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Terminated Phase 3 Interventional

A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

ClinicalTrials.gov ID: NCT01803412

Public ClinicalTrials.gov record NCT01803412. 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.

ClinicalTrials.gov public records Last synced Sep 6, 2026, 1:31 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in US and Canadian Subjects With Duchenne Muscular Dystrophy.

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT01803412
Recruitment status
Terminated
Study type
Interventional
Phase
Phase 3
Lead sponsor
BioMarin Pharmaceutical
Industry
Enrollment
53 participants

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
5 Years and older
Sex
Male
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 30, 2013
Primary completion
May 31, 2016
Completion
Sep 30, 2016
Last update posted
Jun 13, 2018

2013 – 2016

United States locations

U.S. sites
10
U.S. states
9
U.S. cities
10
Facility City State ZIP Site status
UC Davis Medical Center Sacramento California 95817
Shriner's Hospital for Children Tampa Tampa Florida 12502
University of Iowa Iowa City Iowa 52242
Kennedy Krieger Institute Baltimore Maryland 21205
University of Minnesota Minneapolis Minnesota 55455
Columbia University Medical Center New York New York 10032
Duke University Medical Center Durham North Carolina 27710
Cincinnati Children's Hospital Medical Center Cincinnati Ohio 45229
Nationwide Children's Hospital Columbus Ohio 43205
Shriners Hospitals For Children Portland Oregon 97239

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 3 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT01803412, 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 Jun 13, 2018 · Synced Sep 6, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT01803412 live on ClinicalTrials.gov.

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