SUNFISH 2016.

Trial name or title	A study to Investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy (SMA) participants (SUNFISH)
Methods	Randomised, placebo‐controlled, double‐blind trial
Participants	186 participants with SMA types II and III, aged 2–25 years old with a confirmed diagnosis of 5q‐autosomal‐recessive SMA
Interventions	Participants start with treatment in part 1 of the study: Part 1A includes adolescents and adults (aged 12–25 years) with SMA type II or III, ambulatory or non‐ambulatory, receiving 12 weeks of placebo or RO7034067 Part 1B: children with SMA type II or III (aged 2–11 years), ambulatory or non‐ambulatory, receiving 12 weeks of placebo or RO7034067 After 12 weeks of treatment, participants can participate in part 2 of the trial if they meet inclusion criteria. Part 2: participants aged 2–25 years with SMA type II or III, non‐ambulatory with RULM entry item 1A ≥ 2 who have the ability to sit independently as assessed by item 9 of the MFM. 1 group will start with 12 months of placebo and will switch to treatment with RO7034067. 1 group will be treated for 24 months with RO7034067. All participants in part 2 will be offered the opportunity to enter an open‐label extended phase after completing part 2.
Outcomes	Safety (incidence of adverse events), pharmacokinetics (plasma concentrations of RO6885247 and RO6885247 exposure), pharmacodynamics (SMN protein levels in blood and in vivo splicing of SMN2 mRNA in blood), change from baseline in motor scores (MFM‐32, HFMSE, RULM), change from baseline in pulmonary function (FVC, SNIP, FEV1, peak cough flow), change from baseline in quality of life (PedsQL) and (severe) adverse events
Starting date	October 2016
Contact information	Hoffmann‐La Roche
Notes	Study ongoing and recruiting participants

FEV₁: forced expiratory volume in 1 second; FVC: forced vital capacity; HFMS(E): Hammersmith Functional Motor Scale (Expanded); MFM: Motor Function Measure; MMT: manual muscle testing; mRNA: messenger ribonucleic acid; PedsQL: Pediatric Quality of Life Inventory; RULUM: Revised Upper Limb Module; SNIP: sniff nasal inspiratory pressure; RULM; Revised Upper Limb Module; SMA: spinal muscular atrophy; WHO: World Health Organization.