Trial · NCT07082543
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
NCT07082543, Phase 3, Active not recruiting, Azafaros B.V.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis. Trials, record NCT07082543. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07082543
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
- NCT number
- NCT07082543
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 75 participants, estimated
- Lead sponsor
- Azafaros B.V.
- Conditions
- Gangliosidoses, GM2, Gangliosidosis, GM1
- Interventions
- AZ-3102, Placebo
- First posted
- 2025-07-24
- Last updated
- 2026-09-22
Source ClinicalTrials.gov
Registered outcomes22
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score | Baseline to month 18 | Total SARA comprises eight categories with a cumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia) |
| Primary | Change from baseline in functional SARA score | Baseline to month 18 | Functional SARA uses an abbreviated scale that scores 0 to 16, with higher scores indicating more severe impairment |
| Secondary | Change from baseline in SARA score for gait/posture | Baseline to months 6, 12, and 18 | Assessed on a 9-point scale with higher scores indicating inability to complete the task |
| Secondary | Change from baseline in SARA score for speech | Baseline to months 6, 12, and 18 | Assessed on a 7-point scale with higher scores indicating unintelligible speech/anarthria |
| Secondary | Change from baseline in SARA score for kinetics | Baseline to months 6, 12, and 18 | Assessed on a 5-point scale with higher scores indicating more severe impairment |
| Secondary | Change from baseline in Vineland Adaptive Behavior Scale (VABS) | Baseline to months 6, 12, and 18 | Assesses four domains of function: communication, daily living skills, socialization, and motor skills |
| Secondary | Change from baseline in Penetration-Aspiration Scale (PAS) | Baseline to months 6, 12, and 18 | Assessed on an 8-point ordinal scale, with 1 representing the lowest and 8 the highest/most severe score |
| Secondary | Change from baseline in 9-Hole Peg Test (9-HPT-D) | Baseline to months 6, 12, and 18 | Assessed by the number of pegs per second placed in a 50-second, 9-HPT using the dominant hand |
| Secondary | Change from baseline in Goal Attainment Scale (GAS) | Baseline to months 6, 12, and 18 | Assesses individual goals achieved during the study |
| Secondary | Change from baseline in Clinician Global Impression of Change (CGI-C) | Baseline to months 6, 12, and 18 | Assessed on a 7-point scale with higher scores indicating lower improvement |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
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.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
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/NCT07082543
GET https://conductscience.org/api/trials/v1/NCT07082543/registered?page=2