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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline
ClinicalTrials.gov Identifier:
NCT01128855
First received: May 20, 2010
Last updated: July 18, 2013
Last verified: July 2013
  Purpose

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 Intervention 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 Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:


Further study details as provided by GlaxoSmithKline:

Primary Outcome Measures:
  • Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F [ Time Frame: 35 days ] [ Designated as safety issue: No ]
  • Incidence of Adverse Events [ Time Frame: 35 days ] [ Designated as safety issue: Yes ]
  • Incidence of Injection Site Reactions [ Time Frame: 35 days ] [ Designated as safety issue: Yes ]

Enrollment: 20
Study Start Date: July 2010
Study Completion Date: October 2011
Primary Completion Date: October 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   9 Years and older
Genders 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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

No publications provided

Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT01128855     History of Changes
Other Study ID Numbers: 114118
Study First Received: May 20, 2010
Last Updated: July 18, 2013
Health Authority: United States: Institutional Review Board
United States: Food and Drug Administration

Keywords provided by GlaxoSmithKline:
Duchenne
Duchenne Muscular Dystrophy
DMD
968

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

ClinicalTrials.gov processed this record on August 21, 2014