Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Study acronym: WeSMA
StatusCompleted
PhasePhase 4
Started

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Record History

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  1. Trial completed Show 4 more changesShow less
    • Primary and study completion dates changed from estimate of Oct 2, 2026 to Oct 9, 2026
    • Secondary endpoints: details revised at 1 of 1 entry
    • Site details revised at 11 of 38 sites
    • Eligibility criteria expanded, about 4 words
  2. Primary and study completion moved earlier from Dec 31, 2026 to Oct 2, 2026v47
  3. Re-verified, no change to tracked fieldsv46
  4. Enrollment target: 404 to 403v45
  5. Re-verified, no change to tracked fieldsv44
Show 42 earlier versionsHide 42 earlier versions
  1. Primary endpoints: 4 to 1v43 Show 6 more changesShow less
    • Enrollment target: 402 to 404
    • Study sites: 39 to 38
    • Interventions: Risdiplam (Drug), SMA-DAT Application and ADAM Sensor (Device) to Risdiplam (Drug)
    • Eligibility criteria shortened, about 210 words
    • Secondary endpoints: 6 to 1
    • Study arms: 2 to 1
  2. 3 versions Re-verified since May 30, 2025, no change to tracked fieldsv40-v42
  3. Trial status: Recruiting to Active, Not Recruiting
    • Enrollment target: estimated 500 to confirmed 402
    • Site details revised at 39 of 39 sites
    v39
  4. Primary endpoints: 1 to 4v38 Show 5 more changesShow less
    • Interventions: Risdiplam (Drug) to Risdiplam (Drug), SMA-DAT Application and ADAM Sensor (Device)
    • Eligibility criteria expanded, about 226 words
    • Secondary endpoints: 1 to 6
    • Study sites revised (39)
    • Study arms: 1 to 2
  5. Start date: Jun 14, 2022 to Mar 29, 2022
    • Secondary endpoints: details revised at 1 of 1 entry
    • Eligibility criteria shortened, about 2 words
    v37
  6. Primary and study completion dates: Jun 1, 2029 to Dec 31, 2026v36
  7. Study sites: 38 to 39v35
  8. 3 versions Re-verified since Jun 21, 2024, no change to tracked fieldsv32-v34
  9. Study sites: 37 to 38v31
  10. Study sites: 36 to 37v30
  11. Study sites: 32 to 36v29
  12. Study sites: 31 to 32v28
  13. Re-verified, no change to tracked fieldsv27
  14. Study sites: 30 to 31v26
  15. Re-verified, no change to tracked fieldsv25
  16. Study sites: 29 to 30v24
  17. Study sites: 28 to 29v23
  18. Study sites: 27 to 28v22
  19. Re-verified, no change to tracked fieldsv21
  20. Start date: Mar 29, 2022 to Jun 14, 2022
    • Primary and study completion dates: Feb 1, 2029 to Jun 1, 2029
    v20
  21. Study sites: 26 to 27v19
  22. Study sites: 25 to 26v18
  23. Study sites: 24 to 25v17
  24. Re-verified, no change to tracked fieldsv16
  25. Study sites: 21 to 24v15
  26. Study sites: 19 to 21v14
  27. Study sites: 18 to 19v13
  28. Study sites: 15 to 18v12
  29. Study sites: 12 to 15v11
  30. Study sites: 11 to 12v10
  31. Study sites: 10 to 11v9
  32. Study sites: 7 to 10
    • Eligibility criteria shortened, about 15 words
    • Secondary endpoints: 27 to 1
    v8
  33. Study sites: 4 to 7v7
  34. Re-verified, no change to tracked fieldsv6
  35. Study sites: 1 to 4v5
  36. Re-verified, no change to tracked fieldsv4
  37. Start date changed from estimate of Mar 31, 2022 to Mar 29, 2022v3
  38. Trial status: Not Yet Recruiting to Recruiting
    • Start date: Jan 31, 2022 to Mar 31, 2022
    • Study sites: 0 to 1
    v2
  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.
A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Information (USPI) in adult and pediatric participants with SMA. In this study, participants will be followed for approximately 24 months from enrollment or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study if they agree to continue participating in the follow-up assessments. An optional sub-study was planned to assess the feasibility, acceptability, and adherence of remote assessment of motor and bulbar functions in participants with SMA using wearable and smartphone-based biosensors. This substudy was withdrawn upon implementation of protocol version 4.
Trial Details
NCT Number NCT05232929
Lead Sponsor Genentech, Inc.
Conditions
(from sponsor)		 Spinal Muscular Atrophy
Related terms Muscular Atrophy, Spinal
Enrollment 403 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov