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Confirmatory Study of Eteplirsen in DMD Patients (PROMOVI)

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02255552
First Posted: October 2, 2014
Last Update Posted: October 3, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Sarepta Therapeutics
September 25, 2014
October 2, 2014
October 3, 2017
September 2014
January 2019   (Final data collection date for primary outcome measure)
Change in 6-Minute Walk Test (6MWT) distance from baseline [ Time Frame: Baseline, Weeks 12, 24, 36, 48 ]
Same as current
Complete list of historical versions of study NCT02255552 on ClinicalTrials.gov Archive Site
  • The percentage of dystrophin-positive fibers [ Time Frame: Baseline, Weeks 24, 48, 72, 96 ]
  • Maximum inspiratory/expiratory pressure percent predicted (MIP/MEP % predicted) [ Time Frame: Baseline,Weeks 12, 24, 36, 48 ]
  • The percentage of dystrophin-positive fibers [ Time Frame: Baseline, Weeks 24/48/TBD ]
  • Maximum inspiratory/expiratory pressure percent predicted (MIP/MEP % predicted) [ Time Frame: Baseline,Weeks 12, 24, 36, 48 ]
Evaluate the long-term effects of eteplirsen up to 96 weeks [ Time Frame: Week 1-96 ]
Not Provided
 
Confirmatory Study of Eteplirsen in DMD Patients
An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy

The main objective of this study is to provide confirmatory 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).

Sites are currently being initiated into the study. Initiation of approximately 39 planned sites in the United States is expected to be completed by June 2016. The initiated sites can be found in the "Contacts and Locations" section of this posting in addition to a listing of the city and states of sites the investigators are working to initiate. This information will be updated on a rolling basis as additional sites are initiated.

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.

The sponsor is working to initiate approximately 39 sites across the United States. Sites will vary in the following functions:

  1. Local Site (N=39) - Enrolls patients and is the primary contact point for their patients. Sites will perform all protocol activities (including dosing and laboratory assessments), except for functional assessments and biopsies.
  2. Hub Site (N=14) - Performs functional (physical) assessments at specified times per protocol.
  3. Surgical Site (N=2) - Performs muscle biopsies for the Treated group.
Interventional
Phase 3
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Duchenne Muscular Dystrophy (DMD)
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®
  • 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).
    Intervention: Drug: eteplirsen
  • No Intervention: Untreated Group
    Approximately 80 DMD patients not amenable to exon 51 skipping will not receive eteplirsen.
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
160
May 2019
January 2019   (Final data collection date for primary outcome measure)

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.

Sexes Eligible for Study: Male
7 Years to 16 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT02255552
4658-301
No
Not Provided
Plan to Share IPD: No
Sarepta Therapeutics
Sarepta Therapeutics
Not Provided
Study Chair: Chris Mix, MD Sarepta Therapeutics
Study Chair: Catherine Stehman-Breen, MD, MS Sarepta Therapeutics
Sarepta Therapeutics
October 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP