Trial · NCT02960217
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)
NCT02960217, Phase 3, Terminated, Ultragenyx Pharmaceutical Inc; results first posted 2020-04-24
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. 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). Trials, record NCT02960217. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02960217
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- 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 number
- NCT02960217
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 44 participants, actual
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
- Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
- Interventions
- UX007, Placebo
- First posted
- 2016-11-09
- Last updated
- 2020-06-16
- Results first posted
- 2020-04-24
Source ClinicalTrials.gov
Registered outcomes23
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Maintenance Phase Movement Disorder Frequency | Maintenance Phase (up to Week 22) | 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. |
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs | 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. | 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. |
| Primary | Baseline and Post-Baseline Columbia Suicide Severity Rating Scale (C-SSRS) Responses During Double-Blind Treatment Period | Baseline, up to Week 22 | 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. |
| Secondary | Change From Period Baseline in 12 Minute Walk Test (12MWT) Distance at Treatment Week 10 | 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 10 | Walking capacity and endurance, as determined by the distance in meters walked in 12 minutes during the 12MWT. |
| Secondary | Change From Period Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) Health Assessment Questionnaire (Adult Form) Physical Function Score at Treatment Week 10 | 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 10 | 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. |
| Secondary | Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Fatigue Score at Treatment Week 10 | 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 10 | 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. |
| Secondary | Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Sleep Disturbance Score at Treatment Week 10 | 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 10 | 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. |
| Secondary | Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Pain Interference Score at Treatment Week 10 | 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 10 | 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. |
| Secondary | Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Cognitive Function Score at Treatment Week 10 | 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 10 | 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. |
| Secondary | Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Social Roles and Activities Score at Treatment Week 10 | 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 10 | 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. |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 registered outcomesOutcomes23
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Maintenance Phase Movement Disorder Frequency | Maintenance Phase (up to Week 22) | Full Analysis Set: all randomized participants who received at least 1 dose of study drug. | movement disorder events per 4 weeks | MEDIAN |
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs | 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. | Safety Analysis Set: all randomized participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Baseline and Post-Baseline Columbia Suicide Severity Rating Scale (C-SSRS) Responses During Double-Blind Treatment Period | Baseline, up to Week 22 | Safety Analysis Set: all randomized participants who received at least 1 dose of study drug. Participants with a baseline (BL) and postbaseline (PB) assessment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Period Baseline in 12 Minute Walk Test (12MWT) Distance at Treatment Week 10 | 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 10 | Full Analysis Set: all randomized participants who received at least 1 dose of study drug. Participants who had an assessment. | meters | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Period Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) Health Assessment Questionnaire (Adult Form) Physical Function Score at Treatment Week 10 | 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 10 | Full Analysis Set: all randomized adult participants who received at least 1 dose of study drug. Participants who had an assessment. | T-score | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Fatigue Score at Treatment Week 10 | 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 10 | Full Analysis Set: all randomized adult participants who received at least 1 dose of study drug. Participants who had an assessment. | T-score | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Sleep Disturbance Score at Treatment Week 10 | 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 10 | Full Analysis Set: all randomized adult participants who received at least 1 dose of study drug. Participants who had an assessment. | T-score | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Pain Interference Score at Treatment Week 10 | 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 10 | Full Analysis Set: all randomized adult participants who received at least 1 dose of study drug. Participants who had an assessment. | T-score | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Cognitive Function Score at Treatment Week 10 | 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 10 | Full Analysis Set: all randomized adult participants who received at least 1 dose of study drug. Participants who had an assessment. | T-score | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Period Baseline in PROMIS Health Assessment Questionnaire (Adult Form) Social Roles and Activities Score at Treatment Week 10 | 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 10 | Full Analysis Set: all randomized adult participants who received at least 1 dose of study drug. Participants who had an assessment. | T-score | LEAST_SQUARES_MEAN |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 outcomesResults by group92
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Maintenance Phase Movement Disorder Frequency | Double-Blind UX007 | 14.26 | Full Range 0.0 to 112.0 | movement disorder events per 4 weeks | |
| Maintenance Phase Movement Disorder Frequency | Double-Blind Placebo | 11.81 | Full Range 0.5 to 112.0 | movement disorder events per 4 weeks | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs | Double-Blind UX007 | TEAEs | 40 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs | Double-Blind Placebo | TEAEs | 34 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs | Open-Label UX007 | TEAEs | 28 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs | Double-Blind UX007 | Serious TEAEs | 2 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs | Double-Blind Placebo | Serious TEAEs | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs | Open-Label UX007 | Serious TEAEs | 2 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs | Double-Blind UX007 | Treatment-Related TEAEs | 33 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs | Double-Blind Placebo | Treatment-Related TEAEs | 19 | Participants |
1 to 10 of 92Source ClinicalTrials.gov
See all 92 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Maintenance Phase: Treatment Period 1 | STARTED | Double-Blind UX007 Followed by Placebo | 22 | |
| Maintenance Phase: Treatment Period 1 | STARTED | Double-Blind Placebo Followed by UX007 | 21 | |
| Maintenance Phase: Treatment Period 1 | COMPLETED | Double-Blind UX007 Followed by Placebo | 21 | |
| Maintenance Phase: Treatment Period 1 | COMPLETED | Double-Blind Placebo Followed by UX007 | 21 | |
| Maintenance Phase: Treatment Period 1 | NOT COMPLETED | Double-Blind UX007 Followed by Placebo | 1 | |
| Maintenance Phase: Treatment Period 1 | NOT COMPLETED | Double-Blind Placebo Followed by UX007 | 0 | |
| Maintenance Phase: Crossover Washout | STARTED | Double-Blind UX007 Followed by Placebo | 21 | |
| Maintenance Phase: Crossover Washout | STARTED | Double-Blind Placebo Followed by UX007 | 21 | |
| Maintenance Phase: Crossover Washout | COMPLETED | Double-Blind UX007 Followed by Placebo | 21 | |
| Maintenance Phase: Crossover Washout | COMPLETED | Double-Blind Placebo Followed by UX007 | 21 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events84
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal Discomfort | Gastrointestinal disorders | DB UX007 | 4 | 43 | |
| other | Abdominal Discomfort | Gastrointestinal disorders | DB Placebo | 0 | 42 | |
| other | Abdominal Discomfort | Gastrointestinal disorders | OL UX007 | 0 | 33 | |
| other | Abdominal Pain | Gastrointestinal disorders | DB UX007 | 6 | 43 | |
| other | Abdominal Pain | Gastrointestinal disorders | DB Placebo | 1 | 42 | |
| other | Abdominal Pain | Gastrointestinal disorders | OL UX007 | 5 | 33 | |
| other | Abdominal Pain Upper | Gastrointestinal disorders | DB UX007 | 14 | 43 | |
| other | Abdominal Pain Upper | Gastrointestinal disorders | DB Placebo | 6 | 42 | |
| other | Abdominal Pain Upper | Gastrointestinal disorders | OL UX007 | 7 | 33 | |
| other | Anal Incontinence | Gastrointestinal disorders | DB UX007 | 3 | 43 |
1 to 10 of 84Source ClinicalTrials.gov
See all 84 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DB UX007 | 0 | 43 |
| Total, all-cause mortality | DB Placebo | 0 | 42 |
| Total, all-cause mortality | OL UX007 | 0 | 33 |
| Total, other adverse events | DB UX007 | 36 | 43 |
| Total, other adverse events | DB Placebo | 26 | 42 |
| Total, other adverse events | OL UX007 | 22 | 33 |
| Total, serious adverse events | DB UX007 | 2 | 43 |
| Total, serious adverse events | DB Placebo | 1 | 42 |
| Total, serious adverse events | OL UX007 | 2 | 33 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests1
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as |
|---|---|
| Paired Associate Learning | Secondary outcome |
Source ConductCognition
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02960217
GET https://conductscience.org/api/trials/v1/NCT02960217/registered?page=2