SMTC11004 - Phase 1 study in healthy volunteers and DMD patients
Research type
Research Study
Full title
A Phase I, 2-Part, Open-label, Multiple Oral Dose Study of the Safety, Tolerability and Pharmacokinetics of up to 2 Formulations of SMT C1100 in Healthy Adult Male Subjects and a Selected Formulation of SMT C1100 in Paediatric Subjects with Duchenne Muscular Dystrophy (DMD)
IRAS ID
181642
Contact name
Francesco Muntoni
Contact email
Sponsor organisation
Summit (Oxford) Limited
Eudract number
2015-001967-38
Duration of Study in the UK
0 years, 6 months, 16 days
Research summary
This is a phase 1, 2-part, open-label, multiple oral dose study performed by Summit (Oxford) Limited. The purpose of Part A is to investigate how the body handles SMT C1100 and its breakdown products in healthy adult male volunteers. Depending on availability, one or two formulations of SMT C1100 will be studied. It is hoped this will assist the design (choice of formulation, timing of doses and blood samples) of part B when SMT C1100 will be given to children, aged 5-9 years, with Duchenne Muscular Dystrophy (DMD).
SMT C1100 is a drug that may be useful in the treatment of DMD. DMD is a progressive, lethal muscle wasting disease, characterised by a generalised weakness and progressive loss of muscle strength. This condition is caused by changes (mutations) in the genes responsible for the structure and functioning of a person's muscles.
Part A in healthy male adults will be carried out in Quintiles, Drug Research Unit, London. Part B will be an open-label, non-randomised study in paediatric subjects with DMD and will be conducted at up to 8 centres in the UK. Total duration of Part A will be 8 weeks and total duration of Part B will be approximately 9 weeks (this period includes screening period and washout time between treatment periods).
REC name
London - West London & GTAC Research Ethics Committee
REC reference
15/LO/0920
Date of REC Opinion
3 Aug 2015
REC opinion
Further Information Favourable Opinion