The Efficacy and Safety of Venglustat Compared With Cerezyme in Pediatric Participants 2 to 11 Years of Age With Gaucher Disease Type 3 (GD3)
NCT07927140, Phase 3, Not yet recruiting, Sanofi
Trial 10
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
| Field | Value | Source |
|---|---|---|
| Title | The Efficacy and Safety of Venglustat Compared With Cerezyme in Pediatric Participants 2 to 11 Years of Age With Gaucher Disease Type 3 (GD3) | ClinicalTrials.gov |
| NCT number | NCT07927140 | ClinicalTrials.gov |
| Status | Not yet recruiting | ClinicalTrials.gov |
| Phases | Phase 3 | ClinicalTrials.gov |
| Enrollment | 18 participants, estimated | ClinicalTrials.gov |
| Lead sponsor | Sanofi | ClinicalTrials.gov |
| Conditions | Gaucher's Disease Type III | ClinicalTrials.gov |
| Interventions | Venglustat, Imiglucerase | ClinicalTrials.gov |
| First posted | 2026-11-10 | ClinicalTrials.gov |
| Last updated | 2026-11-10 | ClinicalTrials.gov |
Registered outcomes 9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description | Source |
|---|---|---|---|---|
| Primary | Percent change from baseline in magnetic resonance imaging(MRI) spleen volume (in multiples of normal [MN]) at Week 52 | at Week 52 | ClinicalTrials.gov | |
| Secondary | Percent change from baseline in MRI liver volume (in MN) at Week 52 | at Week 52 | ClinicalTrials.gov | |
| Secondary | Change from baseline in hemoglobin level at Week 52 | at Week 52 | ClinicalTrials.gov | |
| Secondary | Percent change from baseline in platelet count at Week 52 | at Week 52 | ClinicalTrials.gov | |
| Secondary | Changes from baseline in Vineland Adaptive Behavior Scales III Adaptive Behavior Composite (ABC) score at Week 52 | at Week 52 | ClinicalTrials.gov | |
| Secondary | Change from baseline in Vineland Adaptive Behavior Scale (VABS) III motor skill score at Week 52 | at Week 52 | ClinicalTrials.gov | |
| Secondary | Percent change from baseline in plasma glucosylceramide (GL-1), glucosylsphingosine (lyso-GL-1), and other potential biomarker levels to Week 52 | at Week 52 | ClinicalTrials.gov | |
| Secondary | Number of participants with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and adverse events of special interest (AESIs) | from baseline to week 104 | ClinicalTrials.gov | |
| Secondary | Plasma venglustat Ctrough at prespecified visits | at Week 12, Week 26, Week 40 | ClinicalTrials.gov |
Outcomes 0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as | Source |
|---|
Results by group 0
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units | Source |
|---|
Participant flow 0
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted | Source |
|---|
Adverse events 0
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 | Source |
|---|
Adverse event totals 0
The totals the trial posted for each group.
| Total | Group | Affected | At risk | Source |
|---|
Papers 0
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as | Source |
|---|
Grants 0
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.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years | Source |
|---|
Trial comparisons 0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|
Tests 0
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 | Source |
|---|
Source 5
| Field | Value | Source |
|---|---|---|
| Release | trials-2026-11-12-0.4.0 | ClinicalTrials.gov |
| Release manifest SHA-256 | 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4 | ClinicalTrials.gov |
| Registry capture | 2026-11-12 | ClinicalTrials.gov |
| Results copy, AACT | 2026-03-23 | ClinicalTrials.gov |
| NIH clinical studies file | 2026-11-13 | ClinicalTrials.gov |
Release trials-2026-11-12-0.4.0
ClinicalTrials.gov's posted results, through CTTI's AACT: ClinicalTrials.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.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
ClinicalTrials.gov: none stated ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
NIH RePORTER's clinical studies and project files: Public domain (NIH RePORTER)