RAINBOWFISH Clinical Trial Results
RAINBOWFISH Clinical Trial Results
Last updated: 5th November 2024
14th October 2024
Majority of children with pre-symptomatic SMA who began risdiplam treatment before six weeks of age can sit, stand and walk independently
At the recent World Muscle Society congress, Roche presented two-year data from the ongoing RAINBOWFISH study. This trial is evaluating the efficacy and safety of Evrysdi® (risdiplam) in children with SMA who began treatment pre-symptomatically, before six weeks of age.
The study analysed outcomes against the number of copies of the SMN2 gene each child had. Generally, fewer SMN2 copy numbers are associated with more severe SMA.
The results showed that the majority of these children achieved key motor milestones, could swallow and feed orally and demonstrated cognitive abilities typical of children without SMA. Importantly, none required permanent ventilation.
The key findings were:
- All children in the study with three or more copies of the SMN2 gene achieved key developmental milestones such as standing and walking, as assessed by developmental and motor function tests. Most met these milestones within the typical World Health Organization (WHO) windows for child development.
- All children with two copies of the SMN2 gene could sit and most could stand and walk independently after two years of treatment.
- After two years of treatment, all children in the study were able to swallow and feed orally with none requiring permanent ventilation.
- After two years of Evrysdi treatment, the children demonstrated cognitive skills on par with those of children without SMA. This study was the first clinical trial in SMA to assess cognition as an exploratory endpoint using a standardised scale.
In contrast, natural history studies suggest that without disease-modifying treatment, children with Type 1 SMA typically would not reach these milestones or live beyond two years of age.
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9th October 2023
At the 28th World Muscle Society (WMS) Congress, Roche confirmed that the RAINBOWFISH clinical trial has met its primary endpoint of 80% of babies sitting without support for at least five seconds after one year of treatment with risdiplam. Without treatment these babies would never be able to sit. This follows the news in August that the European Commission approved risdiplam for babies under two months old with SMA, based on an interim analysis of the RAINBOWFISH study.
23rd March 2022:
Updated interim data, presented virtually at the 2022 MDA Clinical and Scientific Conference on 13th – 16th March, demonstrated the safety and efficacy of Evrysdi for newborns. The majority of babies treated with Evrysdi for at least 12 months were able to stand and walk within timeframes typical of healthy babies.
14th June 2021:
Preliminary results were presented at the Cure SMA 2021 Virtual SMA Research & Clinical Care Meeting, which took place from 9th to 11th June 2021.
All five babies had been treated with risdiplam for at least 12 months:
Motor function:
- Achieved sitting without support, rolling and crawling. Of the five, two had two SMN copies and three had >2 copies.
- Four of the infants were able to stand unaided and walk independently. In addition, four babies reached a maximum score of 64 on the CHOP-INTEND scale, and one scored 63. Data on the primary endpoint, which is the number of infants sitting without support for at least five seconds, will be reported when all patients have reached one year of treatment.
Safety:
- There were no Adverse Events (AEs) leading to withdrawal or study discontinuation.
- The most common AEs were nasal congestion (33%), cough (25%), teething (25%), vomiting (25%), eczema (17%), abdominal pain (17%), diarrhoea (17%), gastroenteritis (17%), papule (rash; 17%) and pyrexia (fever; 17%).