RO7204239 + Risdiplam for Spinal Muscular Atrophy

(MANATEE Trial)

Recruiting at59 trial locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Waitlist Available

Sponsor: Hoffmann-La Roche

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial tests the safety and effectiveness of combining two treatments, RO7204239 and risdiplam, for patients with spinal muscular atrophy (SMA). Risdiplam helps the body produce a protein needed for muscle movement, while RO7204239 allows muscles to grow bigger and stronger. The study includes SMA patients to see if this combination improves their muscle function and overall health.

Research Team

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Eligibility Criteria

This trial is for individuals aged 2-25 with confirmed genetic diagnosis of 5q-autosomal recessive Spinal Muscular Atrophy (SMA). Participants must be able to walk or sit unassisted, as applicable, and have not had certain treatments or surgeries recently. They should not have severe heart issues, skin conditions at injection sites, major recent illnesses, or require daytime ventilation.

Inclusion Criteria

I can walk or run 10 meters in 30 seconds or less without help.

I am between 2 and 10 years old.

I have a genetic diagnosis of 5q-SMA.

Exclusion Criteria

I have not taken any drugs that are cleared by the kidneys in the last 2 weeks.

I have not taken specific medications in the last 90 days.

I have hereditary fructose intolerance.

Treatment Details

Interventions

• Placebo (Other)
• Risdiplam (Small Molecule)
• RO7204239 (Monoclonal Antibodies)

Trial Overview The study tests the safety and effectiveness of RO7204239 combined with Risdiplam in SMA patients. Part 1 determines the proper dose among ambulant/non-ambulant children; Part 2 assesses its impact on a broader age range. The goal is to improve muscle function by increasing SMN protein production and inhibiting myostatin.

Participant Groups

Group I: RO7204239 + Risdiplam Experimental Treatment 2 Interventions

Participants who have not previously been treated with risdiplam will receive risdiplam for at least 8 weeks prior to randomization into a treatment group (Part 1 only). Participants that have been treated with risdiplam for at least 8 continuous weeks immediately prior to joining the study may be immediately randomized to combination therapy, or join the study run-in period (the period between screening and randomization to a treatment group) where they will continue to receive risdiplam monotherapy until randomization. Participants enrolled in Part 1 will receive RO7204239 (low or high dose) + risdiplam for 24 weeks, followed by RO7204239 + risdiplam for 72 weeks. Participants enrolled in Part 2 will receive risdiplam for 8 weeks and then treatment with RO7204239 + risdiplam for 72 weeks. Once the treatment period has completed (Part 1 or Part 2), participants will have the option of treatment with RO7204239 + risdiplam for 2 additional years.

Group II: Placebo + Risdiplam Active Control 2 Interventions

Participants who have not previously been treated with risdiplam will receive risdiplam for at least 8 weeks prior to randomization into a treatment group (Part 1 only). Participants that have been treated with risdiplam for at least 8 continuous weeks immediately prior to joining the study may be immediately randomized to combination therapy, or join the study run-in period (the period between screening and randomization to a treatment group) where they will continue to receive risdiplam monotherapy until randomization. Participants enrolled in Part 1 will receive placebo (low or high dose-matched) + risdiplam for 24 weeks, followed by RO7204239 + risdiplam for 72 weeks. Participants enrolled in Part 2 will receive risdiplam for 8 weeks and then treatment with placebo + risdiplam for 72 weeks. Once the treatment period has completed (Part 1 or Part 2), participants will have the option of treatment with RO7204239 + risdiplam for 2 additional 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)

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