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

NCT01564225 | Completed Phase 1 DMC

• 1 other identifier: ECP-004
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 2

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.

Conditions

X-linked Hypohidrotic Ectodermal Dysplasia

Keywords

XLHED

Outcome Measures

Primary Outcomes (1)

• Number of participants with adverse events

  Up to 42 days

Secondary Outcomes (1)

• Exploratory endpoint: change from baseline in biological activity

  Baseline and 42 days

Study Arms (1)

EDI200

EXPERIMENTAL

Drug: EDI200

Interventions

EDI200 DRUG

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.

EDI200

Eligibility Criteria

• Age: 18 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Edimer Pharmaceuticals: lead

Study Sites (2)

University of California, San Francisco

San Francisco, California, 94143, United States

Location

Community Research

Cincinnati, Ohio, 45226, United States

Location

MeSH Terms

Conditions

Ectodermal Dysplasia 1, Anhidrotic

Interventions

Fc-EDA

Condition Hierarchy (Ancestors)

Ectodermal Dysplasia; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Abnormalities; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• Ophir Klein, MD, PhD

  University of California, San Francisco

  PRINCIPAL INVESTIGATOR

• James Maynard, MD

  Community Research

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 23, 2012
• First Posted: March 27, 2012
• Study Start: May 1, 2012
• Primary Completion: April 1, 2013
• Study Completion: April 1, 2013
• Last Updated: November 27, 2013

Record last verified: 2013-11

Locations

US (2)