BP29420 - RO6885247 in Spinal Muscular Atrophy
Research type
Research Study
Full title
A MULTICENTER, RANDOMIZED, DOUBLE BLIND, PLACEBO CONTROLLED, MULTIPLE DOSE STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RO6885247 FOLLOWING 12 WEEKS OF TREATMENT IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY (MOONFISH)
IRAS ID
161635
Contact name
Hanns Lochmuller
Contact email
Sponsor organisation
F Hoffmann-La Roche Ltd
Eudract number
2014-002246-41
Duration of Study in the UK
0 years, 10 months, 2 days
Research summary
Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder characterized by the progressive loss of nerve function in muscles, which leads to muscle loss and weakness, tremors in fingers and hands, orthopaedic deformities and in certain types of SMA, progressive lung failure and frequent lung infections, to name but a few of the symptoms. SMA is the leading genetic cause of mortality in infants and young children with an incidence of 1 in 6,000-10,000 live births. Treatment is usually supportive, with the goals being to improve the patients’ quality of life and to minimize disability.
The present study will aim to look at whether an experimental medication, taken by mouth or feeding tube every day for 12 weeks is safe, efficacious and well tolerated in children and adults with Spinal Muscular Atrophy (SMA).
A total of approximately 48 patients will take part in the study globally; this number may be revised depending on dosing regimens.
Because SMA develops from a very young age (before 6 months) and by late childhood; and because patients may live into adulthood, patients between 2 and 55 years will be recruited. The study will be in 2 parts: Part 1 in adolescents and adults (12-55y) will be started first, followed by part 2 with children (2-11y). The dose of the study drug may be changed depending on the results of the previous group. Patients in the study will receive either an experimental drug or a placebo to take orally. Depending on the results on safety of the experimental drug, patients may be given access to this after the study.
The study will last approximately 6 months for each patient until the last patient to have enrolled has finished all study visits.
There will be approximately 20 patients recruited at 2 UK sites.
The study is sponsored by F. Hoffman La Roche.
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
14/NE/1171
Date of REC Opinion
28 Nov 2014
REC opinion
Further Information Favourable Opinion