Extension Study of XLHED-Affected Male Subjects treated with EDI200 in Protocol ECP-002

Phase 1

• Conditions: X-linked hypohidrotic ectodermal dysplasia (XLHED) is caused by inherited defects in the ectodysplasin (EDA) gene that disrupt synthesis and/or function of the primary translational product EDA-A1. The absence of normal EDA-A1 expression results in sweat and secretory gland hypoplasia predisposing XLHED-affected infants to serious a potentially life-threatening hyperthermia and pneumonia.; MedDRA version: 18.0Level: LLTClassification code 10072592Term: Hypohidrotic ectodermal dysplasiaSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2013-004565-14-FR
• Lead Sponsor: Edimer Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-06-25
• Last Update Posted: 2 October 2017

Recruitment & Eligibility

• Status: A
• Sex: Male
• Target Recruitment: 10

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)

Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified