An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
- Conditions
- Muscular Atrophy, Spinal
- Registration Number
- NCT04256265
- Lead Sponsor
- Genentech, Inc.
- Brief Summary
This expanded access program (EAP) will provide access to risdiplam for eligible participants with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in the United States for the indication of SMA.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- APPROVED_FOR_MARKETING
- Sex
- All
- Target Recruitment
- Not specified
All Participants:
- Not eligible for treatment with currently approved treatments for SMA, or cannot continue treatment with currently approved medications as documented by the treating physician, or in the treating physician's judgment, the participant is at risk of lack/loss of treatment efficacy of the current therapy.
- The participant does not qualify for and has no access to SMA treatment in the context of an ongoing clinical trial.
- Adequately recovered from any acute illness at the time of screening, and considered clinically well enough to participate, in the opinion of the treating physician.
- Participants with retinopathy of prematurity should have evidence of stable disease.
Type 1 SMA Participants:
- Confirmed diagnosis of 5q-autosomal recessive SMA.
Type 2 SMA Participants:
- Confirmed diagnosis of 5q-autosomal recessive SMA.
- Negative blood pregnancy test at screening (all women of childbearing potential, including those who have had a tubal ligation), and agreement to comply with measures to prevent pregnancy and restrictions on egg and sperm donation.
- Males with female partners of reproductive potential must agree to use highly effective contraception during therapy, and for at least 4 months after treatment discontinuation.
- Inability to meet program requirements.
- Concomitant or previous participation in any investigational drug or device study within 90 days prior to screening or 5 half-lives, whichever is longer.
- Administration of other SMN-2 targeting therapy within 120 days of starting risdiplam therapy.
- Administration of SMA gene therapy within the last 3 months (12 weeks) of receiving risdiplam therapy.
- Any serious medical condition, treatment, or abnormality in clinical laboratory tests that, in the treating physician's judgment, precludes the participant's safe participation in the program.
- Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to risdiplam or to the constituents of its formulation.
- Suspicion of illicit drug or alcohol abuse, in the treating physician's judgment.
- Any prior use of an inhibitor or inducer of flavin-containing monooxygenases 1 (FMO1) or flavin-containing monooxygenases 3 (FMO3) taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing.
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (29)
Arkansas Children's Hospital; Pediatrics
🇺🇸Little Rock, Arkansas, United States
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Ann and Robert H. Lurie Children Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Comprehensive NeuroBehavioral Institute
🇺🇸Plantation, Florida, United States
Stanford University
🇺🇸Palo Alto, California, United States
Indiana Hemophilia & Thrombosis center
🇺🇸Indianapolis, Indiana, United States
University of Louisville
🇺🇸Louisville, Kentucky, United States
Massachusetts General Hospital; Neurology
🇺🇸Boston, Massachusetts, United States
Helen DeVos Children's Hospital at Spectrum Health
🇺🇸Grand Rapids, Michigan, United States
University of Rochester Medical Center
🇺🇸Rochester, New York, United States
Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
NYU Langone
🇺🇸New York, New York, United States
Akron Childrens Hospital
🇺🇸Akron, Ohio, United States
University of Colorado in Denver-Anschutz Medical Campus
🇺🇸Aurora, Colorado, United States
Nemours Children's Hospital
🇺🇸Orlando, Florida, United States
Gillette Spcl Children's Clin; Pediatric Endocrinology
🇺🇸Saint Paul, Minnesota, United States
Southern Illinois University, School of Medicine
🇺🇸Springfield, Illinois, United States
Goryeb Children's Hospital
🇺🇸Morristown, New Jersey, United States
Northwell Hospital
🇺🇸New Hyde Park, New York, United States
St. Louis Children Hospital
🇺🇸Saint Louis, Missouri, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
University of Mississippi Medical Center; Neurology
🇺🇸Jackson, Michigan, United States
Wake Forest Baptist Medical Center
🇺🇸Winston-Salem, North Carolina, United States
University of Virginia Children's Hospital; Developmental
🇺🇸Charlottesville, Virginia, United States
University of Wisconsin American Family; Childrens Hospital
🇺🇸Madison, Wisconsin, United States
Childrens Hospital of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Medical College of Wisconsin, Inc.
🇺🇸Milwaukee, Wisconsin, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States