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

Muscular Dystrophies Clinical Trial

— DEMAND I  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01128855
• Other study ID #: 114118
• Status: Completed
• Phase: Phase 1
• First received: May 20, 2010
• Last updated: July 13, 2017
• Start date: July 12, 2010
• Est. completion date: October 25, 2011

Study information

• Verified date: July 2017
• Source: GlaxoSmithKline
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: October 25, 2011
• Est. primary completion date: October 25, 2011
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 9 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• 3 mg/kg GSK2402968
Weekly subcutaneous injection
• 6 mg/kg GSK2402968
Weekly subcutaneous injection
• 9 mg/kg GSK2402968
Weekly subcutaneous injection
• 12 mg/kg GSK2402968
Weekly subcutaneous injection

Other:
• Placebo
Weekly Placebo

Locations

Country	Name	City	State
France	GSK Investigational Site	Paris cedex 13
United States	GSK Investigational Site	Columbus	Ohio

Sponsors (1)

Lead Sponsor	Collaborator
GlaxoSmithKline

Countries where clinical trial is conducted

United States,  France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F		35 days
Primary	Incidence of Adverse Events		35 days
Primary	Incidence of Injection Site Reactions		35 days

External Links

•   Results for study 114118 can be found on the GSK Clinical Study Register.