A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

Study acronym: HINALEA 2
StatusActive, Not Recruiting
PhasePhase 4
Started

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Record History

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  1. Enrollment closed, study ongoing
    • Final enrollment: 19 participants (planned 28)
    • Study sites: 19 to 17
  2. Eligibility criteria expanded, about 15 words
    • Site details revised at 8 of 19 sites
  3. Site details revised at 1 of 19 sitesv46
  4. Re-verified, no change to tracked fieldsv45
  5. Site details revised at 2 of 19 sitesv44
Show 42 earlier versionsHide 42 earlier versions
  1. Study Status, Contacts/Locations, Referencesv43
  2. 8 versions Re-verified since Oct 23, 2025, no change to tracked fieldsv35-v42
  3. Site details revised at 2 of 19 sitesv34
  4. Re-verified, no change to tracked fieldsv33
  5. Primary completion date: Jan 31, 2027 to Mar 31, 2028v32 Show 4 more changesShow less
    • Study sites: 18 to 19
    • Study completion date: Mar 31, 2028 to Mar 31, 2029
    • Interventions: risdiplam (Drug) to Risdiplam (Drug)
    • Eligibility criteria expanded, about 52 words
  6. 3 versions Re-verified since Apr 11, 2025, no change to tracked fieldsv29-v31
  7. Study sites: 17 to 18v28
  8. Study sites: 16 to 17v27
  9. Study sites: 10 to 16v26
  10. 3 versions Re-verified since Oct 25, 2024, no change to tracked fieldsv23-v25
  11. Study sites: 8 to 10v22
  12. Start date changed from estimate of Aug 16, 2024 to Aug 14, 2024v21
  13. Start date: Jun 30, 2024 to Aug 16, 2024
    • Study sites: 3 to 8
    v20
  14. Study sites: 2 to 3v19
  15. Re-verified, no change to tracked fieldsv18
  16. Start date: May 31, 2024 to Jun 30, 2024
    • Study sites: 1 to 2
    v17
  17. Re-verified, no change to tracked fieldsv16
  18. Start date: Apr 20, 2024 to May 31, 2024v15
  19. Trial status: Not Yet Recruiting to Recruiting
    • Start date: Mar 12, 2024 to Apr 20, 2024
    • Study sites: 0 to 1
    v14
  20. Secondary endpoints expanded, about 23 wordsv13
  21. 2 versions Re-verified since Jan 18, 2024, no change to tracked fieldsv11-v12
  22. Start date: Dec 29, 2023 to Mar 12, 2024v10
  23. 3 versions Re-verified since Nov 2, 2023, no change to tracked fieldsv7-v9
  24. Start date: Oct 31, 2023 to Dec 29, 2023v6
  25. Eligibility criteria revised
    • Secondary endpoints shortened, about 171 words
    v5
  26. Re-verified, no change to tracked fieldsv4
  27. Start date: Jul 31, 2023 to Oct 31, 2023v3
  28. Re-verified, no change to tracked fieldsv2
  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 in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.
Trial Details
NCT Number NCT05861999
Lead Sponsor Hoffmann-La Roche
Conditions
(from sponsor)		 Muscular Atrophy, Spinal
Related terms Muscular Atrophy, Spinal
Enrollment 19 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov