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

Status

Indicates the current recruitment status or the expanded access status.
Approved for marketing

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Genentech, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 3, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 3, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 5, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 5, 2020

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 30, 2020

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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