Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT04042025 · ClinicalTrials.gov registry record · Phase 3
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
A Phase 3 study of SMA and Spinal Muscular Atrophy Type II, sponsored by Novartis Gene Therapies.
- Active
- Registry status
- Phase 3
- Development phase
- 85
- Enrollment target
- 20
- Study locations
NCT04042025: Active Phase 3 study of SMA and Spinal Muscular Atrophy Type II, sponsored by Novartis Gene Therapies.
NCT04042025 is a Phase 3 study of SMA and Spinal Muscular Atrophy Type II that is active but no longer recruiting, run by Novartis Gene Therapies. The registered enrollment target is 85 participants, below the 135-participant average among 6 other SMA trials with a reported enrollment target (37% lower). The trial reports 20 study locations across 17 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04042025, a Phase 3 study of SMA and Spinal Muscular Atrophy Type II, is active but no longer recruiting, sponsored by Novartis Gene Therapies.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 85 participants
- Enrollment target
- 20
- Study locations
Study Summary
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
Primary Outcome
Assessed via the developmental milestone checklist, formed of 10 yes/no questions. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone.
Conditions Studied
Interventions
- BIOLOGICAL Onasemnogene Abeparvovec-xioi
Study Locations (20)
Massachusetts
- Massachusetts General Hospital - Boston
- Boston Children's Hospital - Boston
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
- Clinic for Special Children - Strasburg
Virginia
- Children's Hospital of The King's Daughters - Norfolk
- Virginia Commonwealth University - Richmond
California
- Stanford University Medical Center - Palo Alto
Colorado
- Children's Hospital Colorado - Aurora
Illinois
- Ann Robert H. Lurie Children's Hospital of Chicago - Chicago
Maryland
- John Hopkins Hospital - David M. Rubenstein Child Health Building - Baltimore
Michigan
- Spectrum Health Hospitals Helen DeVos Children's Hospital - Grand Rapids
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 85 participants |
| Start Date | 2020-02-10 |
| Est. Completion | 2035-12-31 |
| Phase | Phase 3 |
What the registry record for NCT04042025 still lists
NCT04042025 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 85 participants, a relatively small participant target, below the 135-participant average among 6 other SMA trials with a reported enrollment target (37% lower).
The record links to 4 conditions, with SMA appearing as the primary indexed condition, and to 1 intervention - of which Onasemnogene Abeparvovec-xioi is the first listed.
NCT04042025 names 20 study sites across 17 states, led by Massachusetts, Pennsylvania, Virginia.
Frequently Asked Questions
What is clinical trial NCT04042025 about?
NCT04042025 is a clinical study titled "Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi". This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitor...
What is the current status of trial NCT04042025?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 85 participants. The study started on 2020-02-10. Estimated completion is 2035-12-31.
What conditions does trial NCT04042025 study?
This clinical trial studies the following conditions: SMA, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type III.
What interventions are being tested in trial NCT04042025?
The interventions under investigation include: Onasemnogene Abeparvovec-xioi (BIOLOGICAL).
Who is sponsoring clinical trial NCT04042025?
This trial is sponsored by Novartis Gene Therapies, which has 6 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04042025 being conducted?
This trial has 20 study locations across California, Colorado, Illinois, Maryland, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for SMA
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
ACTIVE NOT RECRUITING · Phase 3
-
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
ACTIVE NOT RECRUITING · Phase 3
-
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
COMPLETED · Phase 3
-
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
RECRUITING · Phase 2
-
Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb
ACTIVE NOT RECRUITING · NA
-
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
COMPLETED · Phase 2
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT00842283 · 85 participants
Role of Angiogenesis in Dermatologic Diseases: A Potential Therapeutic Target
- NCT02133196 · 85 participants · Phase 2
T Cell Receptor Immunotherapy for Patients With Metastatic Non-Small Cell Lung Cancer
- NCT03823144 · 85 participants · NA
Intravital Microscopy in Human Solid Tumors
- NCT03875638 · 85 participants · Phase 2
Treating Hyperexcitability in AD With Levetiracetam
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI