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

GSK study ID
114673
See study documents
Clinicaltrials.gov ID
NCT01910649
EudraCT ID
2007-004819-54
EU CT Number
Not applicable
Trial status
No longer a GSK study
No longer a GSK study
Overview
Eligibility
Locations
Study documents
Results summary
Plain language summaries
Additional information

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
    Primary completion date:
    2009-25-05
    Observational study model:
    Not applicable
    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)

    This study does not involve prospective enrollment of participants.

    Study documents

    Scientific result summary
    Available language(s): English
    Download document(s)

    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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