Risdiplam for Presymptomatic Spinal Muscular Atrophy

(Rainbowfish Trial)

Recruiting at 20 trial locations

Overseen By Reference Study ID Number: BN40703 https://forpatients.roche.com/

Age: < 18

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 trial is testing a medicine called risdiplam, which is taken by mouth. It aims to help infants who have a genetic diagnosis of spinal muscular atrophy (SMA) but are not yet showing symptoms. The medicine works by helping the body make more of a protein that muscles need to stay healthy. The goal is to see if early treatment can prevent or lessen the severity of SMA.

Research Team

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Eligibility Criteria

This trial is for infants from birth to 6 weeks old with a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. They must be able to travel safely for study visits, have supportive caregivers, and not require invasive ventilation. Infants should weigh in the normal range for their age and have been born after a full-term pregnancy.

Inclusion Criteria

Receiving adequate nutrition and hydration at the time of screening, in the opinion of the investigator

I can safely travel to the study site for all required visits.

Able to complete all study procedures, measurements, and visits, and the parent (or caregiver), in the opinion of the investigator, has adequately supportive psychosocial circumstances

Exclusion Criteria

I have received treatments targeting the SMN2 gene.

My baby or I (if breastfeeding) haven't taken specific drugs affecting liver enzymes recently.

I am not taking oral salbutamol or similar drugs for SMA, but I may use inhaled versions.

Treatment Details

Interventions

• Risdiplam (SMN2 Splicing Modifier)

Trial Overview The trial is testing an oral medication called Risdiplam on infants who are genetically diagnosed with SMA but haven't shown symptoms. The goal is to see if early treatment can prevent or lessen muscle weakness caused by SMA.

Participant Groups

Group I: Open-label Risdiplam Experimental Treatment 1 Intervention

Participants will be enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range.

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)

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