Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

Study acronym: LEAP
StatusActive, Not Recruiting
PhasePhase 2
Started

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Record History

The 50 most recent of 51 record versions are accounted for below. See every version on ClinicalTrials.gov ↗

  1. 2 versions Re-verified since Jun 9, 2026, no change to tracked fieldsv50-v51
  2. Primary and study completion dates: Sep 30, 2025 to Oct 30, 2026v49 Show 4 more changesShow less
    • Primary endpoints revised (2)
    • Eligibility criteria revised
    • Secondary endpoints: 8 to 16
    • Study description revised
  3. Enrollment target: estimated 13 to confirmed 12
    • Study sites revised (7)
    v48
  4. Eligibility criteria expanded, about 85 words
    • Study sites revised (7)
    v47
  5. Primary and study completion dates: Jan 9, 2026 to Sep 30, 2025v46
Show 44 earlier versionsHide 44 earlier versions
  1. Primary and study completion dates: Sep 25, 2025 to Jan 9, 2026v45
  2. Re-verified, no change to tracked fieldsv44
  3. Primary and study completion dates: Jan 11, 2024 to Sep 25, 2025v43 Show 5 more changesShow less
    • Primary endpoints: 3 to 2
    • Study sites: 8 to 7
    • Secondary endpoints revised (8)
    • Study description expanded, about 1 word
    • Study title expanded, about 4 words
  4. Phase: Phase 2/3 to Phase 2v42 Show 11 more changesShow less
    • Enrollment target: 49 to 13
    • Primary and study completion dates: Jan 2023 to Jan 11, 2024
    • Primary endpoints: 5 to 3
    • Trial status: Recruiting to Active, Not Recruiting
    • Study sites: 7 to 8
    • Interventions: imiglucerase (Drug), placebo (Drug), venglustat (GZ402671) (Drug) to imiglucerase (Drug), venglustat (GZ402671) (Drug)
    • Eligibility criteria shortened, about 166 words
    • Secondary endpoints: 23 to 8
    • Study description shortened, about 18 words
    • Study arms: 4 to 1
    • Study title shortened, about 2 words
  5. Primary and study completion dates: Dec 2022 to Jan 2023v41
  6. Primary and study completion dates: Aug 2024 to Dec 2022v40
  7. Site details revised at 1 of 7 sitesv39
  8. Primary and study completion dates: May 2024 to Aug 2024v38 Show 4 more changesShow less
    • Start date: Jan 2017 to Jan 4, 2017
    • Interventions: imiglucerase (Drug), placebo (Drug), venglustat (GZ/SAR402671) (Drug) to imiglucerase (Drug), placebo (Drug), venglustat (GZ402671) (Drug)
    • Secondary endpoints: details revised at 1 of 23 entries
    • Study description expanded, about 7 words
  9. Phase: Phase 2 to Phase 2/3v37 Show 8 more changesShow less
    • Enrollment target: 10 to 49
    • Primary endpoints: 3 to 5
    • Interventions: GZ402671 (Drug) to imiglucerase (Drug), placebo (Drug), venglustat (GZ/SAR402671) (Drug)
    • Eligibility criteria expanded, about 188 words
    • Secondary endpoints: 8 to 23
    • Study arms: 1 to 4
    • Study description expanded, about 1 word
    • Study title expanded, about 2 words
  10. Site details revised at 1 of 7 sitesv36
  11. Primary endpoints: 5 to 3
    • Start date: Jan 4, 2017 to Jan 2017
    • Eligibility criteria revised
    • Study description revised
    v35
  12. Primary and study completion dates: Feb 2024 to May 2024v34
  13. Primary and study completion dates: Oct 2023 to Feb 2024v33
  14. Primary and study completion dates: May 2023 to Oct 2023v32
  15. Primary and study completion dates: Dec 2021 to May 2023v31
  16. Site details revised at 2 of 7 sitesv30
  17. Primary and study completion dates: Nov 2021 to Dec 2021v29
  18. Site details revised at 1 of 7 sitesv28
  19. Re-verified, no change to tracked fieldsv27
  20. Study sites: 6 to 7v26
  21. Primary and study completion dates: Oct 28, 2021 to Nov 2021v25
  22. 3 versions Re-verified since Jul 18, 2018, no change to tracked fieldsv22-v24
  23. Primary and study completion dates: Oct 11, 2021 to Oct 28, 2021v21
  24. 3 versions Re-verified since Jun 26, 2018, no change to tracked fieldsv18-v20
  25. Study sites: 5 to 6v17
  26. Study sites: 4 to 5v16
  27. Primary endpoints: 2 to 5
    • Eligibility criteria expanded, about 171 words
    • Secondary endpoints: 3 to 8
    • Study description expanded, about 12 words
    v15
  28. Primary and study completion dates: Oct 2022 to Oct 11, 2021v14
  29. 2 versions Re-verified since May 10, 2017, no change to tracked fieldsv12-v13
  30. Study sites: 3 to 4v11
  31. Interventions: GZ/SAR402671 (Drug), Imiglucerase (Drug) to GZ402671 (Drug)
    • Eligibility criteria shortened, about 22 words
    v10
  32. Start date: Jan 2017 to Jan 4, 2017v9
  33. Primary and study completion dates: Sep 2022 to Oct 2022
    • Start date: Dec 2016 to Jan 2017
    v8
  34. Study sites: 2 to 3
    • Primary and study completion dates: Jun 2022 to Sep 2022
    • Start date: Nov 2016 to Dec 2016
    v7
  35. Study sites: 1 to 2v6
  36. Trial status: Not Yet Recruiting to Recruiting
    • Start date: Sep 2016 to Nov 2016
    • Study sites: 0 to 1
    v5
  37. Re-verified, no change to tracked fieldsv4
  38. Primary and study completion dates: Nov 2021 to Jun 2022v3
  39. Primary and study completion dates: Oct 2021 to Nov 2021
    • Start date: Aug 2016 to Sep 2016
    v2
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
Sanofi
286 trials monitored
See 12 trials at risk
Part 1: Biomarker evaluation/screening phase Primary Objectives: * Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease Type 3 (GD3) participants that distinguish GD3 from adult Gaucher disease Type 1 (GD1) participants * Screen adult GD3 participants who qualify for treatment with venglustat in Parts 2, Part 3, and Part 4 Parts 2 and 3: Combination treatment phases Primary objectives: * Evaluate short-term (Part 2) and long-term (Part 3) safety and tolerability of venglustat in combination with Cerezyme in adult GD3 participants * Evaluate the change in CSF central nervous system (CNS) biomarkers (glucosylceramide \[GL-1\] and lyso-glucosylceramide \[lyso-GL-1\]) from adult GD3 participants receiving venglustat in combination with Cerezyme (Part 2 only) Part 4: Extended treatment phase with monotherapy Primary objectives: • Evaluate safety and tolerability of venglustat monotherapy in adult GD3 participants who have remained systemically stable on venglustat in combination with Cerezyme Parts 2 and 3: Combination treatment phases Secondary Objectives: * Evaluate the pharmacokinetics (PK) of venglustat in adult GD3 participants * Evaluate the efficacy of venglustat in combination with Cerezyme in systemic disease in adult GD3 participants by assessing spleen volume, liver volume, hemoglobin level and platelet count * Evaluate the efficacy of venglustat in combination with Cerezyme on neurological function in adult GD3 participants by assessing Ataxia using the Scale for the Assessment and Rating of Ataxia (SARA) * Evaluate plasma biomarkers (lyso-GL-1 and GL-1) in adult GD3 participants Part 4: Extended treatment phase with monotherapy Secondary objectives: * Evaluate the efficacy of venglustat in systemic disease in adult GD3 participants by assessing spleen volume, liver volume, hemoglobin level and platelet count * Evaluate the efficacy of venglustat on neurological function in adult GD3 participants by assessing Ataxia using the Scale for the Assessment and Rating of Ataxia (SARA) * Evaluate plasma biomarkers (lyso-GL-1 and GL-1) in adult GD3 participants
Trial Details
NCT Number NCT02843035
Lead Sponsor Genzyme, a Sanofi Company
Conditions
(from sponsor)		 Gaucher Disease Type 1, Gaucher Disease Type 3
Related terms Gaucher Disease
Enrollment 12 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov