Overview

This trial has been terminated.

Condition muscular dystrophies
Treatment drisapersen
Phase phase 2
Sponsor BioMarin Pharmaceutical
Start date March 2008
End date September 2016
Trial size 12 participants
Trial identifier NCT01910649, 114673, 2007-004819-54

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Extension phase of treatment. Intravenous dosing of drisapersen will be investigated as an alternative route of administration
drisapersen PRO051
Subcutaneous and Intravenous

Primary Outcomes

Measure
Acute phase: Safety data
time frame: 18 weeks
Acute phase and Continued Treatment Phase : Pharmacokinetics measured by T1/2, Cmax, Ctrough, 7d, tmax, and volume of distribution and clearance
time frame: 18 weeks
Acute phase and Continued Treatment Phase : Safety as assessed by the collection of adverse events (AEs)
time frame: 72 weeks
Continued Treatment Phase :Safety as assessed by laboratory parameters
time frame: 72 weeks

Secondary Outcomes

Measure
Acute phase: Production of exon skip 51 messenger Ribonucleic acid (mRNA)
time frame: 18 weeks
Acute phase: Presence of dystrophin expression
time frame: 18 weeks
Acute phase: Muscle function
time frame: 18 weeks
Acute phase: Muscle strength
time frame: 18 weeks
Continued Treatment Phase: Exon skip efficiency
time frame: 72 weeks
Continued Treatment Phase Dystrophin expression in muscle biopsy
time frame: 72 weeks
Continued Treatment Phase: Muscle function
time frame: 300 weeks
Continued Treatment Phase: Muscle strength
time frame: 300 weeks

Eligibility Criteria

Male participants from 5 years up to 16 years old.

Inclusion Criteria: - Boys aged between 5 and 16 years inclusive. - Duchenne muscular dystrophy resulting from a mutation correctable by treatment with PRO051. - Not ventilator dependent. - Life expectancy of at least six months. - No previous treatment with investigational medicinal treatment within six months prior to the study. - Willing and able to adhere to the study visit schedule and other protocol requirements. Exclusion Criteria: - Aberrant RNA splicing and/or aberrant response to PRO051, detected by in vitro PRO051 assay during screening. - Known presence of dystrophin in 5% of fibers in a pre-study diagnostic muscle biopsy. - Severe muscle abnormalities defined as increased signal intensity in >50% of the tibialis anterior muscle at MRI. - FEV1 and/or FVC <60% of predicted. - Current or history of liver or renal disease. - Acute illness within 4 weeks prior to treatment (Day 1) which may interfere with the measurements. - Severe mental retardation which in the opinion of the investigator prohibits participation in this study. - Severe cardiac myopathy which in the opinion of the investigator prohibits participation in this study. - Need for mechanical ventilation. - Creatinine concentration above 1.5 times the upper limit of normal (age corrected). - Serum ASAT and/or ALAT concentration(s) which suggest hepatic impairment. - Use of anticoagulants, antithrombotics or antiplatelet agents. - Subject has donated blood less than 90 days before the start of the study. - Current or history of drug and/or alcohol abuse. - Participation in another trial with an investigational product.

Additional Information

Official title A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for Intravenous Dosing as an Alternative Route of Administration
Principal investigator N Goemans, Dr.
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by BioMarin Pharmaceutical.