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Risdiplam for Spinal Muscular Atrophy
(Jewelfish Trial)

Recruiting at 38 trial locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Hoffmann-La Roche

No Placebo Group

Prior Safety Data

Approved in 9 Jurisdictions

Trial Summary

What is the purpose of this trial?

This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.

Research Team

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Eligibility Criteria

This trial is for adults, children, and infants with Spinal Muscular Atrophy who have been part of a previous study or treated with specific drugs. Women must not be pregnant and agree to contraception; men also need to use birth control. Participants should be well enough in the investigator's opinion, and young children may need feeding tubes or ventilation as recommended.

Inclusion Criteria

I have previously participated in the Moonfish study or have been treated with specific drugs for my condition.

Adequately recovered from any acute illness at the time of screening and considered well enough to participate in the opinion of the Investigator For women of childbearing potential: negative blood pregnancy test at screening, agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, and agreement to refrain from donating eggs for at least 28 days after the final dose of study drug

I agree to not have sex or use birth control and not donate sperm.

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Exclusion Criteria

I have a history of cancer that is not considered cured.

I have previously used chloroquine, hydroxychloroquine, retigabin, vigabatrin, or thioridazine.

I have had an eye disease in the last year.

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Treatment Details

Interventions

Risdiplam (Splicing Modifier)

Trial OverviewThe trial tests Risdiplam's safety, tolerability, how it moves through and affects the body (PK/PD) in those with SMA previously enrolled in a related study or treated with certain other medications. It's an exploratory open-label study without comparison groups.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: RisdiplamExperimental Treatment1 Intervention

Participants will receive multiple doses of risdiplam orally once daily for 24 months. After 24-month treatment, participants will be offered the opportunity to enter the open-label extension (OLE) phase for 3 years.

Risdiplam is already approved in Brazil, China for the following indications:

Approved in Brazil as Evrysdi for:

Spinal muscular atrophy (SMA)

Approved in China as Evrysdi for:

Spinal muscular atrophy (SMA)

Find a Clinic Near You

Who Is Running the Clinical Trial?

Hoffmann-La Roche

Lead Sponsor

Trials

2,482

Recruited

1,107,000+

Headquarters

Basel, Switzerland

Known For

Precision medicine

Risdiplam for Spinal Muscular Atrophy (Jewelfish Trial)