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Abstract Details

SUNFISH Part 2: Efficacy and Safety of Risdiplam (RG7916) in Patients with Type 2 or Non-Ambulant Type 3 Spinal Muscular Atrophy (SMA)
Child Neurology and Developmental Neurology
P8 - Poster Session 8 (8:00 AM-9:00 AM)
5-010

To determine the safety and efficacy of risdiplam (RG7916) in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA) treated for 12 months during confirmatory Part 2 of the SUNFISH study.
SMA is a severe, progressive neuromuscular disease caused by reduced levels of survival of motor neuron (SMN) protein due to deletions and/or mutations of the SMN1 gene. A second gene, SMN2, produces only low levels of functional SMN protein. Risdiplam is an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier that increases the levels of functional SMN protein.
SUNFISH (NCT02908685) is a multicenter, two-part, randomized, placebo-controlled, double-blind study (randomized 2:1, risdiplam:placebo) in patients, aged 2–25 years, with Type 2 or Type 3 SMA.
SUNFISH is comprised of two parts: Part 1 (n=51) is a dose-selection study assessing the safety, tolerability and pharmacokinetics/pharmacodynamics of different risdiplam dose levels in patients with Type 2 and Type 3 SMA (ambulant and non-ambulant); confirmatory Part 2 (n=180) assesses the safety and efficacy of the risdiplam dose level that was selected from Part 1 compared with placebo in patients with Type 2 and non-ambulant Type 3 SMA. The primary objective of Part 2 is to evaluate the efficacy of risdiplam compared with placebo in terms of motor function as assessed by the change from baseline in the 32-item Motor Function Measure total score at Month 12.

In SUNFISH Part 1, no drug-related safety findings led to withdrawals from the study following 1 year of treatment with risdiplam (data-cut: 28th June 2019). Here we will report data from confirmatory Part 2 of the SUNFISH study including baseline demographics, safety and efficacy data in participants who have received treatment with risdiplam or placebo for 12 months.

Part 2 of SUNFISH is currently ongoing.
Authors/Disclosures

PRESENTER 		No disclosure on file
Nina Barisic, MD, PhD (University Hospital Centre Zagreb) The institution of Dr. Barisic has received personal compensation in the range of $0-$499 for serving as a Consultant for roche. The institution of Dr. Barisic has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Novartis.
No disclosure on file
Nicholas Deconinck Nicholas Deconinck has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne. Nicholas Deconinck has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for roche. Nicholas Deconinck has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for biogen. Nicholas Deconinck has received publishing royalties from a publication relating to health care.
No disclosure on file
No disclosure on file
No disclosure on file
Andres Nascimento Osorio Andres Nascimento Osorio has nothing to disclose.
Kayoko Saito No disclosure on file
Dmitry Vlodavets No disclosure on file
No disclosure on file
Sabine Fuerst-Recktenwald No disclosure on file
No disclosure on file
No disclosure on file
Ksenija Gorni Dr. Gorni has received personal compensation for serving as an employee of Hoffman La Roche.
Heidemarie Kletzl Heidemarie Kletzl has received personal compensation for serving as an employee of Hoffmann-La Roche.
No disclosure on file
No disclosure on file
John W. Day, MD, PhD (Stanford University School of Medicine) Dr. Day has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis Gene Therapy. Dr. Day has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche/Genentech. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity. Dr. Day has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PepGen. Dr. Day has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Epirium Bio. Dr. Day has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid Biosciences. Dr. Day has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Muscular Dystrophy Association. The institution of Dr. Day has received research support from Astellas Pharma. The institution of Dr. Day has received research support from Novartis Gene Therapy. The institution of Dr. Day has received research support from Biogen. The institution of Dr. Day has received research support from Roche/Genentech. The institution of Dr. Day has received research support from Sanofi/Genzyme. The institution of Dr. Day has received research support from Sarepta. The institution of Dr. Day has received research support from Scholar Rock. The institution of Dr. Day has received research support from AMO Pharma. The institution of Dr. Day has received research support from AnnJi. Dr. Day has received research support from CureSMA. The institution of Dr. Day has received research support from Muscular Dystrophy Association. The institution of Dr. Day has received research support from Ionis Pharmaceuticals. The institution of Dr. Day has received research support from NMD Pharma. The institution of Dr. Day has received research support from SMA Foundation. Dr. Day has received intellectual property interests from a discovery or technology relating to health care.