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

• Register: ClinicalTrials.gov
• Last refreshed on: 7 January 2013
• Main ID: NCT01564225
• Date of registration: 23/03/2012
• Primary sponsor: Edimer Pharmaceuticals
• Public title: A Phase 1, Open-label, Multicenter, Safety and Pharmacokinetic Study of EDI200
• Scientific 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
• Date of first enrolment: May 2012
• Target sample size: 6
• Recruitment status: Recruiting
• URL: http://clinicaltrials.gov/show/NCT01564225
• Study type: Interventional
• Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Countries of recruitment

United States

Contacts

• Name: Ramsey Johnson

• Name: James Maynard, MD
• Affiliation: Community Research

• Name: Ophir Klein, MD, PhD
• Affiliation: University of California, San Francisco

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 40 Years
Gender: Both

Health Condition(s) or Problem(s) studied

X-linked Hypohidrotic Ectodermal Dysplasia

Intervention(s)

Drug: EDI200

Primary Outcome(s)

Number of participants with adverse events [Time Frame: Up to 42 days]

Secondary Outcome(s)

Exploratory endpoint: change from baseline in biological activity [Time Frame: Baseline and 42 days]

Secondary ID(s)

ECP-004

Source(s) of Monetary Support

Please refer to primary and secondary sponsors