An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
1 other identifier
expanded_access
N/A
1 country
29
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.
Trial Health
Trial Health Score
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29 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 3, 2020
CompletedFirst Posted
Study publicly available on registry
February 5, 2020
CompletedOctober 5, 2020
September 1, 2020
February 3, 2020
September 30, 2020
Conditions
Interventions
Risdiplam will be administered orally once daily
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Genentech, Inc.lead
Study Sites (29)
Arkansas Children's Hospital; Pediatrics
Little Rock, Arkansas, 72202, United States
Children's Hospital Los Angeles
Los Angeles, California, 90010, United States
Stanford University
Palo Alto, California, 94304, United States
University of Colorado in Denver-Anschutz Medical Campus
Aurora, Colorado, 80045, United States
Nemours Children's Hospital
Orlando, Florida, 32827, United States
Comprehensive NeuroBehavioral Institute
Plantation, Florida, 33317, United States
Rare Disease Research, LLC
Atlanta, Georgia, 30318, United States
Ann and Robert H. Lurie Children Hospital of Chicago
Chicago, Illinois, 60611, United States
Southern Illinois University, School of Medicine
Springfield, Illinois, 62702, United States
Indiana Hemophilia & Thrombosis center
Indianapolis, Indiana, 46260, United States
University of Iowa
Iowa City, Iowa, 52242, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
University of Louisville
Louisville, Kentucky, 40202, United States
Massachusetts General Hospital; Neurology
Boston, Massachusetts, 02114, United States
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, 49503, United States
University of Mississippi Medical Center; Neurology
Jackson, Michigan, 39216, United States
Gillette Spcl Children's Clin; Pediatric Endocrinology
Saint Paul, Minnesota, 55101, United States
St. Louis Children Hospital
St Louis, Missouri, 63110, United States
Goryeb Children's Hospital
Morristown, New Jersey, 07960, United States
Northwell Hospital
New Hyde Park, New York, 11042, United States
NYU Langone
New York, New York, 10003, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157, United States
Akron Childrens Hospital
Akron, Ohio, 44308, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
University of Virginia Children's Hospital; Developmental
Charlottesville, Virginia, 22903, United States
University of Wisconsin American Family; Childrens Hospital
Madison, Wisconsin, 53792, United States
Childrens Hospital of Wisconsin
Milwaukee, Wisconsin, 53201, United States
Medical College of Wisconsin, Inc.
Milwaukee, Wisconsin, 53226-3596, United States
Related Publications (1)
Kwon JM, Arya K, Kuntz N, Phan HC, Sieburg C, Swoboda KJ, Veerapandiyan A, Assman B, Bader-Weder S, Dickendesher TL, Hansen J, Lin H, Yan Y, Rao VK; US Expanded Access Program Working Group. An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy. Ann Clin Transl Neurol. 2022 Jun;9(6):810-818. doi: 10.1002/acn3.51560. Epub 2022 May 14.
PMID: 35567422DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Clinical Trials
Hoffmann-La Roche
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 3, 2020
First Posted
February 5, 2020
Last Updated
October 5, 2020
Record last verified: 2020-09