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

Muscular Atrophy, Spinal

Drug: Risdiplam
Risdiplam will be administered orally once daily

Eligibility Criteria

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.

Eligibility Gender
Eligibility Age
Minimum: 2 Months
United States

Trial Information Support Line
South San Francisco, California, 94080-4990



Reference Study ID Number: AL41887
(844) 287-3783 (CU-REQUEST)

Clinical Trials
Study Director
Hoffmann-La Roche

Genentech, Inc.
NCT Number
MeSH Terms
Muscular Atrophy
Muscular Atrophy, Spinal