A study to evaluate the safety&activity of DT-200 in healthy subjects
Research type
Research Study
Full title
RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL GROUP PHASE 1b STUDY TO EVALUATE THE SAFETY, PHARMACOKINETICS, ACTIVITY AND EFFICACY OF DT-200 IN NORMAL HEALTHY ADULT MALE SUBJECTS
IRAS ID
215769
Contact name
Annelize Koch
Contact email
Sponsor organisation
Akashi Therapeutics
Eudract number
2016-004100-62
Duration of Study in the UK
0 years, 3 months, 20 days
Research summary
This study is to help development a drug called DT-200. DT-200 is proposed as a treatment for patients with muscular dystrophies, such as Duchenne muscular dystrophy (DMD). DMD is a common form of muscular dystrophy, effecting 1 in 3500 males born. It is also the most common fatal genetic disease, and those diagnosed with DMD in childhood have a life expectancy of 19 years old.
It is a placebo-controlled, parallel 4 arm design, with a 3:1 randomization. A total of 20 patients will receive placebo and 60 patients will be randomized to one of three doses of active drug (0.25, 0.5 and 1 mg/day).
The key objective is to assess the safety and tolerability of DT-200 and also to analyse the activity of the DT-200 by looking at changes in total lean body mass. Total lean body mass will be measured by dual energy x-ray absorptiometry (DXA).
The population who are eligible to take part in the study are healthy male, non-smoking volunteers, aged between 18 and 49 years, as determined by screening tests at Simbec.
Participation in the trial will last for about 11 weeks (from first screening visit to final end of study visit).
REC name
Wales REC 1
REC reference
16/WA/0302
Date of REC Opinion
12 Jan 2017
REC opinion
Further Information Favourable Opinion