TerminatedNCT01910649Updated Nov 6, 2016

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

A Phase 2 interventional study of Drisapersen in Muscular Dystrophies, sponsored by BioMarin Pharmaceutical. Terminated. Open to male participants aged 5 Years to 16 Years. Per ClinicalTrials.gov, last updated 2016-11-06.

Sponsored by BioMarin Pharmaceutical · Phase 2, Interventional, and Treatment

Why this study was terminated

Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD.

Phase: Phase 2
Study type: Interventional
Enrollment: 12
Allocation: Not applicable
Ages: 5 Years to 16 Years
Sex: Male

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 studied

Muscular Dystrophies

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Keywords

drisapersen

In context

Muscular Dystrophies

548 studies on the registry are indexed under Muscular Dystrophies; 89 are open to participants now.

This study's enrollment of 12 is below the median of 24 across 344 interventional studies indexed under Muscular Dystrophies.

Browse Muscular Dystrophies studies →

Lead sponsor

BioMarin Pharmaceutical is the lead sponsor of 110 studies on the registry; 13 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 12 (71%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 5 Years to 16 Years
Sexes eligible: Male
Accepts healthy volunteers: No

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

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.

Study design

Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 12 participants (actual)

Study arms

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

Interventions

DrugDrisapersen
Subcutaneous and Intravenous
Also known as: PRO051

What researchers measure

Primary outcomes

Acute phase: Safety data
Summarized per dose group
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
Plasma concentration versus time profiles of PRO051 (GSK2402968)
Time frame: 18 weeks
Acute phase and Continued Treatment Phase : Safety as assessed by the collection of adverse events (AEs)
Change from baseline and summarized values
Time frame: 72 weeks
Continued Treatment Phase :Safety as assessed by laboratory parameters
Change from baseline and summarized values
Time frame: 72 weeks

Secondary outcomes

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
Timed tests and 6-minutes walk
Time frame: 18 weeks
Acute phase: Muscle strength
Quantitative Muscle Testing \[QMT\]- Cooperative International Neuromuscular Research Group (CINRG) and Manual Muscle Testing \[MMT\]
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
Timed tests and 6-minutes walk
Time frame: 300 weeks
Continued Treatment Phase: Muscle strength
Handheld myometry and spirometry
Time frame: 300 weeks

Study locations

No study locations are listed for this record.

References and documents

Publications

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. Erratum In: N Engl J Med. 2011 Oct 6;365(14):1361. PubMed 21428760 ↗
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. PubMed 27588424 ↗

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Nov 6, 2016, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT01910649
Lead sponsor: BioMarin Pharmaceutical
Responsible party: Sponsor
First posted: Jul 29, 2013
Start date: Mar 2008
Primary completion: Sep 2016
Completion: Sep 2016
Last update: Nov 6, 2016

Study contacts

N Goemans, Dr.

principal investigator · UZ Leuven

Oversight

Data monitoring committee: Yes

View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is terminated, as verified in Nov 2016. You cannot join it, but the record below documents what was studied.

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