JEWELFISH
Research type
Research Study
Full title
AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY
IRAS ID
219406
Contact name
Volker Straub
Contact email
Sponsor organisation
F. Hoffmann-La Roche Ltd
Eudract number
2016-004184-39
Clinicaltrials.gov Identifier
128972, IND No.
Duration of Study in the UK
3 years, 8 months, 0 days
Research summary
Spinal Muscular Atrophy (SMA) is a genetic condition which affects a person`s nerves that control muscle movement such as moving, walking and breathing but doesn`t affect their mental development. It causes the muscles throughout the body to become weak and shrink (atrophy). The muscles closest to the centre of the body, such as the shoulders, hips and back (proximal muscles) are usually affected first and most severely.\nIt is the leading genetic cause of mortality in infants and young children, with an incidence of 1 in approximately 11,000 live births and a carrier frequency estimated at 1 in 50-70 individuals. \n\nThe primary objectives of this study are to evaluate the safety and tolerability of the study medication, RO7034067, and to investigate how the study medication is absorbed, distributed, processed and removed from the body. \nRO7034067 is an investigational new medication (taken by mouth or feeding tube) being studied by F. Hoffmann-La Roche Ltd (Sponsor) for treating Spinal Muscular Atrophy. Animal and human studies have shown that RO7034067 may be useful as a treatment for Spinal Muscular Atrophy (SMA). RO7034067 has not been approved for marketing in any country.\nThe target sample size is up to 24 patients with Type 2 and Type 3 SMA (aged 12 to 60 years) previously enrolled in a study with an SMN2-targeting therapy.\nThe total duration of the study for each enrolled subject will be up to approximately 160 weeks (from screening through completion of follow-up): 30 day screening phase, 104-week treatment period, and 52-week follow-up period.
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
17/NE/0176
Date of REC Opinion
6 Sep 2017
REC opinion
Further Information Favourable Opinion