Status:
TERMINATED
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
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Muscular Dystrophies
Eligibility:
MALE
5-16 years
Phase:
PHASE2
Brief 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 corr...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
March 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2016
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT01910649
Start Date
March 1 2008
End Date
September 1 2016
Last Update
November 7 2016
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