Last updated: 03/05/2020 18:10:09
This study has been divested to Prosensa, GSK will not produce a result
An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen (GSK2402968) in US subjects with Duchenne Muscular Dystrophy
GSK study ID
115501
Clinicaltrials.gov ID
EudraCT ID
Not applicable
EU CT Number
Not applicable
Trial status
No longer a GSK study
No longer a GSK study
Trial overview
Official title: An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen (GSK2402968) in US subjects with Duchenne Muscular Dystrophy
Trial description: This is a phase III, multicenter, open-label, uncontrolled extension study. The Purpose of this study is to evaluate the effects of long-term administration of drisapersen in US male subjects with Duchenne Muscular Dystrophy (DMD), who have previously participated in one of the following eligible studies of drisapersen: DMD114876, DMD114044 and DMD114349. This study does not have a minimum duration of participation. The study participation time of the subjects will vary depending on when they enter from one of the eligible studies, and will be permitted to continue the study until such a time that they withdraw based on protocol-defined criteria or GlaxoSmithKline (GSK) stops the study. Subjects will receive drisapersen 6 milligrams (mg)/kilogram (kg) as Subcutaneous (SC) injection(s) once a week (wk), continuously throughout their duration of participation. For subjects who cannot continue on 6 mg/kg/wk for safety and/or tolerability reasons, but who may benefit from drisapersen, an alternate intermittent dosing will be given as a regimen of 6 mg/kg/wk for 8 weeks followed by 4 weeks off treatment. Subjects withdrawing from this study but who do not withdraw consent, will be followed up for safety and progress as clinically indicated, for at least 20 weeks after the last dose of study treatment, after which a follow-up telephone call will be conducted. If subjects withdraw, they will not be replaced.
Primary purpose:
Treatment
Trial design:
Single Group Assignment
Masking:
None (Open Label)
Allocation:
Non-randomized
Primary outcomes:
Body temperature measurements to assess the safety and tolerability
Timeframe: Up to 48 weeks
Systolic and diastolic blood pressure measurements to assess the safety and tolerability
Timeframe: Up to 48 weeks
Pulse rate and respiratory rate measurements to assess the safety and tolerability
Timeframe: Up to 48 weeks
Incidence and severity of Adverse Events (AEs)
Timeframe: Up to 48 weeks
Laboratory tests to assess the safety and tolerability
Timeframe: Up to 48 weeks
12-Lead Electrocardiogram (ECG) measurements to assess the safety and tolerability
Timeframe: Up to 48 weeks
Echocardiogram measurements to assess the safety and tolerability
Timeframe: Up to 48 weeks
Secondary outcomes:
Pulmonary function assessment
Timeframe: Up to 48 weeks
Functional Outcomes assessment
Timeframe: Up to 48 weeks
Muscle function assessment using 6-minute walking distance (6MWD) test
Timeframe: Up to 48 weeks
Frequency of accidental falls during 6MWD
Timeframe: Up to 48 weeks
North Star Ambulatory Assessment (NSAA)
Timeframe: Up to 48 weeks
Plasma concentration of drisapersen - intermittent dosing regimen
Timeframe: Weeks 0, 7, 12, 19, 24, 31, 36 and 43
Time to major disease milestones
Timeframe: Up to 48 weeks
Plasma concentration of drisapersen - continuous dosing regimen
Timeframe: Weeks 0, 4, 8, 12, 16, 20, 24, 32, 40 and 48
Interventions:
Enrollment:
23
Primary completion date:
Not applicable
Observational study model:
Not applicable
Time perspective:
Not applicable
Clinical publications:
Not applicable
Medical condition
Muscular Dystrophies
Product
drisapersen
Collaborators
Not applicable
Study date(s)
May 2013 to July 2014
Type
Interventional
Phase
2/3
Participation criteria
Sex
Male
Age
5+ years
Accepts healthy volunteers
none
- Participation in an eligible drisapersen study as follows:
- (A) Prior DMD114876 subjects: Subjects who completed both the 24 week double-blind treatment and 24 week post-treatment phases in study DMD114876 OR Subjects who withdrew from the treatment portion of study DMD114876 due to meeting laboratory safety stopping criteria may be eligible to enrol in the extension study if: the laboratory parameters that led to stopping have resolved; the principal investigator (PI) considers the benefit of further treatment with drisapersen outweighs the risk to the individual subject; and following consultation with the Medical Monitor
- Subject had a serious adverse experience or who met safety stopping criteria that remains unresolved from studies DMD115501, DMD114044, or DMD114349, which in the opinion of the investigator could have been attributable to study medication, and which is ongoing. Once resolved, subject may be eligible to enrol following PI consultation with the Medical Monitor
- Use of anticoagulants, antithrombotics or antiplatelet agents, or previous treatment with investigational drugs except for drisapersen, within 28 days of the first administration of study medication
Inclusion and exclusion criteria
Inclusion criteria:
- Participation in an eligible drisapersen study as follows: (A) Prior DMD114876 subjects: Subjects who completed both the 24 week double-blind treatment and 24 week post-treatment phases in study DMD114876 OR Subjects who withdrew from the treatment portion of study DMD114876 due to meeting laboratory safety stopping criteria may be eligible to enrol in the extension study if: the laboratory parameters that led to stopping have resolved; the principal investigator (PI) considers the benefit of further treatment with drisapersen outweighs the risk to the individual subject; and following consultation with the Medical Monitor (B) Prior DMD114044 Subjects: US citizens who completed study DMD114044 in another country and who want to return to the US to participate in study DMD115501, upon agreement by a DMD115501 Investigator OR US citizens who participated in DMD114044 but who had to withdraw from the study due to meeting laboratory safety stopping criteria may be eligible to enrol in DMD115501 if: the laboratory parameters that led to stopping have resolved; the PI considers the benefit of further treatment with drisapersen outweighs the risk to the individual subject; and following consultation with the Medical Monitor and upon agreement by a DMD115501 investigator (C) Prior DMD114349 Subjects: US citizens who participated in and completed study DMD114044 in another country and who entered into the ongoing open-label extension study DMD114349 in a country outside the US who wish to withdraw from DMD114349 and return to the US to participate in study DMD115501, upon agreement by a DMD115501 investigator. Subjects are required to withdraw from DMD114349 to participate in this study
- Continued use of glucocorticoids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on steroids for the duration of the study. Changes to or cessation of glucocorticoids will be at the discretion of the PI in consultation with the subject/parent and the Medical Monitor
- Willing and able to comply with all protocol requirements and procedures (with the exception of those assessments requiring a subject to be ambulant, for those subjects who have lost ambulation)
- Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations)
Exclusion criteria:
- Subject had a serious adverse experience or who met safety stopping criteria that remains unresolved from studies DMD115501, DMD114044, or DMD114349, which in the opinion of the investigator could have been attributable to study medication, and which is ongoing. Once resolved, subject may be eligible to enrol following PI consultation with the Medical Monitor
- Use of anticoagulants, antithrombotics or antiplatelet agents, or previous treatment with investigational drugs except for drisapersen, within 28 days of the first administration of study medication
- Current or anticipated participation in any other investigational clinical studies
- History of significant medical disorder which may confound the interpretation of either efficacy or safety data (e.g. current or history of renal or liver disease/impairment, history of inflammatory illness)
- 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
Trial location(s)
This study does not involve prospective enrollment of participants.
Study documents
No study documents available.
Results overview
Study Results yet to be posted
Recruitment status
No longer a GSK study
Actual primary completion date
Not applicable
Actual study completion date
Not applicable
Plain language summaries
Plain language summaries of clinical trial results for Phase 2-4 clinical trials that were initiated on or after January 2022 will be posted by GSK within one year following study completion.
Additional information about the trial
Additional information
Not applicable
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