An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
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| ClinicalTrials.gov Identifier: NCT04256265 |
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Expanded Access Status :
Approved for marketing
First Posted : February 5, 2020
Last Update Posted : October 5, 2020
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Sponsor:
Genentech, Inc.
Information provided by (Responsible Party):
Genentech, Inc.
- Study Details
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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.
| Condition or disease | Intervention/treatment |
|---|---|
| Muscular Atrophy, Spinal | Drug: Risdiplam |
| Study Type : | Expanded Access |
| See clinical trials of the intervention/treatment in this expanded access record. | |
| Official Title: | An Expanded Access Program for Risdiplam in Patients With Type 1 or Type 2 Spinal Muscular Atrophy |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Spinal muscular atrophy
MedlinePlus related topics:
Spinal Muscular Atrophy
Drug Information
available for:
Risdiplam
Intervention Details:
- Drug: Risdiplam
Risdiplam will be administered orally once daily
Information from the National Library of Medicine
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| Ages Eligible for Study: | 2 Months and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
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.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04256265
Locations
Show 29 study locations
Sponsors and Collaborators
Genentech, Inc.
Investigators
| Study Director: | Clinical Trials | Hoffmann-La Roche |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Genentech, Inc. |
| ClinicalTrials.gov Identifier: | NCT04256265 |
| Other Study ID Numbers: |
AL41887 |
| First Posted: | February 5, 2020 Key Record Dates |
| Last Update Posted: | October 5, 2020 |
| Last Verified: | September 2020 |
Additional relevant MeSH terms:
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Muscular Atrophy Muscular Atrophy, Spinal Atrophy Pathological Conditions, Anatomical Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Spinal Cord Diseases |
Central Nervous System Diseases Motor Neuron Disease Neurodegenerative Diseases Neuromuscular Diseases Risdiplam Neuromuscular Agents Peripheral Nervous System Agents Physiological Effects of Drugs |