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

Primary Purpose

X-linked Hypohidrotic Ectodermal Dysplasia

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
EDI200
Sponsored by
Edimer Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for X-linked Hypohidrotic Ectodermal Dysplasia focused on measuring XLHED

Eligibility Criteria

18 Years - 40 Years (Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of California, San Francisco
  • Community Research

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

EDI200

Arm Description

Outcomes

Primary Outcome Measures

Number of participants with adverse events

Secondary Outcome Measures

Exploratory endpoint: change from baseline in biological activity
Hair number and growth properties Pulmonary function and eNO levels Sweat duct density Sweat rate Saliva quantitation Tearing and dry eye evaluation Skin biopsy for expression profile

Full Information

First Posted
March 23, 2012
Last Updated
November 25, 2013
Sponsor
Edimer Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT01564225
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
Study Type
Interventional

2. Study Status

Record Verification Date
November 2013
Overall Recruitment Status
Completed
Study Start Date
May 2012 (undefined)
Primary Completion Date
April 2013 (Actual)
Study Completion Date
April 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Edimer Pharmaceuticals

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
X-linked Hypohidrotic Ectodermal Dysplasia
Keywords
XLHED

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)

8. Arms, Groups, and Interventions

Arm Title
EDI200
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
EDI200
Intervention Description
Cohort 1 will be dosed at 3 mg/kg/dose. Cohort 2 will be dosed at 10 mg/kg/dose. Both cohorts will receive 2 doses/week for a total of 5 doses.
Primary Outcome Measure Information:
Title
Number of participants with adverse events
Time Frame
Up to 42 days
Secondary Outcome Measure Information:
Title
Exploratory endpoint: change from baseline in biological activity
Description
Hair number and growth properties Pulmonary function and eNO levels Sweat duct density Sweat rate Saliva quantitation Tearing and dry eye evaluation Skin biopsy for expression profile
Time Frame
Baseline and 42 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
40 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Males and females of child-bearing age, age 18-40 years 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) 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 No major medical issues that the investigator considers to be a contraindication of participation No scalp shaving in the month prior to first dose (males only) 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. No treatment with an investigational drug within the last three months Signed written informed consent Exclusion Criteria: 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 Known history of hepatitis B surface antigen (HBsAg) or hepatitis C (HCV) antibody Known history of HIV infection Known hypersensitivity to pilocarpine or pilocarpine-like muscarinic agonists Known hypersensitivity to lidocaine or lidocaine-like agents Presence of pacemakers Subjects who are not able or are not willing to comply with the procedures of this protocol Subject has a condition which in the opinion of the investigator would not allow for safe conduct of the study
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ophir Klein, MD, PhD
Organizational Affiliation
University of California, San Francisco
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
James Maynard, MD
Organizational Affiliation
Community Research
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of California, San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Community Research
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45226
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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