An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

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Conditions studied: Muscular Atrophy, Spinal

In brief

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.

Key facts

Study ID

NCT04256265

Run by

Genentech, Inc.

Last updated by the study team

2020-10-05

Who can join

Age: 0 and older. Sex: any. Healthy volunteers: not accepted.

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.

Where it is running

• Arkansas Children's Hospital; Pediatrics — Little Rock, Arkansas, United States
• Children's Hospital Los Angeles — Los Angeles, California, United States
• Stanford University — Palo Alto, California, United States
• University of Colorado in Denver-Anschutz Medical Campus — Aurora, Colorado, United States
• Nemours Children's Hospital — Orlando, Florida, United States
• Comprehensive NeuroBehavioral Institute — Plantation, Florida, United States
• Rare Disease Research, LLC — Atlanta, Georgia, United States
• Ann and Robert H. Lurie Children Hospital of Chicago — Chicago, Illinois, United States
• Southern Illinois University, School of Medicine — Springfield, Illinois, United States
• Indiana Hemophilia & Thrombosis center — Indianapolis, Indiana, United States
• University of Iowa — Iowa City, Iowa, United States
• University of Kansas Medical Center — Kansas City, Kansas, 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 Mississippi Medical Center; Neurology — Jackson, Michigan, United States
• Gillette Spcl Children's Clin; Pediatric Endocrinology — Saint Paul, Minnesota, United States
• St. Louis Children Hospital — St Louis, Missouri, United States
• Goryeb Children's Hospital — Morristown, New Jersey, United States
• Northwell Hospital — New Hyde Park, New York, United States
• NYU Langone — New York, New York, United States
• University of Rochester Medical Center — Rochester, New York, United States
• Wake Forest Baptist Medical Center — Winston-Salem, North Carolina, United States
• Akron Childrens Hospital — Akron, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States

Full record on ClinicalTrials.gov

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