 30: No change
|
86 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 30: Minimally worse
|
9 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 30: Much worse
|
1 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 30: Very much worse
|
1 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 36: Very much improved
|
2 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 36: Much improved
|
9 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 36: Minimally improved
|
27 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 36: No change
|
74 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 36: Minimally worse
|
8 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 36: Much worse
|
4 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Month 36: Very much worse
|
0 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Last On-Treatment Visit: Very much improved
|
12 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Last On-Treatment Visit: Much improved
|
39 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Last On-Treatment Visit: Minimally improved
|
89 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Last On-Treatment Visit: No change
|
238 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Last On-Treatment Visit: Minimally worse
|
16 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Last On-Treatment Visit: Much worse
|
4 Participants
|
|
Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator
Last On-Treatment Visit: Very much worse
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49)Population: The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points.
The change in seizure burden from the previous visit was assessed by the investigator using following categories: \<25%, ≥25%, ≥50%, ≥75%, 100% (i.e., seizure-free) improvement. Improvement in seizure burden at Baseline was based on comparison with the participant's last visit in the feeder studies (ZX008-1601, ZX008-1503, and ZX008-1602). Number of participants in each of the 5 categories is reported for each visit as compared to last visit.
Outcome measures
| Measure |
Any ZX008 Open Label Dose
n=410 Participants
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Last On-Treatment Visit: >=50%
|
29 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Baseline: <25%
|
242 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Baseline: >=25%
|
67 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Baseline: >=50%
|
42 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Baseline: >=75%
|
35 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Baseline: 100%
|
12 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 6: <25%
|
262 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 6: >=25%
|
51 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 6: >=50%
|
39 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 6: >=75%
|
21 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 6: 100%
|
8 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 12: <25%
|
191 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 12: >=25%
|
33 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 12: >=50%
|
31 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 12: >=75%
|
11 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 12: 100%
|
6 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 18: <25%
|
175 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 18: >=25%
|
22 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 18: >=50%
|
26 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 18: >=75%
|
3 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 18: 100%
|
6 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 24: <25%
|
144 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 24: >=25%
|
24 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 24: >=50%
|
15 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 24: >=75%
|
6 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 24: 100%
|
4 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 30: <25%
|
115 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 30: >=25%
|
14 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 30: >=50%
|
11 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 30: >=75%
|
7 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 30: 100%
|
1 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 36: <25%
|
99 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 36: >=25%
|
13 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 36: >=50%
|
9 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 36: >=75%
|
6 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Month 36: 100%
|
1 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Last On-Treatment Visit: <25%
|
315 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Last On-Treatment Visit: >=25%
|
41 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Last On-Treatment Visit: >=75%
|
17 Participants
|
|
Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator
Last On-Treatment Visit: 100%
|
8 Participants
|
Adverse Events
Any ZX008 Open Label Dose
Serious events: 67 serious events
Other events: 204 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
Any ZX008 Open Label Dose
n=412 participants at risk
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.24%
1/412 • Number of events 3 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Cardiac disorders
Cardiac arrest
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Congenital, familial and genetic disorders
Non-compaction cardiomyopathy
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Congenital, familial and genetic disorders
Pyloric stenosis
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Eye disorders
Vision blurred
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Gastrointestinal disorders
Abdominal distension
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Gastrointestinal disorders
Constipation
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Gastrointestinal disorders
Intestinal obstruction
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
General disorders
Asthenia
|
0.49%
2/412 • Number of events 2 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
General disorders
Complication associated with device
|
0.24%
1/412 • Number of events 2 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
General disorders
Gait disturbance
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
General disorders
Gait inability
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
General disorders
Influenza like illness
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
General disorders
Pyrexia
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Pneumonia
|
1.9%
8/412 • Number of events 8 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Appendicitis
|
0.49%
2/412 • Number of events 2 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Corona virus infection
|
0.49%
2/412 • Number of events 2 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Pneumonia viral
|
0.49%
2/412 • Number of events 2 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Urinary tract infection
|
0.49%
2/412 • Number of events 2 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Sepsis
|
0.24%
1/412 • Number of events 2 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Cellulitis of male external genital organ
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Clostridium difficile colitis
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Dengue haemorrhagic fever
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Ear infection
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Legionella infection
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Nasal abscess
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Pneumonia bacterial
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Pneumonia mycoplasmal
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Tonsillitis
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Injury, poisoning and procedural complications
Animal bite
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Injury, poisoning and procedural complications
Craniocerebral injury
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Injury, poisoning and procedural complications
Craniofacial fracture
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Injury, poisoning and procedural complications
Extradural haematoma
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Injury, poisoning and procedural complications
Jaw fracture
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Injury, poisoning and procedural complications
Lower limb fracture
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Injury, poisoning and procedural complications
Post procedural haemorrhage
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Injury, poisoning and procedural complications
Spinal column injury
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Injury, poisoning and procedural complications
Spinal compression fracture
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Injury, poisoning and procedural complications
Traumatic renal injury
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Injury, poisoning and procedural complications
Upper limb fracture
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Investigations
Weight decreased
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.73%
3/412 • Number of events 3 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Metabolism and nutrition disorders
Diabetic ketoacidosis
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Metabolism and nutrition disorders
Hypophagia
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Seizure
|
4.1%
17/412 • Number of events 22 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Status epilepticus
|
2.2%
9/412 • Number of events 10 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Seizure cluster
|
0.49%
2/412 • Number of events 2 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Epilepsy
|
0.24%
1/412 • Number of events 4 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Altered state of consciousness
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Generalised tonic-clonic seizure
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Haemorrhage intracranial
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Headache
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Parkinsonism
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Petit mal epilepsy
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Somnolence
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Thalamus haemorrhage
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Tremor
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Psychiatric disorders
Aggression
|
0.49%
2/412 • Number of events 2 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Psychiatric disorders
Agitation
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Psychiatric disorders
Hallucination
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Psychiatric disorders
Intentional self-injury
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Psychiatric disorders
Psychotic disorder
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
1.2%
5/412 • Number of events 11 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Skin and subcutaneous tissue disorders
Angioedema
|
0.24%
1/412 • Number of events 1 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
Other adverse events
| Measure |
Any ZX008 Open Label Dose
n=412 participants at risk
Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor.
|
|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
7.0%
29/412 • Number of events 36 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
General disorders
Pyrexia
|
12.9%
53/412 • Number of events 86 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Corona virus infection
|
20.4%
84/412 • Number of events 99 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Nasopharyngitis
|
11.9%
49/412 • Number of events 69 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Infections and infestations
Upper respiratory tract infection
|
5.6%
23/412 • Number of events 28 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
6.1%
25/412 • Number of events 30 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
|
|
Nervous system disorders
Seizure
|
12.6%
52/412 • Number of events 69 • From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment emergent. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60