Last updated: 11/07/2018 07:13:24

A double-blind, escalating dose, randomized, placebo-controlled study assessing PK, safety, tolerability in non-ambulant DMD subjectsDEMAND I

GSK study ID
114118
See study documents
Clinicaltrials.gov ID
NCT01128855
EudraCT ID
2010-024566-22
EU CT Number
Not applicable
Trial status
Study complete
Study complete
Overview
Eligibility
Locations
Study documents
Results summary
Plain language summaries
Additional information

Trial overview

Official title: A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy
Trial description: The purpose of this study is investigate the pharmacokinetics, safety and tolerability of single
subcutaneous administration of GSK2402968 in non-ambulant boys with Duchenne muscular dystrophy
Primary purpose:
Treatment
Trial design:
Parallel Assignment
Masking:
Triple (Participant, Care Provider, Investigator)
Allocation:
Randomized
Primary outcomes:

Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F

Timeframe: 35 days

Incidence of Adverse Events

Timeframe: 35 days

Incidence of Injection Site Reactions

Timeframe: 35 days

Secondary outcomes:
Not applicable
Interventions:
  • Drug: 3 mg/kg GSK2402968
  • Drug: 6 mg/kg GSK2402968
  • Drug: 9 mg/kg GSK2402968
  • Drug: 12 mg/kg GSK2402968
  • Other: Placebo
    • Enrollment:
    20
    Primary completion date:
    Not applicable
    Observational study model:
    Not applicable
    Time perspective:
    Not applicable
    Clinical publications:
    Kevin M. Flanigan, Thomas Voit, Xiomara Q Rosales, Laurent Servais, John E. Kraus, Claire Wardell, Allison Morgan, Susie Dorricott, Joanna Nakielny, Naashika Quarcoo, Lia Liefaard, Tom Drury, Giles Campion, Padraig Wright. Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial. Neuromuscul Disord.2014;24(1):16-24
    Medical condition
    Muscular Dystrophies
    Product
    drisapersen
    Collaborators
    Not applicable
    Study date(s)
    July 2010 to October 2011
    Type
    Interventional
    Phase
    1

    Participation criteria

    Sex
    Male
    Age
    9+ years
    Accepts healthy volunteers
    No
    • Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed
    • by a sponsor approved DNA diagnostic technique covering all DMD gene exons,
    • Any additional mutation (such as an additional missing exon for DMD) that cannot
    • be treated with GSK2402968;
    Inclusion and exclusion criteria
    Inclusion criteria:
    • Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed by a sponsor approved DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation-dependent Probe Amplification), CGH (Comparative Genomic Hybridisation), SCAIP (Single Condition Amplification/Internal Primer) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by treatment with GSK2402968.
    • Age 9 years old or greater at Screening;
    • Male;
    • Non-ambulant (at least 1 year in a wheelchair) within the last 4 years;
    • Life expectancy at least three years;
    • Willingness and ability to comply with all protocol requirements and procedures;
    • QTc <450msec (based on single or average QTc value of triplicate ECGs obtained over a brief recording period). Note: QTc may be either QTcB or QTcF, machine read or manual overread;
    • Subjects must be willing to use adequate contraception (condoms or abstinence), from Screening until at least 5 months after the last dose of study drug;
    • Informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations).
    Exclusion criteria:
    • Any additional mutation (such as an additional missing exon for DMD) that cannot be treated with GSK2402968;
    • Current or history of liver or renal disease;
    • Acute illness within 4 weeks of anticipated administration of study medication, which may interfere with study assessments;
    • Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs, idebenone or other forms of Coenzyme Q10, within 6 months of the first administration of study medication;
    • Start of glucocorticosteroids within 6 months or non-stable use of glucocorticosteroids within 3 months of the anticipated first administration of study medication;
    • Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at Screening;
    • Symptomatic cardiomyopathy;
    • Use of alcohol from Screening through to the 1 month Follow-up visit ;
    • Any Child in Care.

    Trial location(s)

    Location
    Status
    Contact us
    Contact us
    Location
    GSK Investigational Site
    Columbus, Ohio, United States, 43205
    Status
    Study Complete
    Contact us
    Location
    GSK Investigational Site
    Paris cedex 13, France, 75651
    Status
    Study Complete
    Contact us

    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
    Study complete
    Actual primary completion date
    Not applicable
    Actual study completion date
    2011-25-10

    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

    Participate in clinical trial
    Additional information
    Results for study 114118 can be found on the GSK Clinical Study Register.
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