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Trial record 4 of 7 for:    GSK and Duchenne

Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

This study has been terminated.
(The study was formally terminated given that GSK is not submitting an application for regulatory approval for drisapersen in Duchenne Muscular Dystrophy.)
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline
ClinicalTrials.gov Identifier:
NCT01480245
First received: November 23, 2011
Last updated: March 21, 2017
Last verified: March 2017
  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
Intervention Model: Single Group Assignment
Masking: None (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:


Further study details as provided by GlaxoSmithKline:

Primary Outcome Measures:
  • Differences between the 6MWD at baseline and Week 104 [ Time Frame: 104 weeks ]

Secondary Outcome Measures:
  • Timed Function tests [ Time Frame: 104 weeks ]
  • Muscle strength [ Time Frame: 104 weeks ]
  • North Star Ambulatory Assessment Scores [ Time Frame: 104 weeks ]
  • Creatine kinase Serum concentrations [ Time Frame: 104 weeks ]
  • Pulmonary Function [ Time Frame: 104 weeks ]
  • Pediatric Quality of Life Neuromuscular module [ Time Frame: 104weeks ]
  • Clinician Global Impression of Improvement [ Time Frame: 104 weeks ]
  • Health Utilities Index [ Time Frame: 104 weeks ]
  • Frequency of accidental falls during 6 Minute Walk Distance test [ Time Frame: 104 weeks ]
  • Functional Outcomes Assessment [ Time Frame: 104 weeks ]
  • Time to major disease milestones [ Time Frame: 104 weeks ]

Enrollment: 233
Study Start Date: September 2011
Study Completion Date: March 2014
Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Continuous Dosing
GSK2402968 6mg/kg/week
Drug: GSK2402968
6mg/kg/week
Experimental: Intermittent Dosing
GSK2402968 6mg/kg/week
Drug: GSK2402968
6mg/kg/week
No Intervention: Natural History Observation
The objective of this arm will be to explore DMD disease progression in a naturalistic setting once discontinuing active treatment

  Eligibility

Ages Eligible for Study:   5 Years and older   (Child, Adult, Senior)
Sexes 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01480245

  Show 59 Study Locations
Sponsors and Collaborators
GlaxoSmithKline
Investigators
Study Director: GSK Clinical Trials GlaxoSmithKline
  More Information

Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT01480245     History of Changes
Other Study ID Numbers: 114349
Study First Received: November 23, 2011
Last Updated: March 21, 2017

Keywords provided by GlaxoSmithKline:
968
Duchenne
DMD
drisapersen

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

ClinicalTrials.gov processed this record on August 17, 2017