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

NCT ID: NCT01910649

Last Updated: 2016-11-07

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2008-03-31
• Study Completion Date: 2016-09-30

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 corrected by exon 51 skipping.

Conditions

Muscular Dystrophies

Keywords

drisapersen

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Drisapersen

Extension phase of treatment. Intravenous dosing of drisapersen will be investigated as an alternative route of administration

Group Type: EXPERIMENTAL

Drisapersen

Intervention Type: DRUG

Subcutaneous and Intravenous

Interventions

Drisapersen

Subcutaneous and Intravenous

Intervention Type: DRUG

Other Intervention Names

PRO051

Eligibility Criteria

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.

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 16 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

BioMarin Pharmaceutical

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

N Goemans, Dr.

Role: PRINCIPAL_INVESTIGATOR

UZ Leuven

References

Goemans NM, Tulinius M, van den Akker JT, Burm BE, Ekhart PF, Heuvelmans N, Holling T, Janson AA, Platenburg GJ, Sipkens JA, Sitsen JM, Aartsma-Rus A, van Ommen GJ, Buyse G, Darin N, Verschuuren JJ, Campion GV, de Kimpe SJ, van Deutekom JC. Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med. 2011 Apr 21;364(16):1513-22. doi: 10.1056/NEJMoa1011367. Epub 2011 Mar 23.

Reference Type: RESULT

PMID: 21428760 (View on PubMed)

Goemans NM, Tulinius M, van den Hauwe M, Kroksmark AK, Buyse G, Wilson RJ, van Deutekom JC, de Kimpe SJ, Lourbakos A, Campion G. Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study. PLoS One. 2016 Sep 2;11(9):e0161955. doi: 10.1371/journal.pone.0161955. eCollection 2016.

Reference Type: DERIVED

PMID: 27588424 (View on PubMed)

Other Identifiers

2007-004819-54

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

114673

Identifier Type: -

Identifier Source: org_study_id