Last updated: 03/05/2020 21:00:13
This study has been divested to Prosensa
A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy
Clinicaltrials.gov ID
EudraCT ID
EU CT Number
Not applicable
Trial status
No longer a GSK study
No longer a GSK study
Trial overview
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 PRO051 in patients with Duchenne muscular dystrophy
Trial description: The purpose of this study is to determine whether an escalating dose of GSK2402968 is effective in the treatment of boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.
Primary purpose:
Treatment
Trial design:
Single Group Assignment
Masking:
None (Open Label)
Allocation:
N\A
Primary outcomes:
Acute phase and Continued Treatment Phase : Pharmacokinetics measured by T1/2, Cmax, Ctrough, 7d, tmax, and volume of distribution and clearance
Timeframe: 18 weeks
Continued Treatment Phase :Safety as assessed by laboratory parameters
Timeframe: 72 weeks
Acute phase: Safety data
Timeframe: 18 weeks
Acute phase and Continued Treatment Phase : Safety as assessed by the collection of adverse events (AEs)
Timeframe: 72 weeks
Secondary outcomes:
Acute phase: Presence of dystrophin expression
Timeframe: 18 weeks
Continued Treatment Phase: Muscle strength
Timeframe: 72 weeks
Continued Treatment Phase: Exon skip efficiency
Timeframe: 72 weeks
Continued Treatment Phase Dystrophin expression in muscle biopsy
Timeframe: 72 weeks
Acute phase: Production of exon skip 51 messenger Ribonucleic acid (mRNA)
Timeframe: 18 weeks
Acute phase: Muscle function
Timeframe: 18 weeks
Continued Treatment Phase: Muscle function
Timeframe: 72 weeks
Acute phase: Muscle strength
Timeframe: 18 weeks
Interventions:
Drug: GSK2402968
Enrollment:
12
Observational study model:
Not applicable
Primary completion date:
2009-25-05
Time perspective:
Not applicable
Clinical publications:
Goemans NM, Tulinius M, van der Akker JT, et al. Systemic Administration of PRO051 in Duchenne’s Muscular Dystrophy. New Engl J Med. 2011; 364(16): 1513-22.
Medical condition
Muscular Dystrophies
Product
drisapersen
Collaborators
Prosensa Therapeutics
Study date(s)
March 2008 to December 2015
Type
Interventional
Phase
1/2
Participation criteria
Sex
Male
Age
5 - 16 Year
Accepts healthy volunteers
none
- Boys aged between 5 and 16 years inclusive.
- Duchenne muscular dystrophy resulting from a mutation correctable by treatment with PRO051.
- 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.
Inclusion and exclusion 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.
Trial location(s)
No location data available.
Study documents
Scientific result summary
Available language(s): English
If you wish to request for full study report, please contact - [email protected]
Results overview
Refer to study documents
Recruitment status
No longer a GSK study
Actual primary completion date
2009-25-05
Actual study completion date
Not applicable
Plain language summaries
Plain language summaries of clinical trial results for Phase 2-4 clinical trials that were initiated on or after January 2022 will be posted by GSK within one year following study completion.
Additional information about the trial
Additional information
Not applicable
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