Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

GlaxoSmithKline (GSK)

Status and phase: Terminated; Phase 3

Conditions: Muscular Dystrophies

Treatments: Drug: GSK2402968

Study type: Interventional

Funder types: Industry

Identifiers

NCT01480245

114349

Details and patient eligibility

About

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.

Enrollment: 233 patients

Sex: Male

Ages: 5+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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233 participants in 3 patient groups

Continuous Dosing

Experimental group

Description:

GSK2402968 6mg/kg/week

Treatment:

Drug: GSK2402968

Intermittent Dosing

Experimental group

Description:

GSK2402968 6mg/kg/week

Treatment:

Drug: GSK2402968

Natural History Observation

No Intervention group

Description:

The objective of this arm will be to explore DMD disease progression in a naturalistic setting once discontinuing active treatment