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

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Brief Title

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

Official Title

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

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
    

Detailed Description

      X-linked hypohidrotic ectodermal dysplasia (XLHED) is a disorder of ectoderm development in
      which sweat and other secretory gland hypoplasias predispose affected infants to serious and
      potentially life-threatening hyperthermia and pneumonia. Those XLHED patients who survive
      infancy face a host of ectoderm-related clinical conditions including failure to thrive,
      oligodontia and misshapen teeth, mid-face hypoplasia, eczema, chronic dry eyes, asthma,
      respiratory infections, sinusitis and chronic nosebleeds. XLHED is caused by inherited
      defects in the ectodysplasin gene (EDA, www.ncbi.nlm.nih.gov/omim) resulting in a deficiency
      of the ectoderm signaling protein EDA-A1. As is the general case with X-linked disorders,
      hemizygous XLHED males are more consistently and severely affected, while heterozygous XLHED
      females have a more variable phenotype.

      In normal development, EDA-A1 acts as an ectoderm signaling molecule that binds specifically
      to the EDA-A1 receptor (EDAR) triggering initiation and maturation of ectodermal appendages
      into sweat and other secretory glands, tooth buds and hair follicles. In the case of XLHED,
      EDA-A1 deficiency results in the absence or functional hypoplasia of the ectoderm appendages.
      There are no therapies currently available for XLHED that prevent or correct the underlying
      ectodermal abnormalities.

      EDI200 is a fully humanized EDA-A1 replacement molecule under development as a novel
      therapeutic for XLHED. EDI200 comprises the human IgG1 Fc domain linked to the human EDA-A1
      receptor-binding domain. On-target EDI200 activation of the EDA-A1/EDAR signaling pathway in
      vivo is evidenced by the remarkable phenotypic response in preclinical models. In
      XLHED-affected animals, EDA-A1 deficiency is corrected by a single course of EDI200 therapy,
      administered either prenatally (mice) or postnatally (newborn mice and dogs), resulting in a
      significant and sustained improvement in the health of the treated animals. Postnatal studies
      in both mice and dogs demonstrated a consistent and restricted window of efficacy. These
      results support the clinical development of EDI200 as a therapeutic to be administered to
      XLHED-affected patients in the neonatal period or earlier.

      ECP-002, a Phase 2, international, first-in-neonate EDI200 study is enrolling
      treatment-naïve, XLHED-affected male newborns in the first two weeks of life. All subjects
      will meet entry criteria including documentation of an EDA mutation associated with XLHED.
      Following Baseline evaluations, EDI200 dosing is initiated between day-of-life 2 and 14, with
      each study subject receiving a single course of study drug administered at 2 doses/week for a
      total of 5 doses. The treatment study protocol incorporates comprehensive safety,
      pharmacokinetic (PK), immunogenetic, and pharmacodynamic (PD)/efficacy evaluations continuing
      through age 6 months.

      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.
    


Study Type

Observational


Primary Outcome

Growth and development

Secondary Outcome

 Mortality

Condition

X-linked Hypohidrotic Ectodermal Dysplasia

Intervention

EDI200

Study Arms / Comparison Groups

 No treatment
Description:  This is a long-term follow-up study of subjects that received EDI200 as part of protocol ECP-002.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

10

Start Date

March 2014

Completion Date

March 2025

Primary Completion Date

March 2025

Eligibility 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
      

Gender

Male

Ages

6 Months - 1 Year

Accepts Healthy Volunteers

No

Contacts

Kenneth Huttner, MD, PhD, , 

Location Countries

France

Location Countries

France

Administrative Informations


NCT ID

NCT01992289

Organization ID

ECP-002e

Secondary IDs

1R01FD005099-01

Responsible Party

Sponsor

Study Sponsor

Edimer Pharmaceuticals


Study Sponsor

Kenneth Huttner, MD, PhD, Study Director, Edimer Pharmaceuticals


Verification Date

August 2017