A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Study acronym: HINALEA 1
StatusRecruiting
PhasePhase 4
Started

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Record History

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  1. Eligibility criteria expanded, about 16 words
    • Site details revised at 6 of 16 sites
  2. Site details revised at 1 of 16 sitesv45
  3. Site details revised at 1 of 16 sitesv44
  4. 3 versions Re-verified since Apr 2, 2026, no change to tracked fieldsv41-v43
  5. Site details revised at 1 of 16 sitesv40
Show 38 earlier versionsHide 38 earlier versions
  1. 7 versions Re-verified since Aug 22, 2025, no change to tracked fieldsv33-v39
  2. Primary completion date: Jan 31, 2027 to Mar 31, 2028
    • Study completion date: Mar 31, 2028 to Mar 31, 2029
    • Interventions: risdiplam (Drug) to Risdiplam (Drug)
    • Eligibility criteria expanded, about 57 words
    v32
  3. 3 versions Re-verified since Apr 17, 2025, no change to tracked fieldsv29-v31
  4. Site details revised at 1 of 16 sitesv28
  5. Study sites: 15 to 16v27
  6. Site details revised at 2 of 15 sitesv26
  7. Study sites: 9 to 15v25
  8. 2 versions Re-verified since Oct 30, 2024, no change to tracked fieldsv23-v24
  9. Study sites: 5 to 9v22
  10. Re-verified, no change to tracked fieldsv21
  11. Study sites: 3 to 5v20
  12. Study sites: 1 to 3v19
  13. Start date changed from estimate of Jun 30, 2024 to May 30, 2024v18
  14. Start date: Apr 29, 2024 to Jun 30, 2024v17
  15. Re-verified, no change to tracked fieldsv16
  16. Start date: Mar 31, 2024 to Apr 29, 2024v15
  17. Start date: Feb 28, 2024 to Mar 31, 2024v14
  18. Trial status: Not Yet Recruiting to Recruiting
    • Study sites: 0 to 1
    • Start date: Feb 7, 2024 to Feb 28, 2024
    v13
  19. Secondary endpoints expanded, about 23 wordsv12
  20. Start date: Dec 31, 2023 to Feb 7, 2024v11
  21. Re-verified, no change to tracked fieldsv10
  22. Start date: Nov 30, 2023 to Dec 31, 2023v9
  23. Re-verified, no change to tracked fieldsv8
  24. Start date: Oct 31, 2023 to Nov 30, 2023v7
  25. Eligibility criteria revised
    • Secondary endpoints shortened, about 171 words
    v6
  26. Re-verified, no change to tracked fieldsv5
  27. Start date: Jul 31, 2023 to Oct 31, 2023v4
  28. 2 versions Re-verified since May 24, 2023, no change to tracked fieldsv2-v3
  1. Study First Submitted
  • 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.
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.
Trial Details
NCT Number NCT05861986
Lead Sponsor Hoffmann-La Roche
Conditions
(from sponsor)		 Muscular Atrophy, Spinal
Related terms Muscular Atrophy, Spinal
Enrollment 28 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov