Extension Study of XLHED male subjects treated with EDI200 in ECP-002
Research type
Research Study
Full title
Extension Study of XLHED-Affected Male Subjects treated with EDI200 in Protocol ECP-002
IRAS ID
157960
Contact name
Ramsey Johnson
Contact email
Sponsor organisation
Edimer Pharmaceuticals Inc.
Eudract number
2013-004565-14
Clinicaltrials.gov Identifier
Duration of Study in the UK
11 years, 1 months, 25 days
Research summary
The goal of this extension study in XLHED-affected boys is to demonstrate the long-term safety and efficacy of EDI200 and to continue the evaluation of all EDI200-treated ECP-002 subjects up to age 10 years. No additional study drug administration is planned. The efficacy evaluations will incorporate growth and development parameters, frequency of infections and hospitalizations, and age-appropriate assessments of ectoderm-derived organ function. The safety evaluations will include physical examinations, adverse events and concomitant medication documentation, and laboratory testing.
This open-label extension study will enroll as many as 10 XLHED-affected males previously having received EDI200 in the ECP-002 trial. XLHED-affected male infants who completed the ECP-002 trial will be enrolled at age 6 months followed with yearly visits starting at age 1 year. The research will take place at the Children's Department of the University Hospital of Wales, UK.REC name
Wales REC 3
REC reference
15/WA/0006
Date of REC Opinion
13 Jan 2015
REC opinion
Favourable Opinion