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

• Register: EUCTR
• Last refreshed on: 26 March 2024
• Main ID: EUCTR2013-004565-14-DE
• Date of registration: 13/01/2014
• Prospective Registration: Yes
• Primary sponsor: Edimer Pharmaceuticals, Inc.
• Public title: Extension Study of XLHED-Affected Male Subjects treated with EDI200 in Protocol ECP-002
• Scientific title: Extension Study of XLHED-Affected Male Subjects treated with EDI200 in Protocol ECP-002
• Date of first enrolment: 13/02/2014
• Target sample size: 10
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-004565-14
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

France, Germany, United States

Contacts

• Name: Clinical Trials Information
• Affiliation: Edimer Pharmaceuticals, Inc.

• Name: Clinical Trials Information
• Affiliation: Edimer Pharmaceuticals, Inc.

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)

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



Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

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: 17.1 Level: LLT Classification code 10072592 Term: Hypohidrotic ectodermal dysplasia System Organ Class: 100000004850

Intervention(s)

Product Code: EDI200
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human immunoglobulin G1 constant region - human ectodysplasin-A1 receptorbinding domain fusion protein
Current Sponsor code: EDI200
Other descriptive name: Fc: EDA-A1 fusion protein
Concentration unit: mg/l milligram(s)/litre
Concentration type: not less then
Concentration number: 5-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: 1. Growth and development:
Enrollment and anually: physical examination, feeding history, measurement of weight, length, head circumfence (up to 2 years)

2. Dentition:
At ages 2 yrs, 5 yrs, and 10 yrs

Secondary Objective: 1. Efficacy outcomes:
- Mortality, hospitalizations, infections, unexplained fevers and heat intolerance as compared with rate of occurrence in male siblings and historical controls
- Sweat gland density and function as compared with data from end-of-study visit in study ECP-002
- Skin, hair, respiratory and ocular health as compared with male siblings and historical controls

2. Safety outcomes:
- Physical examinations with vital signs
- Adverse events
- Concomitant medications
- Safety laboratory values

Exploratory Outcome Measures
- Craniofacial development
- Exercise thermoregulation
- Immunogenicity

Primary end point(s): 1. Growth and development as compared with age-matched standards:
Complete physical examination in addition to feeding history and measurements of weight, length, and head circumfences

2. Dentition:
Assessed by dental examinations and radiographs

Main Objective: 1. Growth and development as compared with age-matched standards
2. Dentition assessed by dental examinations and radiographs at ages 2 yrs, 5 yrs, and 10 yrs

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: 1. Efficacy outcomes:
a) Mortality, hospitalizations etc. and b) Sweat and c) skin assessments: at enrollment and annually
d) Hair assessments: at age 5 and 10 yrs
e) Pulmonary assessments: annually; Pulmonary function testing and eNO levels starting at age 5 yrs
f) Ocular health: at enrollment and annually; Tear production and OSDI starting at age 5 yrs

2. Safety outcomes:
a) Physical examinations: at enrollment and annually
b) Adverse events and Concomitant medications: continously
c) Safety lab: at enrollment and at age 2 yrs visit

3. Exploratory:
a) Craniofacial development: at enrollment and annually
b) Exercise thermoregulation: at age 7 yrs and a minimum weight of 36 kg
c) Immunogenicity: at enrollment and at age 2 yrs visit

Secondary end point(s): 1. Efficacy outcomes:
a) Mortality, hospitalizations, infections, unexplained fevers and heat intolerance as compared with rate of occurrence in male siblings and historical controls
b) Sweat gland density and function as compared with data from end-of-study visit in study ECP-002
c) Skin health as compared with male siblings and historical controls
d) hair assessments as compared with male siblings and historical controls
e) respiratory assessments as compared with male siblings and historical controls
f) ocular health as compared with male siblings and historical controls

2. Safety outcomes:
a) Physical examinations with vital signs
b) Adverse events
c) Concomitant medications
d) Safety laboratory values

3. Exploratory Outcome Measures:
a) Craniofacial development
b) Exercise thermoregulation
c) Immunogenicity

Secondary ID(s)

NCT01992289

ECP-002e

Source(s) of Monetary Support

Edimer Pharmaceuticals, Inc.

Ethics review

Status: Approved
Approval date: 13/02/2014
Contact: