Clinical Trials

Overview

GYM329

Developed by Roche, GYM329 (RO7204239) is an investigational anti-myostatin antibody that is designed to target skeletal muscles, potentially increasing their size and growth. 

The trial of GYM 329, a study called MANATEE, is a global Phase 2/3 clinical study to evaluate the safety and efficacy of GYM329 (RO7204239) in combination with risdiplam. The trial has two parts; Part 1 is the dose-finding part in SMA patients that are either ambulant (aged 2-10 years) or non-ambulant (aged 5-10 years) within separate cohorts, and Part 2 is the pivotal part in SMA patients aged 2-25 years that are ambulant.

The MANATEE study consists of two parts:

  • Part 1 will assess the safety of two doses of GYM329 in combination with risdiplam, with the aim of selecting the optimal dose of GYM329 for Part 2 of the study. It is expected to enrol 39 ambulant participants in Cohorts A-C, and 54 non-ambulant participants in cohort D
  • Part 2, the main part of the study, will assess the efficacy and safety of the GYM329 dose selected in Part 1 when combined with risdiplam. It is expected to enrol approximately 166 participants. Part 2 will commence once the dose selection in Part 1 is complete.

Participants will be randomly assigned to either a treatment group that receives risdiplam and GYM329, or a treatment group that receives risdiplam and a placebo. All participants in the study will receive a daily dose of risdiplam at the approved dose throughout the study, including those in the placebo group.

Method of Action

GYM329 is an antibody that binds a protein called myostatin. Myostatin plays an important role in the regulation of skeletal muscle size by controlling growth. Inhibiting myostatin may help muscles grow in size and strength.

GYM329 in combination with risdiplam, which is designed to increase the amount of SMN protein throughout the body, has the potential to further improve motor function and outcomes for people living with SMA.

Trial of GYM329

Trial Name
Manatee
Age:
2 to 10 years old & 2 to 25 yrs.
Aim:
This trial will study the safety and efficacy of RO7204239 in combination with risdiplam in patients with spinal muscular atrophy (SMA). The trial has two parts; Part 1 is the dose-finding part in SMA patients that are either ambulant (aged 2-10 years) or non-ambulant (aged 5-10 years) within separate cohorts, and Part 2 is the pivotal part in SMA patients aged 2-25 years that are ambulant.
Status:
Part 1 - Active, not recruiting; Part 2: not yet recruiting
Locations:
Australia, Belgium, Canada, Croatia, Italy, Japan, Netherlands, Poland, Portugal, Spain, UK, USA
Identifier:
NCT05115110

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