Sunfish

• Research type

  Research Study

• Full title

  A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLEBLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RISDIPLAM (RO7034067) IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS.

• IRAS ID

  290294

• Contact name

  Laurent Servais

• Contact email

  laurent.servais@paediatrics.ox.ac.uk

• Sponsor organisation

  Syneos Health

• Eudract number

  2016-000750-35

• Clinicaltrials.gov Identifier

  NCT02908685

• Duration of Study in the UK

  2 years, 0 months, 1 days

• Research summary

  Research Summary

  The participants have been participating in SUNFISH study (risdiplam in SMA) at a clinic abroad and their residence is in the UK. Due to the travel restrictions imposed during the current COVID-19 pandemic, we have arranged for the participants to continue to be seen by a study doctor in Oxford (UK). The study visits at Oxford will be similar to the study visits the participants had abroad, the established schedule will be resumed.
  This research study is being done to try to measure how well a new investigational medicine called risdiplam (also known as RO7034067) works and how safe it is in treating people with Type 2 and 3 Spinal Muscular Atrophy (SMA).As part of this study, the participants may be given a placebo. A placebo is a drug that looks exactly like the investigational medicine, but has no active ingredient. This study includes a first part, Part 1 with 51 participants. Part 1 was the dose-escalation part of the study and allowed the selection of the appropriate dose for the second part (that is Part 2). The participants will be enrolled in Part 2 of the study. This part is a confirmatory part for evaluating the safety of risdiplam and the effects of risdiplam on the participant’s SMA disease. Part 2 enrolled 180 participants, 2-25 years of age. The participants will be in the study for approximately 25 months (plus the 1-month screening period), but they may receive a placebo only for a maximum of 12 months after which he/she will be able to receive active risdiplam drug for 12 months before switching to the open-label extension.
  Once the participants complete treatment and assuming study results are in favour of risdiplam (i.e., overall the results suggest that benefit outweighs side effects and possible risks), it is planned that the participants will be offered continued treatment with risdiplam as part of the open-label extension phase of the study. The open-label extension (OLE) part of the study, which is offered to participants completing the study, is planned to end when the last participants enrolled completes a total of 5 years in the study or earlier, if the medicine is commercially available in the UK or the Sponsor stops development of the medicine.

  Summary of Results

  A first LayPerson Summary (LPS) of the 12-month primary study results was made publicly available in January 2021 on the forpatients.roche.com website.
  2. A second LPS of the 24-month study results was made publicly available in January 2022 on the same website.
  See link: https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Fu2790089.ct.sendgrid.net%2Fls%2Fclick%3Fupn%3Du001.QwnS5iG-2B4nwcunWxC03fkI-2B2FmlpOZCx9oHLzlTYNfEUWaADqb0w2tMtRTl1mlmShcPxbUZWdOX2hBjUwCUHTTWAoGmVwKg6KofbJaSJMtmLfoe3IexOsch-2Fx3dafeFh3Z7xalSUYgOaB4qe05418GbIbzZ2q3-2BOFz4wp-2BKvO7cFPhfAmCo9W-2BvcYXck-2Bb3X-2B0dDIUsav6YG-2F9m6k8ca1Q-3D-3Dk9SC_a04dWCHmDh7gi4hRuvmsd-2FMEDkPNm6D0IRdq0o1v81d4exZ3qVLiRvdEYUBe-2BfaFjLLJvyjHrr1-2FOzymCidDWUb6ojFYPbyq7c-2B2zK0U48uhVx3BX3-2FogUdgRIgc3ER-2BcoDQI4Lm9aG5ufQ-2BSddoKpOwEQ-2FT6EhLeMmMCrkB3Yx2KTpPobPtpHKq0WPD-2B9E6XJad9PvNv0E8KlZJG-2F7Vaw-3D-3D&data=05%7C02%7Cedgbaston.rec%40hra.nhs.uk%7Cf2231d5d684d4cce9ef008dc4f3142c1%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638472320203841008%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C0%7C%7C%7C&sdata=q6KTC2W6hshO65ySuXeuWTEGOI1G3nHv0n%2B7KHzGnsE%3D&reserved=0

  A LPS of the final 5-year results is currently in development and is based on the final CSR issued on 01 March 2024. This final LPS will be released by mid-June 2024 on the forpatients.roche.com website.

• REC name

  West Midlands - Edgbaston Research Ethics Committee

• REC reference

  20/WM/0300

• Date of REC Opinion

  25 Nov 2020

• REC opinion

  Favourable Opinion