Extension Study of XLHED-Affected Male Subjects Treated With EDI200 in Protocol ECP-002

• Status: Active, not recruiting
• Trial end date: 2025-03-01
• Target enrollment: 0
• Participant gender: Male

Summary

The goal of the ECP-002e extension study is to continue the evaluation of all EDI200-treated ECP-002 subjects up to age 10 yrs. 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. Funding Source - FDA OOPD

• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Edimer Pharmaceuticals

Criteria

Inclusion Criteria:

Subjects must meet all of the following criteria to be enrolled:

1. Subject received at least one dose of EDI200 in the neonate study ECP-002

2. Written informed consent of parent(s)

Exclusion Criteria:

Subjects who meet any of the following criteria may not be enrolled in this study:

1. Medically-significant postnatal complications or congenital anomalies outside of those considered to be associated with the diagnosis of XLHED

2. Major protocol violations during enrollment in study ECP-002 as determined by the Sponsor