Trial · NCT07399704
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
NCT07399704, Phase 2, 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 Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease. Trials, record NCT07399704. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07399704
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 Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
- NCT number
- NCT07399704
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 21 participants, estimated
- Lead sponsor
- Azafaros B.V.
- Conditions
- GM2 Gangliosidosis, Niemann-Pick Type C Disease
- Interventions
- AZ-3102
- First posted
- 2026-02-10
- Last updated
- 2026-10-21
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change from baseline in treatment-emergent adverse events (TEAEs) | Through study completion, an average of 4 years | Incidence and severity of all Adverse Events related to study drug treatment, study discontinuation or death |
| Primary | Change from baseline in electrocardiogram (ECG) | Through study completion, an average of 4 years | ECG read out Normal, Abnormal, Not Clinically Significant, Abnormal, Clinically Significant and Not Done. |
| Primary | Change from baseline in seizures | Through study completion, an average of 4 years | Seizure duration (minutes) as per the seizure diary. |
| Primary | Change from baseline in seizures | Through study completion, an average of 4 years | Seizure frequency (number) as per seizure diary. |
| Primary | Maximum observed plasma concentration (Cmax) | Baseline , Month 1 (Cohort 2 only) and Month 6 | |
| Primary | Time to Cmax (Tmax) | Baseline, Month 1 (Cohort 2 only) and Month 6 | |
| Primary | Concentration at trough (Ctrough) | Baseline, Month 1 (Cohort 2 only) and Month 6 | |
| Primary | Area under the plasma concentration-time curve from the time of dosing (zero) to 24 hours post-dose | Baseline, Month 1 (Cohort 2 only) and Month 6 | |
| Secondary | Change from Baseline in the concentrations of Glucosylceramide (GlcCer) C16:0; C18:0 | Baseline, Month 1 (Cohort 2 only) and Month 6 | |
| Secondary | Change from Baseline in the concentrations of Neurofilament light chain (NfL) | Through study completion, an average of 4 years |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 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/NCT07399704
GET https://conductscience.org/api/trials/v1/NCT07399704/registered