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Trial record 2 of 2 for:    eap | sma

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

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ClinicalTrials.gov Identifier: NCT04256265
Expanded Access Status : Available
First Posted : February 5, 2020
Last Update Posted : June 5, 2020
Sponsor:
Information provided by (Responsible Party):
Genentech, Inc.

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

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



Intervention Details:
  • Drug: Risdiplam
    Risdiplam will be administered orally once daily

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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


Contacts
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Contact: Reference Study ID Number: AL41887 www.roche.com/about_roche/roche_worldwide.htm (844) 287-3783 (CU-REQUEST) global-roche-genentech-trials@gene.com

Locations
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United States, Arkansas
Arkansas Children's Hospital; Pediatrics Available
Little Rock, Arkansas, United States, 72202
United States, California
Children's Hospital Los Angeles Available
Los Angeles, California, United States, 90010
United States, Illinois
Ann and Robert H. Lurie Children Hospital of Chicago Available
Chicago, Illinois, United States, 60611
United States, Minnesota
Gillette Spcl Children's Clin; Pediatric Endocrinology Available
Saint Paul, Minnesota, United States, 55101
United States, Ohio
Akron Childrens Hospital Available
Akron, Ohio, United States, 44308
Nationwide Children's Hospital Available
Columbus, Ohio, United States, 43205
United States, Wisconsin
University of Wisconsin American Family; Childrens Hospital Available
Madison, Wisconsin, United States, 53792
Sponsors and Collaborators
Genentech, Inc.
Investigators
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Study Director: Clinical Trials Hoffmann-La Roche
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Responsible Party: Genentech, Inc.
ClinicalTrials.gov Identifier: NCT04256265    
Other Study ID Numbers: AL41887
First Posted: February 5, 2020    Key Record Dates
Last Update Posted: June 5, 2020
Last Verified: June 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
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases