P.063 SUNFISH Part 1 results and Part 2 trial design in patients with type 2/3 spinal muscular atrophy (SMA) receiving risdiplam (RG7916)
| العنوان: | P.063 SUNFISH Part 1 results and Part 2 trial design in patients with type 2/3 spinal muscular atrophy (SMA) receiving risdiplam (RG7916) |
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| المؤلفون: | L Servais, Ksenija Gorni, Janbernd Kirschner, Y. Cleary, Eugenio Mercuri, W. Yeung, Marianne Gerber, Craig Campbell, Nathalie Goemans, Heidemarie Kletzl, Jeppe Buchbjerg, Omar Khwaja, M Pera, Christian Czech, Giovanni Baranello |
| المصدر: | Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques. 46:S31 |
| بيانات النشر: | Cambridge University Press (CUP), 2019. |
| سنة النشر: | 2019 |
| مصطلحات موضوعية: | Oncology, medicine.medical_specialty, business.industry, Survival of motor neuron, General Medicine, SMN1, Spinal muscular atrophy, SMA, Placebo, medicine.disease, nervous system diseases, nervous system, Neurology, Tolerability, Pharmacokinetics, Internal medicine, medicine, Neurology (clinical), Adverse effect, business |
| الوصف: | Background: SMA is characterized by reduced levels of survival of motor neuron (SMN) protein from deletions and/or mutations of the SMN1 gene. While SMN1 produces full-length SMN protein, a second gene, SMN2, produces low levels of functional SMN protein. Risdiplam (RG7916/RO7034067) is an investigational, orally administered, centrally and peripherally distributed small molecule that modulates pre-mRNA splicing of SMN2 to increase SMN protein levels. Methods: SUNFISH (NCT02908685) is an ongoing multicenter, double-blind, placebo-controlled, operationally seamless study (randomized 2:1, risdiplam:placebo) in patients aged 2–25 years, with Type 2/3 SMA. Part 1 (n=51) assesses safety, tolerability, pharmacokinetics and pharmacodynamics of different risdiplam dose levels. Pivotal Part 2 (n=180) assesses safety and efficacy of the risdiplam dose level selected based on Part 1 results. Results: Part 1 results showed a sustained, >2-fold increase in median SMN protein versus baseline following 1 year of treatment. Adverse events were mostly mild, resolved despite ongoing treatment and reflected underlying disease. No drug-related safety findings have led to withdrawal (data-cut 06/17/18). SUNFISH Part 1 exploratory endpoint results and Part 2 study design will also be presented. Conclusions: To date, no drug-related safety findings have led to withdrawal. Risdiplam led to sustained increases in SMN protein levels. |
| تدمد: | 2057-0155 0317-1671 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_________::9a18015a9b40a6ff28a3f3702bb2b47dTest https://doi.org/10.1017/cjn.2019.163Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi...........9a18015a9b40a6ff28a3f3702bb2b47d |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 20570155 03171671 |
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