A Phase 1, Open-label, Multicenter, Safety and Pharmacokinetic Study of EDI200

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Brief Title

A Phase 1, Open-label, Multicenter, Safety and Pharmacokinetic Study of EDI200

Official Title

A Phase 1, Open-label, Multicenter, Safety and Pharmacokinetic Study of EDI200, an Ectodysplasin-A1 Replacement Molecule, in X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) Adults

Brief Summary

      Following discussions with the FDA, a Phase 1 safety study is being initiated in X-Linked
      Hypohidrotic Ectodermal Dysplasia (XLHED)-affected adults to develop safety and exposure data
      for EDI200 in anticipation of dosing XLHED-affected neonates. Selecting XLHED-affected adults
      for this study provides a genetic match and biologic relevance to XLHED-affected neonates.
      Both males and females will be enrolled, providing safety experience with EDI200 that will
      inform the planned neonate study as well as supportive data for potential future trials of
      antenatal EDI200 administration.
    

Detailed Description

      The Phase 1 study will enroll two cohorts of 3 XLHED-affected adults each, for a total of 6
      subjects. The size and scope of the study design are consistent with a drug development
      program in an ultra-rare disease and supported by the absence of safety concerns in a GLP
      non-human primate toxicology study. All subjects must meet entry criteria including
      documentation of an EDA mutation. Mirroring the conditions of use anticipated for the
      XLHED-affected newborn study, the adult cohort study incorporates a multiple-dose regimen
      associated with maximal efficacy in the newborn canine model. Primary outcome measures will
      be safety, tolerability, immunogenicity and pharmacokinetics (PK). While current data does
      not support a likelihood of clinical benefit for the XLHED-affected adult subjects, assays of
      pharmacodynamic/biologic activity are incorporated into the study design as exploratory
      objectives.
    

Study Phase

Phase 1

Study Type

Interventional


Primary Outcome

Number of participants with adverse events

Secondary Outcome

 Exploratory endpoint: change from baseline in biological activity

Condition

X-linked Hypohidrotic Ectodermal Dysplasia

Intervention

EDI200

Study Arms / Comparison Groups

 EDI200
Description:  

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

May 2012

Completion Date

April 2013

Primary Completion Date

April 2013

Eligibility Criteria

        Inclusion Criteria:

          1. Males and females of child-bearing age, age 18-40 years

          2. Weight between 45 and 90 kg and a body mass index (BMI) from 18 to 29 kg/m2
             (calculated using the following formula: weight in kilograms/(height in meters)2)

          3. Both males and females must be documented (via genetic testing) to carry an EDA
             mutation associated with XLHED; or have the clinical signs and symptoms associated
             with HED and/or a family history of HED and provide a blood sample to be sent for
             genetic testing that confirms an EDA mutation associated with XLHED

          4. No major medical issues that the investigator considers to be a contraindication of
             participation

          5. No scalp shaving in the month prior to first dose (males only)

          6. Women must use a "highly effective" method of contraception throughout the trial.
             Highly effective methods of birth control are defined as those, alone or in
             combination, which result in a low failure rate (i.e. less than 1% per year) when used
             consistently and correctly. These methods include implants, injectables, oral
             contraceptives, some intrauterine contraceptive devices, sexual abstinence, tubal
             ligation or a vasectomized partner.

          7. No treatment with an investigational drug within the last three months

          8. Signed written informed consent

        Exclusion Criteria:

          1. Women who are pregnant (confirmed via urine pregnancy test) or breastfeeding at
             screening or planning to become pregnant at any time during the study period

          2. Known history of hepatitis B surface antigen (HBsAg) or hepatitis C (HCV) antibody

          3. Known history of HIV infection

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

          5. Known hypersensitivity to lidocaine or lidocaine-like agents

          6. Presence of pacemakers

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

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

Gender

All

Ages

18 Years - 40 Years

Accepts Healthy Volunteers

No

Contacts

Ophir Klein, MD, PhD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01564225

Organization ID

ECP-004


Responsible Party

Sponsor

Study Sponsor

Edimer Pharmaceuticals


Study Sponsor

Ophir Klein, MD, PhD, Principal Investigator, University of California, San Francisco


Verification Date

November 2013