Clinical trial NCT04256265: An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Official title: An Expanded Access Program for Risdiplam in Patients With Type 1 or Type 2 Spinal Muscular Atrophy
Study type: EXPANDED_ACCESS
Overall status: APPROVED_FOR_MARKETING
Conditions: Muscular Atrophy, Spinal
Lead sponsor: Genentech, Inc.
Sponsor class: INDUSTRY
First posted: 2020-02-05
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT

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.


Eligibility 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital; Pediatrics
state: Arkansas
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Aurora
country: United States
facility: University of Colorado in Denver-Anschutz Medical Campus
state: Colorado
city: Orlando
country: United States
facility: Nemours Children's Hospital
state: Florida
city: Plantation
country: United States
facility: Comprehensive NeuroBehavioral Institute
state: Florida
city: Atlanta
country: United States
facility: Rare Disease Research, LLC
state: Georgia
city: Chicago
country: United States
facility: Ann and Robert H. Lurie Children Hospital of Chicago
state: Illinois
city: Springfield
country: United States
facility: Southern Illinois University, School of Medicine
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Hemophilia & Thrombosis center
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Louisville
country: United States
facility: University of Louisville
state: Kentucky
city: Boston
country: United States
facility: Massachusetts General Hospital; Neurology
state: Massachusetts
city: Grand Rapids
country: United States
facility: Helen DeVos Children's Hospital at Spectrum Health
state: Michigan
city: Jackson
country: United States
facility: University of Mississippi Medical Center; Neurology
state: Michigan
city: Saint Paul
country: United States
facility: Gillette Spcl Children's Clin; Pediatric Endocrinology
state: Minnesota
city: Saint Louis
country: United States
facility: St. Louis Children Hospital
state: Missouri
city: Morristown
country: United States
facility: Goryeb Children's Hospital
state: New Jersey
city: New Hyde Park
country: United States
facility: Northwell Hospital
state: New York
city: New York
country: United States
facility: NYU Langone
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Winston-Salem
country: United States
facility: Wake Forest Baptist Medical Center
state: North Carolina
city: Akron
country: United States
facility: Akron Childrens Hospital
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Charlottesville
country: United States
facility: University of Virginia Children's Hospital; Developmental
state: Virginia
city: Madison
country: United States
facility: University of Wisconsin American Family; Childrens Hospital
state: Wisconsin
city: Milwaukee
country: United States
facility: Childrens Hospital of Wisconsin
state: Wisconsin
city: Milwaukee
country: United States
facility: Medical College of Wisconsin, Inc.
state: Wisconsin
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References:
citation: Kwon JM, Arya K, Kuntz N, Phan HC, Sieburg C, Swoboda KJ, Veerapandiyan A, Assman B, Bader-Weder S, Dickendesher TL, Hansen J, Lin H, Yan Y, Rao VK; US Expanded Access Program Working Group. An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy. Ann Clin Transl Neurol. 2022 Jun;9(6):810-818. doi: 10.1002/acn3.51560. Epub 2022 May 14.