Trial Outcomes & Findings for Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study (NCT NCT03563053)
NCT ID: NCT03563053
Last Updated: 2024-10-08
Results Overview
Assessment of TEAEs, treatment-emergent serious adverse events (TESAE), and adverse events of special interest (AESI) were performed throughout the study, from the time of signing of the ICF at Baseline Visit through to the Final Study Visit (Month 12 or early discontinuation). All patients were to be followed up through 30 days after the Final Visit (Month 12 or early discontinuation) or at least 60 days after the final infusion, whichever was longer.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
104 participants
Primary outcome timeframe
From Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months)
Results posted on
2024-10-08
Participant Flow
Out of a total of 108 patients who completed the full treatment period (including placebo treatment) in the ATTeST - IEDAT-02 study, 104 patients were enrolled in the IEDAT-03-2018 study (OLE-IEDAT) and comprised the Total Set. These patients signed the ICF, completed the ATTeST study assessments, and did not present safety contraindication prior continuation with the EryDex treatment.
Participant milestones
| Measure |
Active Drug
\~14-22 mg dexamethasone sodium phosphate (DSP) for 12 months. The duration of EryDex treatment could be extended further and continue until patients eventually withdrew consent, or the Investigator decided to discontinue treatment based on a risk / benefit assessment.
EryDex System: EryDex System is a combination product that is used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EDS) creating the EDS-end product which is infused into the patients.
EryDex treatment consisted of a dose range correspondent to the ATTeST High Dose (obtained by loading 125 mg of DSP to the EryDex process and that, in the ATTeST, resulted in a mean of 17.4 ± 5.4 mg (mean ± standard deviation) of DSP infused to patients) administered via ex vivo encapsulation into EDS that were infused into the patient with A-T. The dose of EryDex treatment was selected to allow collection of long-term safety data on the dose of erythrocyte encapsulated DSP that was considered more effective among the two different doses that were employed in the ATTeST study, based on the analysis of the ATTeST blinded, Phase 3 study data.
|
|---|---|
|
Overall Study
STARTED
|
104
|
|
Overall Study
Total Set
|
104
|
|
Overall Study
Safety Set
|
104
|
|
Overall Study
Full Analysis Set
|
80
|
|
Overall Study
Month 12
|
64
|
|
Overall Study
Month 24
|
33
|
|
Overall Study
Month 36
|
21
|
|
Overall Study
Month 48
|
7
|
|
Overall Study
Month 50.5
|
1
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
104
|
Reasons for withdrawal
| Measure |
Active Drug
\~14-22 mg dexamethasone sodium phosphate (DSP) for 12 months. The duration of EryDex treatment could be extended further and continue until patients eventually withdrew consent, or the Investigator decided to discontinue treatment based on a risk / benefit assessment.
EryDex System: EryDex System is a combination product that is used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EDS) creating the EDS-end product which is infused into the patients.
EryDex treatment consisted of a dose range correspondent to the ATTeST High Dose (obtained by loading 125 mg of DSP to the EryDex process and that, in the ATTeST, resulted in a mean of 17.4 ± 5.4 mg (mean ± standard deviation) of DSP infused to patients) administered via ex vivo encapsulation into EDS that were infused into the patient with A-T. The dose of EryDex treatment was selected to allow collection of long-term safety data on the dose of erythrocyte encapsulated DSP that was considered more effective among the two different doses that were employed in the ATTeST study, based on the analysis of the ATTeST blinded, Phase 3 study data.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
17
|
|
Overall Study
Due to the COVID-19 pandemic
|
12
|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
study terminated by sponsor
|
71
|
Baseline Characteristics
Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study
Baseline characteristics by cohort
| Measure |
Overall Population (Total Set)
n=104 Participants
The Total Set (N=104) included all patients who provided informed consent or assent as documented by a date of informed consent or date of assent on the 'Informed Consent' Electronic Case Report form (eCRF) page.
|
|---|---|
|
Age, Categorical
<=18 years
|
104 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
11.4 years
STANDARD_DEVIATION 4.57 • n=5 Participants
|
|
Sex: Female, Male
Female
|
49 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
55 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
99 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
21 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
80 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Belgium
|
3 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
25 participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
10 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Australia
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Tunisia
|
7 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
13 participants
n=5 Participants
|
|
Region of Enrollment
India
|
15 participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
11 participants
n=5 Participants
|
|
Region of Enrollment
Norway
|
5 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
11 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months)Population: Safety Population (SAF): the Safety Analysis Set consisted of all patients who provided informed consent or assent and who received any dose of study medication during IEDAT-03-2018 study. The Safety Analysis Set was used for all safety analyses.
Assessment of TEAEs, treatment-emergent serious adverse events (TESAE), and adverse events of special interest (AESI) were performed throughout the study, from the time of signing of the ICF at Baseline Visit through to the Final Study Visit (Month 12 or early discontinuation). All patients were to be followed up through 30 days after the Final Visit (Month 12 or early discontinuation) or at least 60 days after the final infusion, whichever was longer.
Outcome measures
| Measure |
Overall Population (SAF)
n=104 Participants
The Total Set (N=104) included all patients who provided informed consent or assent as documented by a date of informed consent or date of assent on the 'Informed Consent' Electronic Case Report form (eCRF) page.
|
|---|---|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
Number of TEAEs
|
1232 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
Number of Serious TEAEs
|
19 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
Number of TEAE leading to Death
|
0 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
Number of TEAE leading to Permanent Withdrawal of Treatment
|
5 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
Adverse Events During COVID-19 Interruption - On Dose
|
5 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
Adverse Events During COVID-19 Interruption - Off Dose
|
3 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
Adverse Events During COVID-19 Interruption - Restart
|
55 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
TEAE by Worst Intensity - Mild
|
1028 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
TEAE by Worst Intensity - Moderate
|
187 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
TEAE by Worst Intensity - Severe
|
17 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
TEAE by Closest Relationship to Treatment - Probable
|
283 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
TEAE by Closest Relationship to Treatment - Possible
|
167 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
TEAE by Closest Relationship to Treatment - Unlikely
|
178 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
TEAE by Closest Relationship to Treatment - Not related
|
604 Number of events
|
|
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
Number of AESI
|
83 Number of events
|
SECONDARY outcome
Timeframe: From Baseline (Visit 1- Day 0) to Month 36Population: Full Analysis Set Population (FAS): the FAS comprised all patients who entered IEDAT-03-2018 study, had a Baseline Efficacy Assessment in IEDAT-03-2018 and who received at least one dose of study medication and had at least one post-Baseline Efficacy Assessment of the ICARS in this extension study.
The EQ-5D included single item measures of 5 health dimensions: * mobility, * self-care, * usual activities, * pain / discomfort, and * anxiety / depression. The EQ-5D-5L included five levels of severity (i.e., no problems, slight problems, moderate problems, severe problems, and extreme problems) for each of the five EQ-5D dimensions. These levels were scored from 1 = no problems to 5 = extreme problems: from 5, min/worst, to 25, best/max).The EQ-5D results were converted into a continuous index score: as for this index score, the closer the value is to 1, the better is the health status. The efficacy data focus mainly up to Month 36 as afterwards the number of patients / assessments dropped below 20% of the initial sample size, which was required for the efficacy evaluations.
Outcome measures
| Measure |
Overall Population (SAF)
n=80 Participants
The Total Set (N=104) included all patients who provided informed consent or assent as documented by a date of informed consent or date of assent on the 'Informed Consent' Electronic Case Report form (eCRF) page.
|
|---|---|
|
Change From Baseline in Quality of Life Using EQ-5D-5L Scale to Month 36
to Month 6
|
0.015 score on a scale
Standard Deviation 0.1131
|
|
Change From Baseline in Quality of Life Using EQ-5D-5L Scale to Month 36
to Month 12
|
-0.022 score on a scale
Standard Deviation 0.1145
|
|
Change From Baseline in Quality of Life Using EQ-5D-5L Scale to Month 36
to Month 18
|
0.007 score on a scale
Standard Deviation 0.1233
|
|
Change From Baseline in Quality of Life Using EQ-5D-5L Scale to Month 36
to Month 24
|
0.011 score on a scale
Standard Deviation 0.1746
|
|
Change From Baseline in Quality of Life Using EQ-5D-5L Scale to Month 36
to Month 30
|
-0.021 score on a scale
Standard Deviation 0.1117
|
|
Change From Baseline in Quality of Life Using EQ-5D-5L Scale to Month 36
to Month 36
|
0.011 score on a scale
Standard Deviation 0.1242
|
SECONDARY outcome
Timeframe: From Baseline (Visit 1- Day 0) to Month 36Population: The FAS comprised all patients who entered IEDAT-03-2018 study, had a Baseline Efficacy Assessment in IEDAT-03-2018 and who received at least one dose of study medication and had at least one post-Baseline Efficacy Assessment of the ICARS in this extension study.
The CGI-C scale assesses the change in the patient's clinical status from baseline using a 7-point scale, ranging from 1 (very much improved) to 7 (very much worse), with a score of 4 indicating no change. Clinicians were required to conduct a full clinical interview and examination of the patient. The interview and examination assessed various aspects of the patient's appearance (grooming, evidence of falls, etc.), ataxia, cognition (orientation, calculation ability, language, ability to follow commands, memory, etc.), apraxia, dysarthria, extrapyramidal motor symptoms, activities of daily living, and mood. The higher the score the worse the outcome. The efficacy data focus mainly up to Month 36 as afterwards the number of patients / assessments dropped below 20% of the initial sample size, which was required for the efficacy evaluations.
Outcome measures
| Measure |
Overall Population (SAF)
n=80 Participants
The Total Set (N=104) included all patients who provided informed consent or assent as documented by a date of informed consent or date of assent on the 'Informed Consent' Electronic Case Report form (eCRF) page.
|
|---|---|
|
Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
Month 6 - Improved (Score 1-3)
|
9 Participants
|
|
Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
Month 6 - No change or Worsened (Score 4-7)
|
68 Participants
|
|
Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
Month 12 - Improved (Score 1-3)
|
14 Participants
|
|
Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
Month 12 - No change or Worsened (Score 4-7)
|
45 Participants
|
|
Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
Month 18 - Improved (Score 1-3)
|
6 Participants
|
|
Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
Month 18 - No change or Worsened (Score 4-7)
|
41 Participants
|
|
Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
Month 24 - Improved (Score 1-3)
|
5 Participants
|
|
Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
Month 24 - No change or Worsened (Score 4-7)
|
26 Participants
|
|
Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
Month 30 - Improved (Score 1-3)
|
2 Participants
|
|
Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
Month 30 - No change or Worsened (Score 4-7)
|
17 Participants
|
|
Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
Month 36 - Improved (Score 1-3)
|
3 Participants
|
|
Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
Month 36 - No change or Worsened (Score 4-7)
|
16 Participants
|
SECONDARY outcome
Timeframe: From Baseline (Visit 1- Day 0) to Month 36Population: The FAS comprised all patients who entered IEDAT-03-2018 study, had a Baseline Efficacy Assessment in IEDAT-03-2018 and who received at least one dose of study medication and had at least one post-Baseline Efficacy Assessment of the ICARS in this extension study.
The CGI-S scale measures global severity of illness at a given point in time, and is usually rated on a 7-point, Likert-type scale ranging from 1 (normal, not ill at all) to 7 (among the most extremely ill patients). No version of the CGI-S exists which has been specifically adapted for use in patients with A-T; therefore, a 5-point version was developed that considered the severity of the following symptoms of A-T: ataxia (walking), dysarthria, dysmetria, extrapyramidal symptoms (chorea, myoclonus, dystonia, and tremor), and eye movements. Ratings of none (0), mild (1), moderate (2), severe (3), and very severe (4) were selected based on the level of symptomatology. The higher the score the worse the outcome. The efficacy data focus mainly up to Month 36 as afterwards the number of patients / assessments dropped below 20% of the initial sample size, which was required for the efficacy evaluations.
Outcome measures
| Measure |
Overall Population (SAF)
n=80 Participants
The Total Set (N=104) included all patients who provided informed consent or assent as documented by a date of informed consent or date of assent on the 'Informed Consent' Electronic Case Report form (eCRF) page.
|
|---|---|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Baseline - CGI-S Score - 0
|
2 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Baseline - CGI-S Score - 1
|
18 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Baseline - CGI-S Score - 2
|
31 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Baseline - CGI-S Score - 3
|
23 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Baseline - CGI-S Score - 4
|
0 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 6 - CGI-S Score - 0
|
1 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 6 - CGI-S Score - 1
|
20 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 6 - CGI-S Score - 2
|
30 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 6 - CGI-S Score - 3
|
22 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 6 - CGI-S Score - 4
|
3 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 12 - CGI-S Score - 0
|
0 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 12 - CGI-S Score - 1
|
12 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 12 - CGI-S Score - 2
|
28 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 12 - CGI-S Score - 3
|
17 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 12 - CGI-S Score - 4
|
2 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 18 - CGI-S Score - 0
|
0 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 18 - CGI-S Score - 1
|
13 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 18 - CGI-S Score - 2
|
21 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 18 - CGI-S Score - 3
|
12 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 18 - CGI-S Score - 4
|
1 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 24 - CGI-S Score - 0
|
0 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 24 - CGI-S Score - 1
|
10 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 24 - CGI-S Score - 2
|
10 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 24 - CGI-S Score - 3
|
9 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 24 - CGI-S Score - 4
|
2 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 30 - CGI-S Score - 0
|
0 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 30 - CGI-S Score - 1
|
5 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 30 - CGI-S Score - 2
|
10 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 30 - CGI-S Score - 3
|
4 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 30 - CGI-S Score - 4
|
0 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 36 - CGI-S Score - 0
|
0 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 36 - CGI-S Score - 1
|
3 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 36 - CGI-S Score - 2
|
10 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 36 - CGI-S Score - 3
|
6 Participants
|
|
Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
Month 36 - CGI-S Score - 4
|
0 Participants
|
SECONDARY outcome
Timeframe: From Baseline (Visit 1- Day 0) to Month 36Population: The FAS comprised all patients who entered IEDAT-03-2018 study, had a Baseline Efficacy Assessment in IEDAT-03-2018 and who received at least one dose of study medication and had at least one post-Baseline Efficacy Assessment of the ICARS in this extension study.
The International Cooperative Ataxia Rating Scale (ICARS) was an assessment of the degree of impairment in patients with cerebellar ataxia and was administered in its entirety; however, the primary efficacy assessment was based on the modified (m)ICARS, which excluded the Oculomotor domain (items 17 to 19) and items 8 to 12 of the Kinetic Functions domain of the ICARS. The mICARS was a 54 points maximum score (min 0) questionnaire divided into 3 sections: * Posture and Gait Disturbance section-7 items (min score 0, max score 34) * Kinetic Function-2 items (min 0, max 12) * Speech Disorder- 2 items (min 0, max 8). An higher scores - both for total and subscores - indicate a higher level of disease impairment. The subscores are added to give the total score. The efficacy data focus mainly up to Month 36 as afterwards the number of patients / assessments dropped below 20% of the initial sample size, which was required for the efficacy evaluations.
Outcome measures
| Measure |
Overall Population (SAF)
n=80 Participants
The Total Set (N=104) included all patients who provided informed consent or assent as documented by a date of informed consent or date of assent on the 'Informed Consent' Electronic Case Report form (eCRF) page.
|
|---|---|
|
Change From Baseline of the Modified International Cooperative Ataxia Rating Scale (mICARS) Until Month 36
to Month 6
|
0.7 score on a scale
Standard Deviation 4.09
|
|
Change From Baseline of the Modified International Cooperative Ataxia Rating Scale (mICARS) Until Month 36
to Month 12
|
1.5 score on a scale
Standard Deviation 3.36
|
|
Change From Baseline of the Modified International Cooperative Ataxia Rating Scale (mICARS) Until Month 36
to Month 18
|
2.7 score on a scale
Standard Deviation 4.28
|
|
Change From Baseline of the Modified International Cooperative Ataxia Rating Scale (mICARS) Until Month 36
to Month 24
|
3.9 score on a scale
Standard Deviation 4.96
|
|
Change From Baseline of the Modified International Cooperative Ataxia Rating Scale (mICARS) Until Month 36
to Month 30
|
4.5 score on a scale
Standard Deviation 5.41
|
|
Change From Baseline of the Modified International Cooperative Ataxia Rating Scale (mICARS) Until Month 36
to Month 36
|
34.0 score on a scale
Standard Deviation 5.17
|
Adverse Events
Overall Population (SAF)
Serious events: 12 serious events
Other events: 98 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Overall Population (SAF)
n=104 participants at risk
The Safety Analysis Set consisted of all patients who provided informed consent or assent and who received any dose of study medication during IEDAT-03-2018 study . The Safety Analysis Set was used for all safety analyses.
|
|---|---|
|
Infections and infestations
Lower respiratory tract infection
|
0.96%
1/104 • Number of events 1 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Infections and infestations
Pneumonia
|
0.96%
1/104 • Number of events 1 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Infections and infestations
Viral infection
|
0.96%
1/104 • Number of events 1 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
B-Cell limphoma
|
0.96%
1/104 • Number of events 1 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hodgkin's Disease
|
0.96%
1/104 • Number of events 1 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Odontogenic cyst
|
0.96%
1/104 • Number of events 1 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Injury, poisoning and procedural complications
Forearm fracture
|
1.9%
2/104 • Number of events 2 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Blood and lymphatic system disorders
Trombocytopenic Purpura
|
0.96%
1/104 • Number of events 5 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Gastrointestinal disorders
Dysphagia
|
0.96%
1/104 • Number of events 1 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
General disorders
Pyrexia
|
0.96%
1/104 • Number of events 2 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Nervous system disorders
Central nervous system lesion
|
0.96%
1/104 • Number of events 1 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal swelling
|
0.96%
1/104 • Number of events 1 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Surgical and medical procedures
Gastrointestinal Tube Insertion
|
0.96%
1/104 • Number of events 1 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
Other adverse events
| Measure |
Overall Population (SAF)
n=104 participants at risk
The Safety Analysis Set consisted of all patients who provided informed consent or assent and who received any dose of study medication during IEDAT-03-2018 study . The Safety Analysis Set was used for all safety analyses.
|
|---|---|
|
Infections and infestations
Upper respiratory tract infection
|
22.1%
23/104 • Number of events 36 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Infections and infestations
Nasopharyngitis
|
20.2%
21/104 • Number of events 30 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Infections and infestations
Corona Virus Infection
|
16.3%
17/104 • Number of events 19 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Infections and infestations
Bronchitis
|
9.6%
10/104 • Number of events 15 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Infections and infestations
Urinary Tract Infection
|
5.8%
6/104 • Number of events 7 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
General disorders
Pyrexia
|
31.7%
33/104 • Number of events 54 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
General disorders
Fatigue
|
15.4%
16/104 • Number of events 20 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Injury, poisoning and procedural complications
Infusion Related Reaction
|
26.9%
28/104 • Number of events 199 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Injury, poisoning and procedural complications
Fall
|
6.7%
7/104 • Number of events 10 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Gastrointestinal disorders
Diarrhoea
|
22.1%
23/104 • Number of events 32 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Gastrointestinal disorders
Vomiting
|
19.2%
20/104 • Number of events 38 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Gastrointestinal disorders
Nausea
|
6.7%
7/104 • Number of events 9 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Skin and subcutaneous tissue disorders
Rash
|
8.7%
9/104 • Number of events 11 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
6.7%
7/104 • Number of events 10 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
6.7%
7/104 • Number of events 7 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
18.3%
19/104 • Number of events 42 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
11.5%
12/104 • Number of events 17 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
6.7%
7/104 • Number of events 10 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Investigations
Serum Ferritin Decreased
|
7.7%
8/104 • Number of events 9 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Investigations
Coronavirus Test Positive
|
6.7%
7/104 • Number of events 8 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Investigations
Blood Lactate Dehydrogenase Increased
|
5.8%
6/104 • Number of events 8 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Nervous system disorders
Headache
|
14.4%
15/104 • Number of events 22 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Metabolism and nutrition disorders
Iron Deficiency
|
15.4%
16/104 • Number of events 29 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Blood and lymphatic system disorders
Anaemia
|
5.8%
6/104 • Number of events 9 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Product Issues
Product contamination
|
12.5%
13/104 • Number of events 19 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin Papilloma
|
7.7%
8/104 • Number of events 10 • TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).
All AEs that occurred during this IEDAT-03-2018 study are defined as TEAEs, as all patients received EryDex treatment in the study.
|
Additional Information
Irene Maccabruni, M.Sc.
Quince Therapeutics (former Erydel SpA)
Phone: +39 02 36504470
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place