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Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)

NCT01775462 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2016-01-20

No results posted yet for this study

Summary

This Phase 2 first-in-neonate EDI200 study will enroll treatment-naïve, XLHED-affected male newborns in the first two weeks of life. All subjects will meet entry criteria including documentation of an Ectodysplasin (EDA) mutation associated with XLHED. Following Baseline evaluations, EDI200 dosing will be initiated between day-of-life 2 and 14, with each study subject receiving 2 doses/week for a total of 5 doses. The study will enroll subjects in two cohorts with subjects in cohort 1 dosed at 3 mg/kg/dose, associated with partial efficacy, and cohort 2 dosed at 10 mg/kg/dose where enhanced efficacy was demonstrated in the most relevant preclinical model. Given the challenge of identifying families where the subject is yet to be born, it is expected that cohort size and time for recruitment will be variable.

Conditions

  • X-Linked Hypohidrotic Ectodermal Dysplasia

Interventions

DRUG

EDI200

3 or 10 mg/kg of EDI200

Sponsors & Collaborators

  • Edimer Pharmaceuticals

    lead INDUSTRY

Principal Investigators

  • Kenneth Huttner, MD, PhD · Edimer Pharmaceuticals

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
48 Hours
Max Age
14 Days
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-04-30
Primary Completion
2015-12-31
Completion
2015-12-31

Countries

  • United States
  • France
  • Germany
  • Italy
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01775462 on ClinicalTrials.gov