A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD114044)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline
ClinicalTrials.gov Identifier:
NCT01254019
First received: October 21, 2010
Last updated: July 24, 2014
Last verified: June 2014

October 21, 2010
July 24, 2014
December 2010
June 2013   (final data collection date for primary outcome measure)
To assess the efficacy of subcutaneous 6 mg/kg GSK2402968 versus placebo; specifically to have 90% power to detect a difference in 6MWD between GSK2402968 and placebo of 30 meters, assuming a common standard deviation of 55meters [ Time Frame: one year ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT01254019 on ClinicalTrials.gov Archive Site
  • To evaluate the safety and tolerability of subcutaneous 6 mg/kg GSK2402968 with respect to AEs, ECG results, vital signs and laboratory tests [ Time Frame: one year ] [ Designated as safety issue: Yes ]
  • Plasma concentration of subcutaneous 6 mg/kg GSK2402968 calculated at each time point for each subject (sample size (n), mean, standard deviation (SD), percentage of coefficient of variation (%CV), geometric mean, median, minimum, and maximum [ Time Frame: one year ] [ Designated as safety issue: No ]
  • To evaluate the difference on quality of life of between GSK2402968 and placebo using PedsQOL, CGI-I and HUI [ Time Frame: one year ] [ Designated as safety issue: No ]
  • To evaluate the safety and tolerability of subcutaneous 6 mg/kg GSK2402968 with respect to AEs, ECG results, vital signs and laboratory tests [ Time Frame: one year ] [ Designated as safety issue: Yes ]
  • Plasma concerntration of subcutaneous 6 mg/kg GSK2402968 calculated at each time point for each subject (sample size (n), mean, standard deviation (SD), percentage of coefficient of variation (%CV), geometric mean, median, minimum, and maximum [ Time Frame: one year ] [ Designated as safety issue: No ]
  • To evaluate the diffrence on quality of life of between GSK2402968 and placebo using PedsQOL, CGI-I and HUI [ Time Frame: one year ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
A Phase III, Randomized, Double Blind, Placebo-controlled Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

The purpose of this study is to determine whether GSK2402968 is effective in the treatment of ambulant boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.

Not Provided
Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Muscular Dystrophies
Drug: GSK2402968 6mg/kg/week
subcutaneous
  • Experimental: GSK2402968
    6mg/kg
    Intervention: Drug: GSK2402968 6mg/kg/week
  • Experimental: Placebo
    dose-matched
    Intervention: Drug: GSK2402968 6mg/kg/week
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
186
June 2013
June 2013   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Ambulant subjects with Duchenne muscular dystrophy resulting from a mutation/deletion within the DMD gene, confirmed by a state-of-the-art 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 GSK2402968-induced DMD exon 51 skipping.
  • Males, aged at least 5 years, and with life expectancy of at least 1 year
  • Able to complete 6MWD test with minimal distance of at least 75m at each predrug visit. In addition, results of 6MWD must be within 20% of each other at each pre-drug visit
  • Receiving glucocorticoids for a minimum of 6 months immediately prior to screening, with no significant change in total daily dosage or dosing regimen for a minimum of 3 months immediately prior to screening and a reasonable expectation that total daily dosage and dosing regimen will not change significantly for the duration of the study
  • QTc <450msec (based on single or average QTc value of triplicate ECGs obtained over a brief recording period), or <480 msec for subjects with Bundle Branch Block. Note: QTc may be either QTcB or QTcF, and machine read or manual overread.
  • Subjects, where appropriate, must be willing to use adequate contraception (condoms or abstinence) for the duration of the study and for at least 5 months after the last dose of study drug.
  • Willing and able to comply with all protocol requirements and procedures,
  • Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations).
  • French subjects: In France, a subject will be eligible for inclusion in this study only if either affiliated to or a beneficiary of a social security category.

Exclusion Criteria:

  • Any additional missing exon for DMD that cannot be treated with GSK2402968
  • Current or history of liver or renal disease or impairment
  • Acute illness within 4 weeks of the first anticipated administration of study medication which may interfere with study assessments
  • Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs, within 6 months of the first administration of study medication; and idebenone or other forms of Coenzyme Q10 within 1 month of the first administration of study medication.
  • Current or anticipated participation in any investigational clinical studies
  • Positive hepatitis B surface antigen, hepatitis C antibody test (if verified via RIBA or PCA testing), or human immunodeficiency virus (HIV) test at screening,
  • Symptomatic cardiomyopathy. If subject has a left ventricular ejection fraction <45% at Screening, the investigator should discuss inclusion of subject in the study with the medical monitor,
  • Children in Care. The definition of a Child in Care is a child who has been placed under the control or protection of an agency, organisation, institution or entity by the courts, the government or a government body, acting in accordance with powers conferred on them by law or regulation. The definition of a child in care can include a child cared for by foster parents or living in a care home or institution, provided that the arrangement falls within the definition above. The definition of a child in care does not include a child who is adopted or has an appointed legal guardian.
Male
5 Years and older
No
Contact information is only displayed when the study is recruiting subjects
Argentina,   Belgium,   Brazil,   Canada,   Chile,   Czech Republic,   Denmark,   France,   Germany,   Hungary,   Italy,   Japan,   Korea, Republic of,   Netherlands,   Norway,   Poland,   Russian Federation,   Spain,   Taiwan,   Turkey
 
NCT01254019
114044
Yes
GlaxoSmithKline
GlaxoSmithKline
Not Provided
Study Director: GSK Clinical Trials GlaxoSmithKline
GlaxoSmithKline
June 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP