Last updated: 11/03/2018 23:28:26

A study to evaluate potential biomarkers of muscular dystrophy

GSK study ID
205703
See study documents
Clinicaltrials.gov ID
Not applicable
EudraCT ID
Not applicable
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 study to evaluate potential biomarkers of muscular dystrophy
Trial description: The study will explore to determine whether tetranor Prostaglandin D2 metabolite (PGDM) levels (expressed as nanogram [ng]/milligram [mg] creatinine) are increased in the urine of males diagnosed with Duchenne Muscular Dystrophy (DMD) when compared to healthy, aged-matched subjects. Data from this study will be used to determine whether tetranor PGDM is a useful marker of pharmacodynamic activity for experimental therapies that act in the Prostaglandin D2 (PGD2) pathway in both preclinical and clinical studies in DMD. In addition, relationship between PGDM level (and other biomarkers) and ambulatory status will be explored.
Primary purpose:
Not applicable
Trial design:
Not applicable
Masking:
Not applicable
Allocation:
Not applicable
Primary outcomes:

Creatinine levels in urine at baseline

Timeframe: Up to 15 days

Tetranor PGDM levels in urine at baseline

Timeframe: Up to 15 days

Secondary outcomes:

Change from baseline in urine biomarkers in DMD subjects

Timeframe: Baseline and 12 month

Change from baseline in ambulatory status in DMD subjects

Timeframe: Baseline and 12 month

Interventions:
  • Other: Ambulatory status
  • Other: Urine sample collection
    • Enrollment:
    71
    Primary completion date:
    2018-20-04
    Observational study model:
    Case-Control
    Time perspective:
    Prospective
    Clinical publications:
    Not applicable
    Medical condition
    Muscular Dystrophies
    Product
    Not applicable
    Collaborators
    Not applicable
    Study date(s)
    October 2016 to April 2018
    Type
    Observational
    Phase
    Not applicable

    Participation criteria

    Sex
    Male
    Age
    5Years - 16Years
    Accepts healthy volunteers
    Yes
    • Males >= 5 and =< 16 years of age at the time of informed consent
    • Diagnosis (by family/clinical history) of DMD or healthy male controls based on medical history may not have known myopathy/muscular dystrophy or muscle contractures (cerebral palsy)
    • Previously participated in the same study
    Inclusion and exclusion criteria
    Inclusion criteria:
    • Males >= 5 and =< 16 years of age at the time of informed consent
    • Diagnosis (by family/clinical history) of DMD or healthy male controls based on medical history may not have known myopathy/muscular dystrophy or muscle contractures (cerebral palsy)
    Exclusion criteria:
    • Previously participated in the same study

    Trial location(s)

    This study does not involve prospective enrollment of participants.

    Study documents

    Statistical analysis plan
    Available language(s): English
    Download document(s)
    Scientific result summary
    Available language(s): English
    Download document(s)
    Protocol
    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
    2018-20-04
    Actual study completion date
    2018-20-04

    Plain language summaries

    Not applicable. GSK’s transparency policy provides for Plain Language Summaries for Interventional studies.

    Additional information about the trial

    Not applicable
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