NCT01128855

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

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jul 2010

Geographic Reach
2 countries

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 20, 2010

Completed
4 days until next milestone

First Posted

Study publicly available on registry

May 24, 2010

Completed
2 months until next milestone

Study Start

First participant enrolled

July 12, 2010

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 25, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 25, 2011

Completed
Last Updated

July 18, 2017

Status Verified

July 1, 2017

Enrollment Period

1.3 years

First QC Date

May 20, 2010

Last Update Submit

July 13, 2017

Conditions

Muscular Dystrophies

Keywords

DuchenneDuchenne Muscular DystrophyDMD968

Outcome Measures

Primary Outcomes (3)

  • Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F

    35 days

  • Incidence of Adverse Events

    35 days

  • Incidence of Injection Site Reactions

    35 days

Study Arms (4)

Cohort 1

EXPERIMENTAL

3 mg/kg GSK2402968 / placebo

Drug: 3 mg/kg GSK2402968Other: Placebo

Cohort 2

EXPERIMENTAL

6 mg/kg GSK2402968 / placebo

Drug: 6 mg/kg GSK2402968Other: Placebo

Cohort 3

EXPERIMENTAL

9 mg/kg GSK2402968 / placebo

Drug: 9 mg/kg GSK2402968Other: Placebo

Cohort 4

EXPERIMENTAL

12 mg/kg GSK2402968 / placebo

Drug: 12 mg/kg GSK2402968Other: Placebo

Interventions

3 mg/kg GSK2402968DRUG

Weekly subcutaneous injection

Cohort 1
6 mg/kg GSK2402968DRUG

Weekly subcutaneous injection

Cohort 2
9 mg/kg GSK2402968DRUG

Weekly subcutaneous injection

Cohort 3
12 mg/kg GSK2402968DRUG

Weekly subcutaneous injection

Cohort 4
PlaceboOTHER

Weekly Placebo

Cohort 1Cohort 2Cohort 3Cohort 4

Eligibility Criteria

Age9 Years+
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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).

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

GSK Investigational Site

Columbus, Ohio, 43205, United States

Location

GSK Investigational Site

Paris, 75651, France

Location

Related Links

MeSH Terms

Conditions

Muscular DystrophiesMuscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-Linked

Study Officials

  • GSK Clinical Trials

    GlaxoSmithKline

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 20, 2010

First Posted

May 24, 2010

Study Start

July 12, 2010

Primary Completion

October 25, 2011

Study Completion

October 25, 2011

Last Updated

July 18, 2017

Record last verified: 2017-07

Locations

FR(1)US(1)