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Clinical Trial Details — Status: Approved for marketing

Administrative data

NCT number NCT04256265
Other study ID # AL41887
Secondary ID
Status Approved for marketing
Phase
First received
Last updated

Study information

Verified date September 2020
Source Genentech, Inc.
Contact n/a
Is FDA regulated No
Health authority
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
Est. completion date
Est. primary completion date
Accepts healthy volunteers
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.

Study Design


Related Conditions & MeSH terms


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, 

See also
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Active, not recruiting NCT05618379 - Adult Spinal Muscular Atrophy (SMA) China Registry
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