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 further investigated the selected dose, for 24 months. Five-year data provided as the final readout of the trial were announced in June 2024.
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 (HINALEA 1 – NCT05861986), while the other will include participants that have shown a plateau or decline in function post-gene therapy (HINALEA 2 – NCT05861999).
This study will evaluate the pharmacokinetics and safety of risdiplam in pre-symptomatic individuals with a genetic diagnosis of 5q SMA and under 20 days of age at first dose. The study is due to start in early 2024 and is expected enrol approximately 10 participants.
ACTIVENESS is a multi-country, non-interventional, prospective, longitudinal study to assess the long-term effectiveness of risdiplam in SMA. Study countries will be primarily in Europe, although non-European countries may be included to achieve the target sample size of 600 patients.
Study to develop a Tablet form of Risdiplam
A Phase 1 study (NCT04718181) was completed in 2023.
The study was designed to assess how well a tablet of 5mg risdiplam that could be swallowed whole or dispersed in a small amount of bottled water would work.
Results were presented at the SMA Europe congress in February 2024. They showed that, whether swallowed whole or dispersed:
- The tablet resulted in the same concentration of risdiplam in the body as the currently available oral solution
- The tablets may be given with or without food. Food had no effect on how well the tablet were absorbed. This was similarly demonstrated for the oral solution.
- No new safety signals were identified.
In the first half of 2024 Roche plans to apply to the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) for the tablet formulation to be considered for licencing.
In Spring 2024, Roche announced plans for this new, observational clinical trial (ISRCTN31399857).
It aims to collect patient-reported, fertility-related outcomes in adult males living with SMA who have been treated with risdiplam in the US.