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Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

This study is currently recruiting participants.
Verified May 2013 by GlaxoSmithKline
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline
ClinicalTrials.gov Identifier:
NCT01480245
First received: November 23, 2011
Last updated: May 16, 2013
Last verified: May 2013
History of Changes
  Purpose

The purpose of this study is to explore long-term safety, tolerability and efficacy of GSK2402968 in DMD subjects who previously participated in either DMD114117 or DMD114044.


Condition Intervention Phase
Muscular Dystrophies
 Drug: GSK2402968
 Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by GlaxoSmithKline:

Primary Outcome Measures:
  • Differences between the 6MWD at baseline and Week 104 [ Time Frame: 104 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Timed Function tests [ Time Frame: 104 weeks ] [ Designated as safety issue: No ]
  • Muscle strength [ Time Frame: 104 weeks ] [ Designated as safety issue: No ]
  • North Star Ambulatory Assessment Scores [ Time Frame: 104 weeks ] [ Designated as safety issue: No ]
  • Creatine kinase Serum concentrations [ Time Frame: 104 weeks ] [ Designated as safety issue: No ]
  • Pulmonary Function [ Time Frame: 104 weeks ] [ Designated as safety issue: No ]
  • Pediatric Quality of Life Neuromuscular module [ Time Frame: 104weeks ] [ Designated as safety issue: No ]
  • Clinician Global Impression of Improvement [ Time Frame: 104 weeks ] [ Designated as safety issue: No ]
  • Health Utilities Index [ Time Frame: 104 weeks ] [ Designated as safety issue: No ]
  • Frequency of accidental falls during 6 Minute Walk Distance test [ Time Frame: 104 weeks ] [ Designated as safety issue: No ]
  • Functional Outcomes Assessment [ Time Frame: 104 weeks ] [ Designated as safety issue: No ]
  • Time to major disease milestones [ Time Frame: 104 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 220
Study Start Date: September 2011
Estimated Study Completion Date: December 2014
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Continuous Dosing
GSK2402968 6mg/kg/week
 Drug: GSK2402968
Experimental: Intermittent Dosing
GSK2402968 6mg/kg/week
 Drug: GSK2402968
No Intervention: Natural History

  Eligibility

Ages Eligible for Study:   5 Years and older
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Previous participation in either DMD114117 or DMD114044
  • Continued use of glucocorticoids
  • Willing and able to comply with all protocol requirements
  • Able to give informed consent
  • French subjects: Eligible for inclusion only if either affiliated to or a beneficiary of a social security category.

Exclusion Criteria:

  • Subject experienced a serious adverse event or who met safety stopping criteria that remains unresolved from DMD114117 or DMD114044, which in opinion of the investigator could have been attributable to study medication and is ongoing,
  • Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs,except GSK2402968, within 1 month of the first administration of study medication,
  • Current or anticipated participation in any investigational clinical studies,
  • History of significant medical disorder which may confound the interpretation of either efficacy or safety data e.g. current history of renal or liver disease/impairment, history of inflammatory disease.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01480245

Contacts
Contact: US GSK Clinical Call Center 877-379-3718 GSKClinicalSupportHD@gsk.com

  Show 54 Study Locations
Sponsors and Collaborators
GlaxoSmithKline
Investigators
Study Director: GSK Clinical Trials GlaxoSmithKline
  More Information

No publications provided

Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT01480245     History of Changes
Other Study ID Numbers: 114349
Study First Received: November 23, 2011
Last Updated: May 16, 2013
Health Authority: Belgium: Local Medical Ethics Committee

Keywords provided by GlaxoSmithKline:
968
Duchenne
DMD
drisapersen

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on May 23, 2013