A Phase 1, Open-label, Multicenter, Safety and Pharmacokinetic Study of EDI200
Status:
Completed
Trial end date:
2013-04-01
Target enrollment:
Participant gender:
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.