Risdiplam for Spinal Muscular Atrophy

(Jewelfish Trial)

Recruiting at 38 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Hoffmann-La Roche

Must be taking: Nusinersen, Olesoxime, AVXS-101

Must not be taking: Chloroquine, Hydroxychloroquine, Retigabin, Vigabatrin

Disqualifiers: Unstable diseases, Drugs of abuse, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 9 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 24 months

174 Participants Needed

What You Need to Know Before You Apply

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.

Who Is on the Research Team?

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Are You a Good Fit for This Trial?

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.

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.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple doses of risdiplam orally once daily

24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may opt into continuation of treatment long-term

3 years

What Are the Treatments Tested in This Trial?

Interventions

• Risdiplam

Trial Overview The 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.

How Is the Trial Designed?

Group I: Risdiplam Experimental Treatment 1 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 United States, European Union, Brazil, China for the following indications:

Approved in United States as Evrysdi for:

• Spinal muscular atrophy (SMA) in patients 2 months of age and older

Approved in European Union as Evrysdi for:

• 5q-autosomal recessive SMA with a clinical diagnosis of SMA types 1, 2, or 3 or with one to four survival motor neuron 2 (SMN2) copies

Approved in Brazil as Evrysdi for:

• Spinal muscular atrophy (SMA)

Approved in China as Evrysdi for:

• Spinal muscular atrophy (SMA)

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

Top Products

Avastin, Herceptin, Rituxan, Accu-Chek

Dr. Levi Garraway

Hoffmann-La Roche

Chief Medical Officer since 2019

MD from the University of Basel

Dr. Thomas Schinecker

Hoffmann-La Roche

Chief Executive Officer since 2023

PhD in Molecular Biology from New York University