- Trial Name
- MANATEE
- Age:
- Part 1: 2-10 years; Part 2: 2-25 years
- Aim:
- This trial will study the safety and efficacy of GYM329 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
- Trial Name
- RAINBOWFISH
- Age:
- Up to 6 weeks old
- Aim:
- To investigate the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in infants aged from birth to 6 weeks who have been genetically diagnosed with SMA but are not yet presenting with symptoms
- Status:
- Active, not recruiting
- Locations:
- Belgium, Italy, Poland, Russian Federation, Australia, Brazil, China, Saudi Arabia, Taiwan & USA
- Trial Name
- JEWELFISH
- Age:
- 6 Months to 60 Years old
- Aim:
- To investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with SMA previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.
- Status:
- Completed
- Locations:
- Belgium, France, Germany, Italy, the Netherlands, Poland, Switzerland, UK & USA
- Trial Name
- SUNFISH
- Age:
- 2 to 25 Years old
- Aim:
- To investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam in Type 2 and 3 SMA patients
- Status:
- Complete
- Locations:
- Belgium, Croatia, France, Italy, Poland, Russian Federation, Serbia, Spain, Switzerland, Turkey, Ukraine, Brazil, Canada, China, Japan, Saudi Arabia and USA
- Trial Name
- FIREFISH
- Age:
- Type 1 SMA – 1 to 7 Months old
- Aim:
- To investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of risdiplam in infants with Type 1 SMA
- Status:
- Complete
- Locations:
- Belgium, Croatia, France, Germany, Italy, Poland, Russian Federation, Serbia, Spain, Turkey, UK, Brazil, China, Japan and USA
- Trial Name
- PUPFISH
- Age:
- <20 days at first dose
- Aim:
- This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
- Status:
- Recruiting
- Locations:
- Belgium, Canada, Germany, Italy, Norway, Poland, US
- Trial Name
- HINALEA 1
- Age:
- < 2 years of age
- Aim:
- This study will evaluate the effectiveness and safety of risdiplam administered as an early intervention in paediatric patients with spinal muscular atrophy after gene therapy
- Status:
- Recruiting
- Locations:
- Poland, US (more sites to be added in coming months)
- Trial Name
- HINALEA 2
- Age:
- < 2 years of age
- Aim:
- This study will evaluate the effectiveness and safety of risdiplam administered as in paediatric patients with spinal muscular atrophy who experienced a plateau or decline in function after gene therapy
- Status:
- Recruiting
- Locations:
- Germany, Israel, Poland, Qatar, UK, US
