A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD114044)
This study has been completed.
Information provided by (Responsible Party):
First received: October 21, 2010
Last updated: December 19, 2013
Last verified: December 2013
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.
Drug: GSK2402968 6mg/kg/week
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
||A Phase III, Randomized, Double Blind, Placebo-controlled Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
Primary Outcome Measures:
- 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 ]
Secondary Outcome Measures:
- 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 ]
| Study Start Date:
| Study Completion Date:
| Primary Completion Date:
||June 2013 (Final data collection date for primary outcome measure)
Drug: GSK2402968 6mg/kg/week
Drug: GSK2402968 6mg/kg/week
|Ages Eligible for Study:
||5 Years and older
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- 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.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01254019
||GSK Clinical Trials
No publications provided
History of Changes
|Other Study ID Numbers:
|Study First Received:
||October 21, 2010
||December 19, 2013
||France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Canada: Health Canada
Germany: Bundesinstitut für Arzneimittel und Medizinprodukte
Europe: European Medicines Agency
Keywords provided by GlaxoSmithKline:
Duchenne Muscular Dystrophy
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on March 06, 2014
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn