Muscular Atrophy, SpinalNCT04256265
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
A study testing Risdiplam in people with Muscular Atrophy, Spinal, run by Genentech, Inc. at 29 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 2 months and older
- Conditions the study is for
- Muscular Atrophy, Spinal
- What is being tested?
- Risdiplam
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 29 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 months and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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:
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- 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.
Who cannot join
- 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.
Trial locations and contacts
29 sites in 1 country.
29 sites in 1 country
United States29 sites
Akron Childrens Hospital
Akron, Ohio, 44308
Rare Disease Research, LLC
Atlanta, Georgia, 30318
University of Colorado in Denver-Anschutz Medical Campus
Aurora, Colorado, 80045
Massachusetts General Hospital; Neurology
Boston, Massachusetts, 02114
University of Virginia Children's Hospital; Developmental
Charlottesville, Virginia, 22903
Ann and Robert H. Lurie Children Hospital of Chicago
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, 49503
Indiana Hemophilia & Thrombosis center
Indianapolis, Indiana, 46260
University of Iowa
Iowa City, Iowa, 52242
University of Mississippi Medical Center; Neurology
Jackson, Michigan, 39216
University of Kansas Medical Center
Kansas City, Kansas, 66160
Arkansas Children's Hospital; Pediatrics
Little Rock, Arkansas, 72202
Children's Hospital Los Angeles
Los Angeles, California, 90010
University of Louisville
Louisville, Kentucky, 40202
University of Wisconsin American Family; Childrens Hospital
Madison, Wisconsin, 53792
Childrens Hospital of Wisconsin
Milwaukee, Wisconsin, 53201
Medical College of Wisconsin, Inc.
Milwaukee, Wisconsin, 53226-3596
Goryeb Children's Hospital
Morristown, New Jersey, 07960
Northwell Hospital
New Hyde Park, New York, 11042
NYU Langone
New York, New York, 10003
Nemours Children's Hospital
Orlando, Florida, 32827
Stanford University
Palo Alto, California, 94304
Comprehensive NeuroBehavioral Institute
Plantation, Florida, 33317
University of Rochester Medical Center
Rochester, New York, 14642
Gillette Spcl Children's Clin; Pediatric Endocrinology
Saint Paul, Minnesota, 55101
Southern Illinois University, School of Medicine
Springfield, Illinois, 62702
St. Louis Children Hospital
St Louis, Missouri, 63110
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157
About this study
Official title: 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.
- Conditions
- Muscular Atrophy, Spinal
- Being tested
- Risdiplam
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RisdiplamDrug
Risdiplam will be administered orally once daily
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Muscular Atrophy, SpinalStudies of the same condition, open and under way.
- More trials run by Genentech, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Genentech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04256265
- Sponsor's protocol ID
- AL41887
- Status last verified
- Sep 2020
- First submitted
- 3 Feb 2020
- Last updated
- 5 Oct 2020
- Results posted
- No
Publications
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.
PubMed 35567422
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Expanded Access Program for Risdiplam in Patients With Type 1 or Type 2 Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT04256265
- Acronym / sponsor ID
- AL41887
- Conditions
- Muscular Atrophy, Spinal
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Genentech, Inc.Industry (pharma, biotech, devices)
- First posted
- 5 Feb 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
Who may qualify
- Ages
- 2 months and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- 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.
- Confirmed diagnosis of 5q-autosomal recessive SMA.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RisdiplamRisdiplam will be administered orally once daily | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2020.
- Derived from this study: PubMed 35567422Kwon 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
29 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Risdiplam in people with Muscular Atrophy, Spinal, run by Genentech, Inc. at 29 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04256265, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Oct 2020.