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Confirmatory Study of Eteplirsen in DMD Patients (PROMOVI)

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2016 by Sarepta Therapeutics
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics
ClinicalTrials.gov Identifier:
NCT02255552
First received: September 25, 2014
Last updated: August 16, 2016
Last verified: August 2016
  Purpose

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.

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.


Condition Intervention Phase
Duchenne Muscular Dystrophy (DMD)
Drug: eteplirsen
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-Label, Multi-Center, 48-Week Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy

Resource links provided by NLM:


Further study details as provided by Sarepta Therapeutics:

Primary Outcome Measures:
  • Change in 6-Minute Walk Test (6MWT) distance from baseline [ Time Frame: Baseline, Weeks 12, 24, 36, 48 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • The percentage of dystrophin-positive fibers [ Time Frame: Baseline, Weeks 24/48/72/96 ] [ Designated as safety issue: No ]
  • Maximum inspiratory/expiratory pressure percent predicted (MIP/MEP % predicted) [ Time Frame: Baseline, Weeks 12, 24, 36, 48 ] [ Designated as safety issue: No ]

Other Outcome Measures:
  • Evaluate the long-term effects of eteplirsen up to 96 weeks [ Time Frame: Week 1-96 ] [ Designated as safety issue: No ]

Estimated Enrollment: 160
Study Start Date: September 2014
Estimated Study Completion Date: May 2019
Estimated Primary Completion Date: January 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
Drug: eteplirsen
Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks.
Other Name: AVI-4658
No Intervention: Untreated Group
Approximately 80 DMD patients not amenable to exon 51 skipping will not receive eteplirsen.

Detailed Description:

This is an open-label, multi-center, 48-week 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 at week 48, dosing will continue to week 96 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. 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.
  Eligibility

Ages Eligible for Study:   7 Years to 16 Years   (Child)
Genders 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 6 months
  • 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.

  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: NCT02255552

Contacts
Contact: Kara Boniface trialinfo@sarepta.com

  Show 39 Study Locations
Sponsors and Collaborators
Sarepta Therapeutics
Investigators
Study Director: Petra Duda, MD, PhD Sarepta Therapeutics
Study Director: Edward M. Kaye, MD Sarepta Therapeutics
  More Information

Additional Information:
Responsible Party: Sarepta Therapeutics
ClinicalTrials.gov Identifier: NCT02255552     History of Changes
Other Study ID Numbers: 4658-301 
Study First Received: September 25, 2014
Last Updated: August 16, 2016
Health Authority: United States: Food and Drug Administration
Individual Participant Data  
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

ClinicalTrials.gov processed this record on December 06, 2016