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

NCT01992289 | Unknown

• 2 other identifiers: ECP-002e1R01FD005099-01
• Study Type: observational
• Target: 10
• Locations: 5 countries
• Sites: 7

Brief 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

Conditions

X-linked Hypohidrotic Ectodermal Dysplasia

Keywords

XLHED; HED; Hypohidrotic ectodermal dysplasia; Christ-Siemens-Touraine Syndrome

Outcome Measures

Primary Outcomes (2)

• Growth and development

  Growth and development as compared with age-matched standards

  Annually up to age 10 years

• Dentition

  Dentition assessed by dental examinations and radiographs at ages 2 yrs, 5 yrs, and 10 yrs

  Change from baseline dentition at 2, 5 and 10 years

Secondary Outcomes (12)

• Mortality

  Annually up to age 10 years

• Hospitalizations

  Annually up to age 10 years

• Infections

  Annually up to age 10 years

• Unexplained fevers

  Annually up to age 10 years

• Heat intolerance

  Annually up to age 10 years

Other Outcomes (3)

• Craniofacial development

  Annually up to age 10 years

• Exercise thermoregulation

  Change from baseline exercise thermoregulation at 7, 8, 9 or 10 years (done once)

• Immunogenicity

  Change from baseline immunogenicity at 2 years

Study Arms (1)

No treatment

This is a long-term follow-up study of subjects that received EDI200 as part of protocol ECP-002.

Drug: EDI200

Interventions

EDI200 DRUG

Long-term follow-up study of subjects that received EDI200 as part of protocol ECP-002

Also known as: APO200

No treatment

Eligibility Criteria

• Age: 6 Months - 1 Year
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

This is an open-label extension study without additional study drug administration on 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 followed with yearly visits starting at age 1 year.

You may qualify if:

• Subjects must meet all of the following criteria to be enrolled:
• Subject received at least one dose of EDI200 in the neonate study ECP-002
• Written informed consent of parent(s)

You may not qualify if:

• Subjects who meet any of the following criteria may not be enrolled in this study:
• Medically-significant postnatal complications or congenital anomalies outside of those considered to be associated with the diagnosis of XLHED
• Major protocol violations during enrollment in study ECP-002 as determined by the Sponsor

Sponsors & Collaborators

• Edimer Pharmaceuticals: lead

Study Sites (7)

University of California, San Francisco

San Francisco, California, 94143, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Hôpital Necker-Enfants Malades

Paris, 75015, France

Location

Universitätsklinikum Erlangen

Erlangen, Bavaria, 91054, Germany

Location

Azienda Ospedaliera-Polo Universitario "Luigi Sacco"

Milan, 20157, Italy

Location

University Hospital of Wales

Cardiff, CF14 4XW, United Kingdom

Location

MeSH Terms

Conditions

Ectodermal Dysplasia 1, Anhidrotic

Interventions

Fc-EDA

Condition Hierarchy (Ancestors)

Ectodermal Dysplasia; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Abnormalities; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• Kenneth Huttner, MD, PhD

  Edimer Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 12, 2013
• First Posted: November 25, 2013
• Study Start: March 1, 2014
• Primary Completion: March 1, 2025
• Study Completion: March 1, 2025
• Last Updated: September 5, 2017

Record last verified: 2017-08

Locations

FR (1); US (3); IT (1); GB (1); DE (1)