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

Phase 1

Completed

• Conditions: Muscular Dystrophies
• Interventions: Drug: 6 mg/kg GSK2402968; Drug: 12 mg/kg GSK2402968; Drug: 3 mg/kg GSK2402968; Drug: 9 mg/kg GSK2402968; Other: Placebo

• Registration Number: NCT01128855
• Lead Sponsor: GlaxoSmithKline

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2010-05-20
• Study First Submitted QC: 2010-05-21
• Study First Posted: 2010-05-24
• Study Start: 2010-07-12
• Primary Completion: 2011-10-25
• Study Completion: 2011-10-25
• Status Verified: 2017-07
• Last Update Submitted: 2017-07-13
• Last Update Posted: 2017-07-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 20

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort 2	6 mg/kg GSK2402968	6 mg/kg GSK2402968 / placebo
Cohort 3	Placebo	9 mg/kg GSK2402968 / placebo
Cohort 4	12 mg/kg GSK2402968	12 mg/kg GSK2402968 / placebo
Cohort 1	Placebo	3 mg/kg GSK2402968 / placebo
Cohort 1	3 mg/kg GSK2402968	3 mg/kg GSK2402968 / placebo
Cohort 2	Placebo	6 mg/kg GSK2402968 / placebo
Cohort 4	Placebo	12 mg/kg GSK2402968 / placebo
Cohort 3	9 mg/kg GSK2402968	9 mg/kg GSK2402968 / placebo

Primary Outcome Measures

Name	Time	Method
Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F	35 days
Incidence of Adverse Events	35 days
Incidence of Injection Site Reactions	35 days

Trial Locations

Locations (1)

GSK Investigational Site; 🇫🇷 Paris cedex 13, France