Trial · NCT05222906
Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3
NCT05222906, Phase 3, Active not recruiting, Sanofi
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3. Trials, record NCT05222906. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05222906
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3
- NCT number
- NCT05222906
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 43 participants, actual
- Lead sponsor
- Sanofi
- Conditions
- Gaucher's Disease Type III
- Interventions
- Venglustat, imiglucerase
- First posted
- 2022-02-03
- Last updated
- 2026-07-13
- Other registries' IDs
- eudract:2021-005402-10
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Scale for Assessment and Rating of Ataxia (SARA) modified total score | From baseline to Week 52 | |
| Primary | Change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score | From baseline to Week 52 | |
| Secondary | Percent change in spleen volume | From baseline to Week 52 | |
| Secondary | Percent change in liver volume | From baseline to Week 52 | |
| Secondary | Change in hemoglobin level | From baseline to Week 52 | |
| Secondary | Percent change in platelet count | From baseline to Week 52 | |
| Secondary | Percent change in CSF GL-1 and lyso-GL-1 levels | From baseline to Week 52 | |
| Secondary | Percent change in plasma GL-1 and lyso-GL-1 levels | From baseline to Week 52 | |
| Secondary | Number of patients with treatment emergent adverse events (TEAEs)/ serious adverse events (SAEs)/ adverse events of special interest (AESIs) | From baseline to max of 4.5 years | |
| Secondary | Change in score of Beck Depression Inventory II (BDI-II) during the treatment-emergent period (for participants 13 years of age and above at baseline) | From baseline to Week 52 |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 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/NCT05222906
GET https://conductscience.org/api/trials/v1/NCT05222906/registered