Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT04851873 · ClinicalTrials.gov registry record · Phase 3
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
A Phase 3 study, sponsored by Novartis Pharmaceuticals.
- Completed
- Registry status
- Phase 3
- Development phase
- 24
- Enrollment target
NCT04851873: Completed Phase 3 study, sponsored by Novartis Pharmaceuticals.
NCT04851873 is a Phase 3 study that has completed, run by Novartis Pharmaceuticals. The registered enrollment target is 24 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04851873, a Phase 3 study, has completed, sponsored by Novartis Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 24 participants
- Enrollment target
Study Summary
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥ 8.5 kg and ≤ 21 kg, over a 12 month period.
Primary Outcome
An AE is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study.
Interventions
- GENETIC OAV101
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2021-09-08 |
| Est. Completion | 2023-06-13 |
| Phase | Phase 3 |
What the finished NCT04851873 record still lists
NCT04851873 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 24 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower).
The record links to 0 conditions, and to 1 intervention - of which OAV101 is the first listed.
NCT04851873 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04851873 about?
NCT04851873 is a clinical study titled "Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)". To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥ 8.5 kg and ≤ 21 kg, over a 12 month period.
What is the current status of trial NCT04851873?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 24 participants. The study started on 2021-09-08. Estimated completion is 2023-06-13.
What interventions are being tested in trial NCT04851873?
The interventions under investigation include: OAV101 (GENETIC).
Who is sponsoring clinical trial NCT04851873?
This trial is sponsored by Novartis Pharmaceuticals, which has 1,375 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04851873's enrollment target sits among peer trials
24 1943rd of 2000 higher than 49 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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
- NCT02331979 · 24 participants · NA
Improving Bladder Function in SCI by Neuromodulation
- NCT03096197 · 24 participants · NA
Exoskeleton and Spinal Cord Stimulation for SCI
- NCT03223922 · 24 participants · NA
Neurocognitive Functioning With Genu-Sparing Whole Brain Radiation Therapy for Brain Metastases
- NCT03326921 · 24 participants · Phase 1
HA-1 T TCR T Cell Immunotherapy for the Treatment of Patients With Relapsed or Refractory Acute Leukemia After Donor Stem Cell Transplant
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