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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01992289
• Date of registration: 12/11/2013
• Prospective Registration: Yes
• Primary sponsor: Edimer Pharmaceuticals
• Public title: Extension Study of XLHED-Affected Male Subjects Treated With EDI200 in Protocol ECP-002 ECP-002e
• Scientific title: Extension Study of XLHED-Affected Male Subjects Treated With EDI200 in Protocol ECP-002
• Date of first enrolment: March 2014
• Target sample size: 10
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT01992289
• Study type: Observational
• Phase: N/A

Countries of recruitment

France, Germany, Italy, United Kingdom, United States

Contacts

• Name: Kenneth Huttner, MD, PhD
• Affiliation: Edimer Pharmaceuticals

Key inclusion & exclusion 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



Age minimum: 6 Months
Age maximum: 1 Year
Gender: Male

Health Condition(s) or Problem(s) studied

X-linked Hypohidrotic Ectodermal Dysplasia

Intervention(s)

Drug: EDI200

Primary Outcome(s)

Dentition [Time Frame: Change from baseline dentition at 2, 5 and 10 years]

Growth and development [Time Frame: Annually up to age 10 years]

Secondary Outcome(s)

Hair growth [Time Frame: Change from baseline hair growth at 5 and 10 years]

Sweat function [Time Frame: Annually up to age 10 years]

Heat intolerance [Time Frame: Annually up to age 10 years]

Infections [Time Frame: Annually up to age 10 years]

Ocular health [Time Frame: Annually up to age 10 years]

Sweat gland density [Time Frame: Annually up to age 10 years]

Hospitalizations [Time Frame: Annually up to age 10 years]

Unexplained fevers [Time Frame: Annually up to age 10 years]

Skin health [Time Frame: Annually up to age 10 years]

Mortality [Time Frame: Annually up to age 10 years]

Number of participants with adverse events [Time Frame: Annually up to age 10 years]

Respiratory health [Time Frame: Annually up to age 10 years]

Secondary ID(s)

ECP-002e

1R01FD005099-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors