Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT01803412 · ClinicalTrials.gov registry record · Phase 3
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
A Phase 3 study, sponsored by BioMarin Pharmaceutical.
- Terminated
- Registry status
- Phase 3
- Development phase
- 53
- Enrollment target
NCT01803412: Clinical Trial Phase 3 study, sponsored by BioMarin Pharmaceutical.
NCT01803412 is a Phase 3 study that was terminated before completion, run by BioMarin Pharmaceutical. The registered enrollment target is 53 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (93% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01803412, a Phase 3 study, was terminated before completion, sponsored by BioMarin Pharmaceutical.
- TERMINATED
- Registry status
- Phase 3
- Development phase
- 53 participants
- Enrollment target
Study Summary
This is a phase III, multicenter, open-label, uncontrolled extension study in male subjects with DMD open to eligible US and Canadian subjects who previously participated in the following studies of drisapersen: DMD114876, DMD114044 and DMD114349. Subjects will receive 6mg/kg subcutaneous drisapersen on a weekly basis. For subjects who have previously experienced significant safety or tolerability issues or who experience these during the study, there is the potential of an alternate intermittent dosing arm that will be given as a regimen of 6 mg/kg weekly for 8 weeks followed by 4 weeks off treatment. For subjects who experience or have previously experienced significant safety/tolerability issues, side effects or reactions or intermittent dosing, intravenous dosing will be made available.
Primary Outcome
AEs will be assessed from Pre-baseline visit until 20 weeks after the subject has either completed the study or withdrawn from treatment early. AEs are any untoward medical occurrence in a patient or clinical investigation subject, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product
Interventions
- DRUG Drisapersen
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 53 participants |
| Start Date | 2013-05-01 |
| Est. Completion | 2016-10-01 |
| Phase | Phase 3 |
Why NCT01803412 stopped before completion
NCT01803412 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 53 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (93% lower).
The record links to 0 conditions, and to 1 intervention - of which Drisapersen is the first listed.
NCT01803412 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01803412 about?
NCT01803412 is a clinical study titled "A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects". This is a phase III, multicenter, open-label, uncontrolled extension study in male subjects with DMD open to eligible US and Canadian subjects who previously participated in the following studies of drisapersen: DMD114876, DMD114044 and DMD114349. Subjects will receive 6mg/kg subcutaneous drisaperse...
What is the current status of trial NCT01803412?
This trial is currently terminated. It is a Phase 3 study. The enrollment target is 53 participants. The study started on 2013-05-01. Estimated completion is 2016-10-01.
What interventions are being tested in trial NCT01803412?
The interventions under investigation include: Drisapersen (DRUG).
Who is sponsoring clinical trial NCT01803412?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01803412's enrollment target sits among peer trials
53 1839th of 2000 higher than 160 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
- NCT04177004 · 53 participants · Phase 1
Human Lysozyme Goat Milk for the Prevention of Graft Versus Host Disease in Patients With Blood Cancer Undergoing a Donor Stem Cell Transplant
- NCT04199741 · 53 participants · Phase 1
PET/CT Imaging of Small Cell Lung Cancer Using 89Zr-DFO-SC16.56
- NCT04835129 · 53 participants · Phase 2
Isatuximab, Pomalidomide, Elotuzumab and Dexamethasone in Relapsed and/or Refractory Multiple Myeloma
- NCT04913805 · 53 participants · Phase 2
Matching Perfusion and Metabolic Activity in HFpEF
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