NCT01564225

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

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Edimer Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 40 Years
• Healthy volunteers: Not accepted

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.

Conditions

• X-linked Hypohidrotic Ectodermal Dysplasia

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2012-05-01
• Primary completion: 2013-04-01
• Completion: 2013-04-01
• First posted: 2012-03-27
• Last updated: 2013-11-27

Locations

2 sites across 1 country: United States