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

Study Overview
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Brief Summary
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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.

Official Title
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An Expanded Access Program for Risdiplam in Patients With Type 1 or Type 2 Spinal Muscular Atrophy

Conditions
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Muscular Atrophy, Spinal

Intervention / Treatment
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* Drug: Risdiplam


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 2 Months

Sexes Eligible for Study
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All

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Risdiplam|Risdiplam will be administered orally once daily|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |