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

Muscular Atrophy, Spinal Clinical Trial

Official title:

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

Clinical Trial Details — Status: Approved for marketing

Administrative data

• NCT number: NCT04256265
• Other study ID #: AL41887
• Status: Approved for marketing

Study information

• Verified date: September 2020
• Source: Genentech, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

Recruitment information / eligibility

• Status: Approved for marketing
• Enrollment: 0
• Gender: All
• Age group: 2 Months and older

Eligibility

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.

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal

Intervention

Drug:
• Risdiplam
Risdiplam will be administered orally once daily

Locations

Country	Name	City	State
United States	Akron Childrens Hospital	Akron	Ohio
United States	Rare Disease Research, LLC	Atlanta	Georgia
United States	University of Colorado in Denver-Anschutz Medical Campus	Aurora	Colorado
United States	Massachusetts General Hospital; Neurology	Boston	Massachusetts
United States	University of Virginia Children's Hospital; Developmental	Charlottesville	Virginia
United States	Ann and Robert H. Lurie Children Hospital of Chicago	Chicago	Illinois
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Helen DeVos Children's Hospital at Spectrum Health	Grand Rapids	Michigan
United States	Indiana Hemophilia & Thrombosis center	Indianapolis	Indiana
United States	University of Iowa	Iowa City	Iowa
United States	University of Mississippi Medical Center; Neurology	Jackson	Michigan
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	Arkansas Children's Hospital; Pediatrics	Little Rock	Arkansas
United States	Children's Hospital Los Angeles	Los Angeles	California
United States	University of Louisville	Louisville	Kentucky
United States	University of Wisconsin American Family; Childrens Hospital	Madison	Wisconsin
United States	Childrens Hospital of Wisconsin	Milwaukee	Wisconsin
United States	Medical College of Wisconsin, Inc.	Milwaukee	Wisconsin
United States	Goryeb Children's Hospital	Morristown	New Jersey
United States	Northwell Hospital	New Hyde Park	New York
United States	NYU Langone	New York	New York
United States	Nemours Children's Hospital	Orlando	Florida
United States	Stanford University	Palo Alto	California
United States	Comprehensive NeuroBehavioral Institute	Plantation	Florida
United States	University of Rochester Medical Center	Rochester	New York
United States	St. Louis Children Hospital	Saint Louis	Missouri
United States	Gillette Spcl Children's Clin; Pediatric Endocrinology	Saint Paul	Minnesota
United States	Southern Illinois University, School of Medicine	Springfield	Illinois
United States	Wake Forest Baptist Medical Center	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Genentech, Inc.

Country where clinical trial is conducted

United States,