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

NCT ID: NCT01128855

Last Updated: 2017-07-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-07-12
• Study Completion Date: 2011-10-25

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

Conditions

Muscular Dystrophies

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Cohort 1

3 mg/kg GSK2402968 / placebo

Group Type: EXPERIMENTAL

3 mg/kg GSK2402968

Intervention Type: DRUG

Weekly subcutaneous injection

Placebo

Intervention Type: OTHER

Weekly Placebo

Cohort 2

6 mg/kg GSK2402968 / placebo

Group Type: EXPERIMENTAL

6 mg/kg GSK2402968

Intervention Type: DRUG

Weekly subcutaneous injection

Placebo

Intervention Type: OTHER

Weekly Placebo

Cohort 3

9 mg/kg GSK2402968 / placebo

Group Type: EXPERIMENTAL

9 mg/kg GSK2402968

Intervention Type: DRUG

Weekly subcutaneous injection

Placebo

Intervention Type: OTHER

Weekly Placebo

Cohort 4

12 mg/kg GSK2402968 / placebo

Group Type: EXPERIMENTAL

12 mg/kg GSK2402968

Intervention Type: DRUG

Weekly subcutaneous injection

Placebo

Intervention Type: OTHER

Weekly Placebo

Interventions

3 mg/kg GSK2402968

Weekly subcutaneous injection

Intervention Type: DRUG

6 mg/kg GSK2402968

Weekly subcutaneous injection

Intervention Type: DRUG

9 mg/kg GSK2402968

Weekly subcutaneous injection

Intervention Type: DRUG

12 mg/kg GSK2402968

Weekly subcutaneous injection

Intervention Type: DRUG

Placebo

Weekly Placebo

Intervention Type: OTHER

Eligibility 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.

• Minimum Eligible Age: 9 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

GlaxoSmithKline

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

GSK Clinical Trials

Role: STUDY_DIRECTOR

GlaxoSmithKline

Locations

GSK Investigational Site

Columbus, Ohio, United States

GSK Investigational Site

Paris, , France

Countries

United States; France

Other Identifiers

114118

Identifier Type: -

Identifier Source: org_study_id