Trial Outcomes & Findings for Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) (NCT NCT02960217)
NCT ID: NCT02960217
Last Updated: 2020-06-16
Results Overview
The frequency of paroxysmal movement disorders captured as disabling movement disorder events (normalized to a 4-week rate) observed during the Maintenance Phase in participants treated with UX007 versus placebo, as recorded by the subject/caregiver in an event-based daily Glut1 DS symptom diary.
TERMINATED
PHASE3
44 participants
Maintenance Phase (up to Week 22)
2020-06-16
Participant Flow
During a 6-week Run-in Period, participants recorded disabling paroxysmal movement disorder events in a daily electronic Glut1 DS movement disorder diary. One participant was randomized but did not receive any treatment due to a protocol violation; this participant was not included in any analysis population.
Participant milestones
| Measure |
Double-Blind UX007 Followed by Placebo
Double-Blind Maintenance Phase: Participants received UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks. After a washout period of 2 weeks, they then received placebo for 10 weeks.
Participants had the option of rolling into the Open-Label Extension Phase, to continue UX007 treatment (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for up to 3 years.
|
Double-Blind Placebo Followed by UX007
Double-Blind Maintenance Phase: Participants received placebo for 10 weeks. After a washout period of 2 weeks, they then received UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
Participants had the option of rolling into the Open-Label Extension Phase, to continue UX007 treatment (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for up to 3 years.
|
|---|---|---|
|
Maintenance Phase: Treatment Period 1
STARTED
|
22
|
21
|
|
Maintenance Phase: Treatment Period 1
COMPLETED
|
21
|
21
|
|
Maintenance Phase: Treatment Period 1
NOT COMPLETED
|
1
|
0
|
|
Maintenance Phase: Crossover Washout
STARTED
|
21
|
21
|
|
Maintenance Phase: Crossover Washout
COMPLETED
|
21
|
21
|
|
Maintenance Phase: Crossover Washout
NOT COMPLETED
|
0
|
0
|
|
Maintenance Phase: Treatment Period 2
STARTED
|
21
|
21
|
|
Maintenance Phase: Treatment Period 2
COMPLETED
|
20
|
18
|
|
Maintenance Phase: Treatment Period 2
NOT COMPLETED
|
1
|
3
|
|
Open-Label Extension Phase
STARTED
|
20
|
13
|
|
Open-Label Extension Phase
COMPLETED
|
0
|
0
|
|
Open-Label Extension Phase
NOT COMPLETED
|
20
|
13
|
Reasons for withdrawal
| Measure |
Double-Blind UX007 Followed by Placebo
Double-Blind Maintenance Phase: Participants received UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks. After a washout period of 2 weeks, they then received placebo for 10 weeks.
Participants had the option of rolling into the Open-Label Extension Phase, to continue UX007 treatment (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for up to 3 years.
|
Double-Blind Placebo Followed by UX007
Double-Blind Maintenance Phase: Participants received placebo for 10 weeks. After a washout period of 2 weeks, they then received UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
Participants had the option of rolling into the Open-Label Extension Phase, to continue UX007 treatment (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for up to 3 years.
|
|---|---|---|
|
Maintenance Phase: Treatment Period 1
Adverse Event
|
1
|
0
|
|
Maintenance Phase: Treatment Period 2
Adverse Event
|
0
|
1
|
|
Maintenance Phase: Treatment Period 2
Withdrawal by Subject
|
0
|
1
|
|
Maintenance Phase: Treatment Period 2
Lack of Efficacy
|
1
|
0
|
|
Maintenance Phase: Treatment Period 2
Other, Not Specified
|
0
|
1
|
|
Open-Label Extension Phase
Adverse Event
|
1
|
0
|
|
Open-Label Extension Phase
Sponsor Decision
|
18
|
11
|
|
Open-Label Extension Phase
Lack of Efficacy
|
1
|
1
|
|
Open-Label Extension Phase
Other, Not Specified
|
0
|
1
|
Baseline Characteristics
Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Baseline characteristics by cohort
| Measure |
Double-Blind UX007 Followed by Placebo
n=22 Participants
Double-Blind Maintenance Period: Participants received UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks. After a washout period of 2 weeks, they then received placebo for 10 weeks.
Participants had the option of rolling into the Open-Label Extension Period, to continue UX007 treatment for up to 3 years.
|
Double-Blind Placebo Followed by UX007
n=21 Participants
Double-Blind Maintenance Period: Participants received placebo for 10 weeks. After a washout period of 2 weeks, they then received UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
Participants had the option of rolling into the Open-Label Extension Period, to continue UX007 treatment for up to 3 years.
|
Total
n=43 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
23.41 years
STANDARD_DEVIATION 13.156 • n=5 Participants
|
18.37 years
STANDARD_DEVIATION 5.730 • n=7 Participants
|
20.95 years
STANDARD_DEVIATION 10.425 • n=5 Participants
|
|
Age, Customized
< 18 years old
|
7 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Age, Customized
>/= 18 years old
|
15 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
24 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
17 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
34 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
18 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
34 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Missing
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Maintenance Phase (up to Week 22)Population: Full Analysis Set: all randomized participants who received at least 1 dose of study drug.
The frequency of paroxysmal movement disorders captured as disabling movement disorder events (normalized to a 4-week rate) observed during the Maintenance Phase in participants treated with UX007 versus placebo, as recorded by the subject/caregiver in an event-based daily Glut1 DS symptom diary.
Outcome measures
| Measure |
Double-Blind UX007
n=43 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=42 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Maintenance Phase Movement Disorder Frequency
|
14.26 movement disorder events per 4 weeks
Interval 0.0 to 112.0
|
11.81 movement disorder events per 4 weeks
Interval 0.5 to 112.0
|
—
|
PRIMARY outcome
Timeframe: From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, and 305.0 (122.71) days for open-label UX007.Population: Safety Analysis Set: all randomized participants who received at least 1 dose of study drug.
An Adverse Event (AE) was defined as any untoward medical occurrence, whether or not considered drug related. Serious adverse events (SAE) was defined as an AE that at any dose, in the view of either the Investigator or Ultragenyx, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability (substantial disruption of the ability to conduct normal life functions); a congenital anomaly/birth defect; other important medical event. All reported AEs with with a start date that occurred or worsened in severity on or after the first dose of study drug in the corresponding treatment period and before the first dose of study drug in the next treatment period were defined as TEAEs. AEs were graded as 1=mild, 2=moderate, 3=severe, 4=life=threatening, 5=death.
Outcome measures
| Measure |
Double-Blind UX007
n=43 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=42 Participants
Placebo for 10 weeks.
|
Open-Label UX007
n=33 Participants
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs
TEAEs Leading to Study Discontinuation
|
2 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs
TEAEs
|
40 Participants
|
34 Participants
|
28 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs
Serious TEAEs
|
2 Participants
|
1 Participants
|
2 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs
Treatment-Related TEAEs
|
33 Participants
|
19 Participants
|
17 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs
Treatment-Related Serious TEAEs
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs
Grade 3 or 4 TEAEs
|
4 Participants
|
3 Participants
|
3 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs
Grade 4 TEAEs
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs
Gastrointestinal TEAEs
|
32 Participants
|
17 Participants
|
18 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs
TEAEs Leading to Treatment Discontinuation
|
2 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs
TEAEs Leading to Death
|
0 Participants
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Baseline, up to Week 22Population: Safety Analysis Set: all randomized participants who received at least 1 dose of study drug. Participants with a baseline (BL) and postbaseline (PB) assessment.
The C-SSRS is a participant-rated questionnaire to assess suicidal ideation, suicidal behavior, and self-injurious behavior with no suicidal intent (yes or no responses). Positive responses (i.e., 'Yes') to C-SSRS questions correspond to events in these categories with the exception of the category 'No events'. Suicidal ideation includes the following subcategories: passive; active-nonspecific; active-method/no intent/no plan; active-intent/with or without method/no plan; active-method/intent/plan. Suicidal behavior includes the following subcategories: suicide attempt; interrupted attempt; aborted attempt; preparatory actions toward immanent suicidal behaviors; completed suicide. Suicidal ideation and/or suicidal behavior category includes participants with positive responses in the category suicidal ideation and/or suicidal behavior.
Outcome measures
| Measure |
Double-Blind UX007
n=40 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=40 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Baseline and Post-Baseline Columbia Suicide Severity Rating Scale (C-SSRS) Responses During Double-Blind Treatment Period
PB: Suicidal Behavior
|
0 Participants
|
1 Participants
|
—
|
|
Baseline and Post-Baseline Columbia Suicide Severity Rating Scale (C-SSRS) Responses During Double-Blind Treatment Period
BL: No Events
|
38 Participants
|
38 Participants
|
—
|
|
Baseline and Post-Baseline Columbia Suicide Severity Rating Scale (C-SSRS) Responses During Double-Blind Treatment Period
PB: No Events
|
40 Participants
|
39 Participants
|
—
|
|
Baseline and Post-Baseline Columbia Suicide Severity Rating Scale (C-SSRS) Responses During Double-Blind Treatment Period
BL: Suicidal Ideation
|
1 Participants
|
1 Participants
|
—
|
|
Baseline and Post-Baseline Columbia Suicide Severity Rating Scale (C-SSRS) Responses During Double-Blind Treatment Period
PB: Suicidal Ideation
|
0 Participants
|
0 Participants
|
—
|
|
Baseline and Post-Baseline Columbia Suicide Severity Rating Scale (C-SSRS) Responses During Double-Blind Treatment Period
BL: Suicidal Behavior
|
1 Participants
|
1 Participants
|
—
|
|
Baseline and Post-Baseline Columbia Suicide Severity Rating Scale (C-SSRS) Responses During Double-Blind Treatment Period
BL: Suicidal Ideation and/or Behavior
|
2 Participants
|
2 Participants
|
—
|
|
Baseline and Post-Baseline Columbia Suicide Severity Rating Scale (C-SSRS) Responses During Double-Blind Treatment Period
PB: Suicidal Ideation and/or Behavior
|
0 Participants
|
1 Participants
|
—
|
|
Baseline and Post-Baseline Columbia Suicide Severity Rating Scale (C-SSRS) Responses During Double-Blind Treatment Period
BL: Self-Injurious Behavior, No Suicidal Intent
|
1 Participants
|
1 Participants
|
—
|
|
Baseline and Post-Baseline Columbia Suicide Severity Rating Scale (C-SSRS) Responses During Double-Blind Treatment Period
PB: Self-Injurious Behavior, No Suicidal Intent
|
0 Participants
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized participants who received at least 1 dose of study drug. Participants who had an assessment.
Walking capacity and endurance, as determined by the distance in meters walked in 12 minutes during the 12MWT.
Outcome measures
| Measure |
Double-Blind UX007
n=36 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=38 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in 12 Minute Walk Test (12MWT) Distance at Treatment Week 10
|
-15.9 meters
Standard Error 25.63
|
-33.0 meters
Standard Error 24.91
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized adult participants who received at least 1 dose of study drug. Participants who had an assessment.
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013; NIH 2015). It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Physical Function Mobility Domain, increases in score indicate greater mobility.
Outcome measures
| Measure |
Double-Blind UX007
n=23 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=25 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) Health Assessment Questionnaire (Adult Form) Physical Function Score at Treatment Week 10
|
-0.9 T-score
Standard Error 0.67
|
-0.9 T-score
Standard Error 0.65
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized adult participants who received at least 1 dose of study drug. Participants who had an assessment.
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013; NIH 2015). It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Fatigue Domain, decreases in score indicate less fatigue.
Outcome measures
| Measure |
Double-Blind UX007
n=23 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=25 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Fatigue Score at Treatment Week 10
|
2.9 T-score
Standard Error 1.40
|
0.9 T-score
Standard Error 1.36
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized adult participants who received at least 1 dose of study drug. Participants who had an assessment.
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013; NIH 2015). It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Sleep Disturbance Domain, decreases in score indicate less sleep disturbance.
Outcome measures
| Measure |
Double-Blind UX007
n=23 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=25 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Sleep Disturbance Score at Treatment Week 10
|
0.4 T-score
Standard Error 1.24
|
0.7 T-score
Standard Error 1.20
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized adult participants who received at least 1 dose of study drug. Participants who had an assessment.
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013; NIH 2015). It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Pain Interference Domain, decreases in scores indicate less pain interference.
Outcome measures
| Measure |
Double-Blind UX007
n=23 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=25 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Pain Interference Score at Treatment Week 10
|
4.5 T-score
Standard Error 1.64
|
3.7 T-score
Standard Error 1.58
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized adult participants who received at least 1 dose of study drug. Participants who had an assessment.
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013; NIH 2015). It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. The Cognitive Function Domain measures cognitive function impairment. Decreases in score indicate less cognitive function impairment.
Outcome measures
| Measure |
Double-Blind UX007
n=23 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=25 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Cognitive Function Score at Treatment Week 10
|
2.0 T-score
Standard Error 1.29
|
1.2 T-score
Standard Error 1.25
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized adult participants who received at least 1 dose of study drug. Participants who had an assessment.
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013; NIH 2015). It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Social Roles and Activities Domain, decreases in score indicate worse /less or decrease of performance in social roles and activities.
Outcome measures
| Measure |
Double-Blind UX007
n=23 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=25 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Social Roles and Activities Score at Treatment Week 10
|
-4.1 T-score
Standard Error 1.35
|
-2.3 T-score
Standard Error 1.32
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized adult participants who received at least 1 dose of study drug. Participants who had an assessment.
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013; NIH 2015). It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Anxiety Domain, decreases in scores indicate less anxiety.
Outcome measures
| Measure |
Double-Blind UX007
n=23 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=25 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Anxiety Score at Treatment Week 10
|
3.1 T-score
Standard Error 1.49
|
0.5 T-score
Standard Error 1.45
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized pediatric participants who received at least 1 dose of study drug. Participants who had an assessment.
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013; NIH 2015) via parent/proxy. It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Mobility Domain, decreases in score indicate less mobility.
Outcome measures
| Measure |
Double-Blind UX007
n=16 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=13 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in PROMIS Health Assessment Questionnaire (Pediatric/Parent-Proxy Form) Mobility Score at Treatment Week 10
|
-1.0 T-score
Standard Error 1.62
|
0.0 T-score
Standard Error 1.73
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized pediatric participants who received at least 1 dose of study drug. Participants who had an assessment.
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013; NIH 2015) via parent/proxy. It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Upper Extremity Domain, decreases in score indicate less upper extremity movement.
Outcome measures
| Measure |
Double-Blind UX007
n=16 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=13 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in PROMIS Health Assessment Questionnaire (Pediatric/Parent-Proxy Form) Upper Extremity Score at Treatment Week 10
|
-0.4 T-score
Standard Error 1.91
|
1.3 T-score
Standard Error 2.11
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized pediatric participants who received at least 1 dose of study drug. Participants who had an assessment.
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013; NIH 2015) via parent/proxy. It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Fatigue Domain, decreases in score indicate less fatigue.
Outcome measures
| Measure |
Double-Blind UX007
n=16 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=13 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in PROMIS Health Assessment Questionnaire (Pediatric/Parent-Proxy Form) Fatigue Score at Treatment Week 10
|
-1.8 T-score
Standard Error 2.40
|
-0.6 T-score
Standard Error 2.64
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized pediatric participants who received at least 1 dose of study drug. Participants who had an assessment.
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013; NIH 2015) via parent/proxy. It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Pain Interference Domain, decreases in score indicate less pain interference.
Outcome measures
| Measure |
Double-Blind UX007
n=16 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=13 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in PROMIS Health Assessment Questionnaire (Pediatric/Parent-Proxy Form) Pain Interference Score at Treatment Week 10
|
3.2 T-score
Standard Error 1.84
|
1.6 T-score
Standard Error 1.96
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized pediatric participants who received at least 1 dose of study drug. Participants who had an assessment.
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013; NIH 2015) via parent/proxy. It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Peer Relationships Domain, decreases in score indicate worse functioning in peer relationships.
Outcome measures
| Measure |
Double-Blind UX007
n=16 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=13 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in PROMIS Health Assessment Questionnaire (Pediatric/Parent-Proxy Form) Peer Relationships Score at Treatment Week 10
|
2.0 T-score
Standard Error 1.57
|
-0.0 T-score
Standard Error 1.64
|
—
|
SECONDARY outcome
Timeframe: Week 10Population: Full Analysis Set: all randomized participants who received at least 1 dose of study drug. Participants who had an assessment.
Participant/caregiver global impression of change in clinical status using the CGI-I. The CGI-I is a 7-point scale that assesses how much the participant's condition has improved or worsened relative to a baseline state at the beginning of the intervention and rated as: 1=very much better; 2=much better; 3=a little better; 4=no change; 5=a little worse; 6=much worse; 7=very much worse.
Outcome measures
| Measure |
Double-Blind UX007
n=38 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=40 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Clinical Global Impression - Improvement (CGI-I) at Treatment Week 10
|
3.5 score on a scale
Standard Error 0.20
|
3.6 score on a scale
Standard Error 0.20
|
—
|
SECONDARY outcome
Timeframe: Maintenance Phase (up to 22 weeks)Population: Full Analysis Set: all randomized participants (or parent/proxy) who received at least 1 dose of study drug.
Duration of disabling paroxysmal movement disorder events observed during the Maintenance Period of treatment, as recorded by the subject/caregiver in an event-based daily electronic Glut1 DS symptom diary.
Outcome measures
| Measure |
Double-Blind UX007
n=43 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=42 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Duration of Movement Disorder Events During Maintenance Phase
|
0.9 hours
Standard Deviation 1.98
|
0.7 hours
Standard Deviation 1.53
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized participants who received at least 1 dose of study drug. Participants who had an assessment.
Cognitive function as measured by the CANTAB. CANTAB measures neuropsychological function using a standardized, computerized battery of tests designed to assess visual memory, working memory, new learning and reaction time. SSP Span Length assesses the cognitive domain of sequential memory, with scores on a discrete, ordinal scale from 2 to 9; higher scores indicate better function.
Outcome measures
| Measure |
Double-Blind UX007
n=13 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=14 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in Cambridge Neuropsychological Test Automated Battery (CANTAB), Spatial Span (SSP) Span Length Scores at Treatment Week 10
|
0.1 units on a scale
Standard Error 0.31
|
0.6 units on a scale
Standard Error 0.29
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized participants who received at least 1 dose of study drug. Participants who had an assessment.
CANTAB measures neuropsychological function using a standardized, computerized battery of tests designed to assess visual memory, working memory, new learning and reaction time. SWMBE assesses the cognitive domain of working memory, with scores on a discrete, ordinal scale from 0 to 360; lower scores indicate better function.
Outcome measures
| Measure |
Double-Blind UX007
n=13 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=14 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in CANTAB, Spatial Working Memory Between Errors (SWMBE) Scores at Treatment Week 10
|
-0.2 units on a scale
Standard Error 2.94
|
0.2 units on a scale
Standard Error 2.83
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized participants who received at least 1 dose of study drug. Participants who had an assessment.
CANTAB measures neuropsychological function using a standardized, computerized battery of tests designed to assess visual memory, working memory, new learning and reaction time. SWMS assesses the cognitive domain of executive function/strategy, with scores on a discrete, ordinal scale from 4 to 28; lower scores indicate better function.
Outcome measures
| Measure |
Double-Blind UX007
n=13 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=14 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Period Baseline in CANTAB, Spatial Working Memory Strategy (SWMS) Scores at Treatment Week 10
|
0.6 units on a scale
Standard Error 0.77
|
-0.4 units on a scale
Standard Error 0.74
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized participants (or parent/proxy) who received at least 1 dose of study drug. Participants who had an assessment.
CANTAB measures neuropsychological function using a standardized, computerized battery of tests designed to assess visual memory, working memory, new learning and reaction time. PALTEA assesses the cognitive domain of episodic memory/new learning, with scores on a discrete, ordinal scale from 0 to 137; lower scores indicate better function.
Outcome measures
| Measure |
Double-Blind UX007
n=13 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=15 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Baseline in CANTAB, Paired Associates Learning Total Errors (PALTEA) at Treatment Week 10
|
-2.0 units on a scale
Standard Error 4.74
|
-8.5 units on a scale
Standard Error 4.41
|
—
|
SECONDARY outcome
Timeframe: Period Baseline (defined as the last non-missing assessment prior to or on the date of the first dose of study drug for each double-blind Treatment Period), Week 10Population: Full Analysis Set: all randomized participants (or parent/proxy) who received at least 1 dose of study drug. Participants who had an assessment.
CANTAB measures neuropsychological function using a standardized, computerized battery of tests designed to assess visual memory, working memory, new learning and reaction time. PALFTMS assesses the cognitive domain of episodic memory, with scores on a discrete, ordinal scale from 0 to 27; higher scores indicate better function.
Outcome measures
| Measure |
Double-Blind UX007
n=13 Participants
UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
Double-Blind Placebo
n=15 Participants
Placebo for 10 weeks.
|
Open-Label UX007
Open-Label Extension Phase: Participants continued UX007 treatment for up to 3 years.
|
|---|---|---|---|
|
Change From Baseline in CANTAB, Paired Associates Learning First Trial Memory Score (PALFTMS) at Treatment Week 10
|
-0.1 units on a scale
Standard Error 1.12
|
0.9 units on a scale
Standard Error 1.04
|
—
|
Adverse Events
DB UX007
DB Placebo
OL UX007
Serious adverse events
| Measure |
DB UX007
n=43 participants at risk
Double-Blind Maintenance Phase: Participants received UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
DB Placebo
n=42 participants at risk
Double-Blind Maintenance Phase: Participants received placebo for 10 weeks.
|
OL UX007
n=33 participants at risk
Open-Label Extension Phase: Participants continued UX007 treatment (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for up to 3 years.
|
|---|---|---|---|
|
Injury, poisoning and procedural complications
Head Injury
|
2.3%
1/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Musculoskeletal and connective tissue disorders
Limb Asymmetry
|
0.00%
0/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
3.0%
1/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Nervous system disorders
Movement Disorder
|
2.3%
1/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
3.0%
1/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Nervous system disorders
Psychomotor Hyperactivity
|
0.00%
0/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
2.4%
1/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
Other adverse events
| Measure |
DB UX007
n=43 participants at risk
Double-Blind Maintenance Phase: Participants received UX007 (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for 10 weeks.
|
DB Placebo
n=42 participants at risk
Double-Blind Maintenance Phase: Participants received placebo for 10 weeks.
|
OL UX007
n=33 participants at risk
Open-Label Extension Phase: Participants continued UX007 treatment (dosed according to an age- and weight-based strategy, up to a maximum daily administration of 130 g) for up to 3 years.
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal Discomfort
|
9.3%
4/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Gastrointestinal disorders
Abdominal Pain
|
14.0%
6/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
2.4%
1/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
15.2%
5/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
32.6%
14/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
14.3%
6/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
21.2%
7/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Gastrointestinal disorders
Anal Incontinence
|
7.0%
3/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Gastrointestinal disorders
Constipation
|
2.3%
1/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
2.4%
1/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
9.1%
3/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Gastrointestinal disorders
Diarrhoea
|
51.2%
22/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
11.9%
5/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
27.3%
9/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Gastrointestinal disorders
Nausea
|
16.3%
7/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
11.9%
5/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
12.1%
4/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Gastrointestinal disorders
Vomiting
|
30.2%
13/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
19.0%
8/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
24.2%
8/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
General disorders
Fatigue
|
7.0%
3/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
11.9%
5/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
9.1%
3/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
General disorders
Gait Disturbance
|
0.00%
0/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
2.4%
1/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
6.1%
2/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
General disorders
Pyrexia
|
9.3%
4/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
2.4%
1/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
6.1%
2/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Infections and infestations
Gastroenteritis
|
7.0%
3/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
3.0%
1/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Infections and infestations
Gastroenteritis Viral
|
0.00%
0/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
2.4%
1/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
6.1%
2/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Infections and infestations
Influenza
|
7.0%
3/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
6.1%
2/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Infections and infestations
Viral Upper Respiratory Tract Infection
|
11.6%
5/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
4.8%
2/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
6.1%
2/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Investigations
Blood Ketone Body Increased
|
7.0%
3/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
4.8%
2/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Investigations
Weight Increased
|
7.0%
3/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
2.4%
1/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
6.1%
2/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Metabolism and nutrition disorders
Decreased Appetite
|
11.6%
5/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
2.4%
1/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
3.0%
1/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Nervous system disorders
Dizziness
|
2.3%
1/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
4.8%
2/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
6.1%
2/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Nervous system disorders
Dyskinesia
|
7.0%
3/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
4.8%
2/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
9.1%
3/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Nervous system disorders
Headache
|
16.3%
7/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
14.3%
6/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
18.2%
6/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Psychiatric disorders
Aggression
|
9.3%
4/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
4.7%
2/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
0.00%
0/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
9.1%
3/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal Pain
|
9.3%
4/43 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
7.1%
3/42 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
3.0%
1/33 • From first dose of study drug through 30-35 days after final dose. Mean (SD) treatment duration was 65.7 (12.06) and 68.3 (7.04) days for double-blind UX007 and placebo, respectively, and 305.0 (122.71) days for open-label UX007.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER