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Trial record 12 of 29 for:    skipping | "muscular dystrophy, duchenne and becker types"

Study of Eteplirsen in DMD Patients (PROMOVI)

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ClinicalTrials.gov Identifier: NCT02255552
Recruitment Status : Active, not recruiting
First Posted : October 2, 2014
Last Update Posted : November 26, 2018
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics

Brief Summary:
The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy (DMD) Drug: eteplirsen Phase 3

Detailed Description:

This is an open-label, multi-center study to evaluate the efficacy and safety of eteplirsen in patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to exon 51 skipping (treated group), with a concurrent control arm of DMD patients not amenable to exon 51 skipping (untreated group). Following primary efficacy endpoints, dosing will continue to week 144 to evaluate the long term effects of eteplirsen.

Patients in the treated group will receive once weekly intravenous (IV) infusions of 30 mg/kg Eteplirsen for 96 weeks, followed by a safety extension (not to exceed 48 weeks). Patients in the untreated group will not receive treatment.

Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six minute walk test. Patients in the treated group will undergo a muscle biopsy at Baseline and a second muscle biopsy over the course of the study. Patients in the untreated group will not undergo muscle biopsy.

Safety, including adverse event monitoring and routine laboratory assessments, will be continuously monitored for all patients.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 110 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
Actual Study Start Date : November 17, 2014
Estimated Primary Completion Date : May 1, 2019
Estimated Study Completion Date : May 1, 2019


Arm Intervention/treatment
Experimental: Treated Group
Approximately 80 patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to treatment by exon 51 skipping will receive 30 mg/kg of eteplirsen weekly for 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Drug: eteplirsen
Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Other Names:
  • AVI-4658
  • EXONDYS 51®

No Intervention: Untreated Group
Approximately 30 DMD patients not amenable to exon 51 skipping will not receive eteplirsen.



Primary Outcome Measures :
  1. Change in 6-Minute Walk Test (6MWT) distance from baseline [ Time Frame: Change from Baseline to Week 96 ]

Secondary Outcome Measures :
  1. The percentage of dystrophin-positive fibers [ Time Frame: Change from Baseline ]
  2. Maximum inspiratory/expiratory pressure percent predicted (MIP/MEP % predicted) [ Time Frame: Change from Baseline ]


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Ages Eligible for Study:   7 Years to 16 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male 7-16 years old
  • Diagnosed with DMD, genotypically confirmed
  • Stable dose of corticosteroids for at least 24 weeks
  • Have intact right and left alternative upper muscle groups
  • Mean 6MWT greater than 300m (primary analysis on 300 to 450 meters)
  • Stable pulmonary and cardiac function: predicted FVC equal to or greater than 50% and LVEF of greater than 50%

Exclusion Criteria:

  • Previous treatment with drisapersen or any other RNA antisense agent or any gene therapy within the last 6 months
  • Participation in any other DMD interventional clinical study within 12 weeks
  • Major surgery within 3 months
  • Presence of other clinically significant illness
  • Major change in the physical therapy regime within 3 months

Other inclusion/exclusion criteria apply.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02255552


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Sponsors and Collaborators
Sarepta Therapeutics
Investigators
Study Director: Medical Director Sarepta Therapeutics, Inc.

Responsible Party: Sarepta Therapeutics
ClinicalTrials.gov Identifier: NCT02255552     History of Changes
Other Study ID Numbers: 4658-301
First Posted: October 2, 2014    Key Record Dates
Last Update Posted: November 26, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Sarepta Therapeutics:
DMD, Duchenne, Eteplirsen, dystrophy, dystrophin, exon 51

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