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

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

Status
Completed
Phases
Phase 1
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT01128855
Acronym
DEMAND I
Enrollment
20
Registered
2010-05-24
Start date
2010-07-12
Completion date
2011-10-25
Last updated
2017-07-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Muscular Dystrophies

Keywords

Duchenne, Duchenne Muscular Dystrophy, DMD, 968

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

Interventions

DRUG12 mg/kg GSK2402968

Weekly subcutaneous injection

OTHERPlacebo

Weekly Placebo

DRUG3 mg/kg GSK2402968

Weekly subcutaneous injection

DRUG6 mg/kg GSK2402968

Weekly subcutaneous injection

DRUG9 mg/kg GSK2402968

Weekly subcutaneous injection

Sponsors

GlaxoSmithKline
Lead SponsorINDUSTRY

Study design

Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)

Eligibility

Sex/Gender
MALE
Age
9 Years to No maximum
Healthy volunteers
No

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.

Design outcomes

Primary

MeasureTime frame
Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F35 days
Incidence of Adverse Events35 days
Incidence of Injection Site Reactions35 days

Countries

France, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026