An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
Public ClinicalTrials.gov record NCT03921528. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
Study identification
- NCT ID
- NCT03921528
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 58 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 2 Years to 21 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 22, 2019 (Actual)
- Primary completion
- Jan 19, 2021 (Actual)
- Completion
- Feb 28, 2024 (Actual)
- Last update posted
- Dec 2, 2024
2019 – 2024
United States locations
- U.S. sites
- 11
- U.S. states
- 11
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Phoenix Children's Hospital | Phoenix | Arizona | 85016 | — |
| Stanford University | Palo Alto | California | 94304 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| The Johns Hopkins University | Baltimore | Maryland | 21287 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Helen DeVos Children's Hospital | Grand Rapids | Michigan | 49503 | — |
| Columbia University | New York | New York | 10032 | — |
| Wake Forest School of Medicine | Winston-Salem | North Carolina | 27157 | — |
| Oregon Health & Science University | Portland | Oregon | 97239 | — |
| Childrens Medical Center Dallas | Dallas | Texas | 75235 | — |
| Children's Hospital of The King's Daughters | Norfolk | Virginia | 23507 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 5 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03921528, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Dec 2, 2024 · Synced Oct 21, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03921528 live on ClinicalTrials.gov.