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A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration

NCT01910649 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2016-11-07

No results posted yet for this study

Summary

The purpose of the extension phase of this study is to determine whether Drisapersen is effective in the treatment of boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.

Conditions

  • Muscular Dystrophies

Interventions

DRUG

Drisapersen

Subcutaneous and Intravenous

Sponsors & Collaborators

Principal Investigators

  • N Goemans, Dr. · UZ Leuven

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Max Age
16 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-03-31
Primary Completion
2016-09-30
Completion
2016-09-30

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01910649 on ClinicalTrials.gov