Results and Updates for Each Risdiplam Trial
Results and Updates for Each Risdiplam Trial
This trial began in December 2016 and with infants aged 1 – 7 months who had a clinical diagnosis of SMA Type 1. The first part of the trial enrolled 62 infants to investigate an exploratory dose, for 12 weeks. This was completed in November 2021. The second part with 41 infants is further investigating the dose selected, for 24 months. The estimated completion date of the study is mid November 2023.
This trial began in October 2016 with children and adults ages 2 – 25 years who had a clinical diagnosis of SMA Type 2 or Type 3. The first part of the trial enrolled 231 children and adults to investigate an exploratory dose, for 12 weeks. This was completed in September 2019. The second part with 180 children and adults is further investigating the dose selected, for 24 months. The estimated completion date of the study is early September 2023.
This trial began in March 2017 with children and adults ages 6 months – 60 years who had a clinical diagnosis of SMA Type 1, 2 or 3. The study enrolled 174 participants – 76 were previously treated with nusinersen and 14 with onasemnogene abeparvovec (zolgensma). The remaining 83 participants had been treated with compounds then being developed by Roche. The estimated completion date of the study is the end of January 2025.
This trial began in August 2019 with 25 pre-symptomatic infants aged up to 6 weeks genetically diagnosed with 5q SMA. Its primary completion date was June 2021. The estimated completion date of the study is early March 2026.
Please note: There are no plans to bring these trials to the UK at the moment.
Initiated in mid-2023 and not yet recruiting, these two open-label trials are designed to assess the safety and efficacy of Risdiplam in infants and children with SMA who have previously received Zolgensma. With estimated completion dates in mid-2028, the two studies will each enrol approximately 28 participants with two copies of SMN2 and aged 3 to 24 months. The major difference between the two studies is that one trial will include participants who have not experienced clinically significant decline in function from the time of Zolgensma administration (NCT05861986), while the other will include participants that have shown a plateau or decline in function post-gene therapy (NCT05861999).