First in Human Trial of Topical VT30 in Pts With Venous/Lymphatic Malformations Assoc With PIK3CA or TEK Gene Mutations
Status:
Recruiting
Trial end date:
2022-06-01
Target enrollment:
Participant gender:
Summary
VT30-101 is a 2-part first-in-human trial of topically administered VT30 to subjects with
cutaneous venous malformations, lymphatic malformations, or mixed venolymphatic malformations
associated with PIK3CA or TEK mutations.
Part 1 is a 4-week treatment, open-label, 4-sequence, escalating repeat-application cohort
study, with intra-subject and inter-cohort dose escalation.
Part 2 is a 12-week treatment, randomized, placebo-controlled, double-blind, safety and
exploratory efficacy study. Part 2 will be initiated only after the successful completion of
Part 1 with results that demonstrate the general safety and tolerability of topically applied
VT30. Up to 12 subjects who complete Part 1 may be enrolled into Part 2 of the study.
The primary objective is to evaluate the safety and tolerability of VT30. The study will also
determine the dose and regimen of VT30 to be carried into Part 2 of the protocol. Other aims
include documenting plasma drug levels of VT30 and VT10 and, on an exploratory basis,
examining pharmacologic target engagement and change in potential efficacy readouts.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Venthera, Inc. Venthera, Inc., a BridgeBio company