Clinical trial NCT01128855: A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Muscular Dystrophies
Keywords: Duchenne
Duchenne Muscular Dystrophy
DMD
968
Lead sponsor: GlaxoSmithKline
Sponsor class: INDUSTRY
First posted: 2010-05-24
Enrollment: 20
Sex: MALE
Minimum age (years): 9
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


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.

Primary outcomes:
measure: Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F
timeFrame: 35 days
measure: Incidence of Adverse Events
timeFrame: 35 days
measure: Incidence of Injection Site Reactions
timeFrame: 35 days
Secondary outcomes:
Other outcomes:
Locations:
city: Columbus
country: United States
facility: GSK Investigational Site
state: Ohio
city: Paris cedex 13
country: France
facility: GSK Investigational Site
Study officials:
affiliation: GlaxoSmithKline
name: GSK Clinical Trials
role: STUDY_DIRECTOR
References: