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A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects (DEMAND I)

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ClinicalTrials.gov Identifier: NCT01128855
Recruitment Status : Completed
First Posted : May 24, 2010
Last Update Posted : July 18, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
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

Condition or disease Intervention/treatment Phase
Muscular Dystrophies Drug: 3 mg/kg GSK2402968 Drug: 6 mg/kg GSK2402968 Drug: 9 mg/kg GSK2402968 Drug: 12 mg/kg GSK2402968 Other: Placebo Phase 1

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
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
Actual Study Start Date : July 12, 2010
Primary Completion Date : October 25, 2011
Study Completion Date : October 25, 2011


Arms and Interventions

Arm Intervention/treatment
Experimental: Cohort 1
3 mg/kg GSK2402968 / placebo
Drug: 3 mg/kg GSK2402968
Weekly subcutaneous injection
Other: Placebo
Weekly Placebo
Experimental: Cohort 2
6 mg/kg GSK2402968 / placebo
Drug: 6 mg/kg GSK2402968
Weekly subcutaneous injection
Other: Placebo
Weekly Placebo
Experimental: Cohort 3
9 mg/kg GSK2402968 / placebo
Drug: 9 mg/kg GSK2402968
Weekly subcutaneous injection
Other: Placebo
Weekly Placebo
Experimental: Cohort 4
12 mg/kg GSK2402968 / placebo
Drug: 12 mg/kg GSK2402968
Weekly subcutaneous injection
Other: Placebo
Weekly Placebo


Outcome Measures

Primary Outcome Measures :
  1. Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F [ Time Frame: 35 days ]
  2. Incidence of Adverse Events [ Time Frame: 35 days ]
  3. Incidence of Injection Site Reactions [ Time Frame: 35 days ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   9 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01128855


Locations
United States, Ohio
GSK Investigational Site
Columbus, Ohio, United States, 43205
France
GSK Investigational Site
Paris cedex 13, France, 75651
Sponsors and Collaborators
GlaxoSmithKline
Investigators
Study Director: GSK Clinical Trials GlaxoSmithKline
More Information

Additional Information:
Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT01128855     History of Changes
Other Study ID Numbers: 114118
First Posted: May 24, 2010    Key Record Dates
Last Update Posted: July 18, 2017
Last Verified: July 2017

Keywords provided by GlaxoSmithKline:
Duchenne
Duchenne Muscular Dystrophy
DMD
968

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked