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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Edimer Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01564225
First received: March 23, 2012
Last updated: November 25, 2013
Last verified: November 2013
  Purpose

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.


Condition Intervention Phase
X-linked Hypohidrotic Ectodermal Dysplasia
Drug: EDI200
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Edimer Pharmaceuticals:

Primary Outcome Measures:
  • Number of participants with adverse events [ Time Frame: Up to 42 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Exploratory endpoint: change from baseline in biological activity [ Time Frame: Baseline and 42 days ] [ Designated as safety issue: No ]
    • 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


Enrollment: 6
Study Start Date: May 2012
Study Completion Date: April 2013
Primary Completion Date: April 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: EDI200 Drug: EDI200
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.

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.

  Eligibility

Ages Eligible for Study:   18 Years to 40 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01564225

Locations
United States, California
University of California, San Francisco
San Francisco, California, United States, 94143
United States, Ohio
Community Research
Cincinnati, Ohio, United States, 45226
Sponsors and Collaborators
Edimer Pharmaceuticals
Investigators
Principal Investigator: Ophir Klein, MD, PhD University of California, San Francisco
Principal Investigator: James Maynard, MD Community Research
  More Information

No publications provided

Responsible Party: Edimer Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01564225     History of Changes
Other Study ID Numbers: ECP-004
Study First Received: March 23, 2012
Last Updated: November 25, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Edimer Pharmaceuticals:
XLHED

Additional relevant MeSH terms:
Ectodermal Dysplasia
Ectodermal Dysplasia 1, Anhidrotic
Abnormalities, Multiple
Congenital Abnormalities
Skin Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on September 14, 2014