- Trial Name
- MANATEE
- Age:
- 2 to 10 years old
- Aim:
- To investigate the safety and efficacy of RO7204239 in combination with risdiplam in ambulatory children with SMA
- Status:
- Not yet recruiting
- Locations:
- Belgium, Germany, Italy, the Netherlands, Poland, 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:
- 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:
- Active, not recruiting
- 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:
- Active, not recruiting
- 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:
- Active, not recruiting
- Locations:
- Belgium, Croatia, France, Germany, Italy, Poland, Russian Federation, Serbia, Spain, Turkey, UK, Brazil, China, Japan and USA