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

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Brief Title

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

Official Title

A Phase 2 Open-label, Dose-escalation Study to Evaluate the Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200, an EDA-A1 Replacement Protein, Administered to Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)

Brief 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.
    

Detailed Description

      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 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. This dosing regimen mirrors that used to
      enhance efficacy in the dog XLHED model, considered to be most relevant to the clinical study
      design. 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. The sponsor anticipates enrollment and
      dosing of 6-10 subjects over a 12-18 month period, 3-5 subjects per cohort.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Incidence and severity of adverse events

Secondary Outcome

 To assess the pharmacodynamics/efficacy (growth and development) of EDI200

Condition

X-Linked Hypohidrotic Ectodermal Dysplasia

Intervention

EDI200

Study Arms / Comparison Groups

 EDI200, 3mg/kg
Description:  Five doses of EDI200 given at 3 mg/kg twice weekly

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

6

Start Date

April 2013

Completion Date

December 2015

Primary Completion Date

December 2015

Eligibility Criteria

        Inclusion Criteria:

        Subjects for study drug administration must meet all of the following criteria to be
        enrolled:

          1. Male with genetic confirmation of an XLHED diagnosis.

          2. Subject must be at least 48 hours age and no older than 14 days.

          3. Subject will have reached term (defined as 37 weeks gestation or older) prior to
             receiving first dose study drug.

          4. Written informed consent of both parents (if reasonably available) must be obtained
             for treatment of their XLHED-affected male infant.

          5. Neither mother nor the XLHED-affected male infant known to have received an
             investigational study drug in the 9 months prior to study subject enrollment in this
             study.

          6. No major medical issues that the PI considers a contraindication to participation.

        Siblings of subjects receiving study drug must meet all of the following criteria to be
        enrolled in the natural history sub-study (no age limit involved):

          1. Provide written informed consent/assent.

          2. A full or half-sibling of a study subject where the study subject has received at
             least one dose of study drug in the Phase 2 XLHED Neonate Study and has not yet
             completed the study.

          3. No major medical issues that the investigator considers contraindications to
             participation.

        Exclusion Criteria:

        Subjects for study drug administration who meet any of the following criteria cannot 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.

        Siblings of subjects receiving study drug who meet any of the following criteria cannot be
        enrolled in the natural history sub-study:

          1. Known hypersensitivity to pilocarpine or pilocarpine-like muscarinic agonists.

          2. Known hypersensitivity to lidocaine or lidocaine-like agents.

          3. Presence of pacemaker.

          4. Subjects who are not able or are not willing to comply with the procedures of this
             protocol.

          5. Subject has a condition, which in the opinion of the investigator would not allow for
             safe conduct of the study.
      

Gender

Male

Ages

N/A - 14 Days

Accepts Healthy Volunteers

No

Contacts

Kenneth Huttner, MD, PhD, , 

Location Countries

France

Location Countries

France

Administrative Informations


NCT ID

NCT01775462

Organization ID

ECP-002


Responsible Party

Sponsor

Study Sponsor

Edimer Pharmaceuticals


Study Sponsor

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


Verification Date

January 2016