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

September 30, 2020 updated by: Genentech, Inc.

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

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.

Study Overview

Status

Approved for marketing

Conditions

Intervention / Treatment

Study Type

Expanded Access

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital; Pediatrics
    • California
      • Los Angeles, California, United States, 90010
        • Children's Hospital Los Angeles
      • Palo Alto, California, United States, 94304
        • Stanford University
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado in Denver-Anschutz Medical Campus
    • Florida
      • Orlando, Florida, United States, 32827
        • Nemours Children's Hospital
      • Plantation, Florida, United States, 33317
        • Comprehensive NeuroBehavioral Institute
    • Georgia
      • Atlanta, Georgia, United States, 30318
        • Rare Disease Research, LLC
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann and Robert H. Lurie Children Hospital of Chicago
      • Springfield, Illinois, United States, 62702
        • Southern Illinois University, School of Medicine
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Indiana Hemophilia & Thrombosis Center
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Kentucky
      • Louisville, Kentucky, United States, 40202
        • University of Louisville
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital; Neurology
    • Michigan
      • Grand Rapids, Michigan, United States, 49503
        • Helen DeVos Children's Hospital at Spectrum Health
      • Jackson, Michigan, United States, 39216
        • University of Mississippi Medical Center; Neurology
    • Minnesota
      • Saint Paul, Minnesota, United States, 55101
        • Gillette Spcl Children's Clin; Pediatric Endocrinology
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • St. Louis Children Hospital
    • New Jersey
      • Morristown, New Jersey, United States, 07960
        • Goryeb Children's Hospital
    • New York
      • New Hyde Park, New York, United States, 11042
        • Northwell Hospital
      • New York, New York, United States, 10003
        • NYU Langone
      • Rochester, New York, United States, 14642
        • University of Rochester Medical Center
    • North Carolina
      • Winston-Salem, North Carolina, United States, 27157
        • Wake Forest Baptist Medical Center
    • Ohio
      • Akron, Ohio, United States, 44308
        • Akron Childrens Hospital
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • University of Virginia Children's Hospital; Developmental
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • University of Wisconsin American Family; Childrens Hospital
      • Milwaukee, Wisconsin, United States, 53201
        • Childrens Hospital of Wisconsin
      • Milwaukee, Wisconsin, United States, 53226-3596
        • Medical College of Wisconsin, Inc.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 months and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genentech, Inc.

Investigators

  • Study Director: Clinical Trials, Hoffmann-La Roche

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

February 3, 2020

First Submitted That Met QC Criteria

February 3, 2020

First Posted (Actual)

February 5, 2020

Study Record Updates

Last Update Posted (Actual)

October 5, 2020

Last Update Submitted That Met QC Criteria

September 30, 2020

Last Verified

September 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AL41887

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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