Study of Eteplirsen in DMD Patients
PROMOVI
An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
1 other identifier
interventional
109
1 country
37
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Nov 2014
Longer than P75 for phase_3
37 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 25, 2014
CompletedFirst Posted
Study publicly available on registry
October 2, 2014
CompletedStudy Start
First participant enrolled
November 17, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 14, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
June 14, 2019
CompletedResults Posted
Study results publicly available
July 1, 2020
CompletedJanuary 25, 2021
December 1, 2020
4.6 years
September 25, 2014
June 12, 2020
December 31, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change From Baseline in the 6 Minute Walk Test (6MWT) Distance at Week 96
6MWT was performed by standardized procedures for all participants. Participants were asked to walk a set course of 25 meters for 6 minutes (timed), and the distance walked (in meters) was recorded. Change from baseline in 6MWT distance at Week 96 was reported.
Baseline, Week 96
Secondary Outcomes (6)
Change From Baseline in Dystrophin Protein Levels Determined by Western Blot at Week 96
Baseline, Week 96
Number of Participants Having Ability to Rise Independently From the Floor Determined Based on North Star Ambulatory Assessment (NSAA) at Week 96
Week 96
Number of Participants Who Lost Ambulation (LOA) by Week 96
Up to Week 96
Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Weeks 96
Baseline, Week 96
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Scores at Week 96
Baseline, Week 96
- +1 more secondary outcomes
Study Arms (2)
Treated Group
EXPERIMENTALApproximately 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).
Untreated Group
NO INTERVENTIONApproximately 30 DMD patients not amenable to exon 51 skipping will not receive eteplirsen.
Interventions
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).
Eligibility Criteria
You may qualify if:
- 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%
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (37)
Neuromuscular Research Center
Phoenix, Arizona, 85028, United States
David Geffen School of Medicine at UCLA
Los Angeles, California, 90095, United States
University of California, Davis Medical Center
Sacramento, California, 95817, United States
Rady Children's Hospital, U.C. San Diego
San Diego, California, 92130, United States
Stanford University School of Medicine/Medical Center
Stanford, California, 94305, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Connecticut Children's Medical Center
Hartford, Connecticut, 06106, United States
Children's National Health System
Washington D.C., District of Columbia, 20010, United States
The University of Florida, Powell Gene Therapy Center
Gainesville, Florida, 32610, United States
NW FL Clinical Research Group, LLC
Gulf Breeze, Florida, 32561, United States
Nemours Children's Hospital
Orlando, Florida, 32827, United States
Rare Disease Research Center
Atlanta, Georgia, 30318, United States
Emory University
Atlanta, Georgia, 30324, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
University of Iowa Children's Hospital
Iowa City, Iowa, 52242, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Children's Hospital Boston
Boston, Massachusetts, 02115, United States
Children's Hospital of Michigan
Detroit, Michigan, 48201, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, 03756, United States
Columbia University Medical Center
New York, New York, 10032, United States
University of Rochester Clinical Research Center
Rochester, New York, 14642, United States
Levine Childrens Hospital, Carolinas Medical Center
Charlotte, North Carolina, 28207, United States
Cincinnati Children's Hospital Medical Center (CCHMC)
Cincinnati, Ohio, 45229, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Shriners Hospital for Children
Portland, Oregon, 97239, United States
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
The University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
University of Utah
Salt Lake City, Utah, 84132, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Related Publications (1)
Brogna C, Coratti G, Pane M, Ricotti V, Messina S, D'Amico A, Bruno C, Vita G, Berardinelli A, Mazzone E, Magri F, Ricci F, Mongini T, Battini R, Bello L, Pegoraro E, Baranello G, Previtali SC, Politano L, Comi GP, Sansone VA, Donati A, Bertini E, Muntoni F, Goemans N, Mercuri E; on behalf on the International DMD group. Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53. PLoS One. 2019 Jun 25;14(6):e0218683. doi: 10.1371/journal.pone.0218683. eCollection 2019.
PMID: 31237898BACKGROUND
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Director
- Organization
- Sarepta Therapeutics, Inc.
Study Officials
- STUDY DIRECTOR
Medical Director
Sarepta Therapeutics, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 25, 2014
First Posted
October 2, 2014
Study Start
November 17, 2014
Primary Completion
June 14, 2019
Study Completion
June 14, 2019
Last Updated
January 25, 2021
Results First Posted
July 1, 2020
Record last verified: 2020-12
Data Sharing
- IPD Sharing
- Will not share