NCT04409145

First in Human Trial of Topical VT30 in Pts With Venous/Lymphatic Malformations Assoc With PIK3CA or TEK Gene Mutations

Terminated Phase 1, PHASE2 Last updated 4 August 2022

What this trial tests

Phase 1, PHASE2 trial testing VT30 in Venous Malformation in 15 participants. Terminated before completion.

Timeline

1 October 2020

Primary endpoint
13 April 2022

13 April 2022

Quick facts

Lead sponsor	Venthera, Inc., a BridgeBio company
Phase	Phase 1, PHASE2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	15
Start date	1 October 2020
Primary completion	13 April 2022
Estimated completion	13 April 2022
Sites	15 locations across United States

Drugs / interventions tested

• VT30 — full drug profile →

Conditions studied

• Venous Malformation — all drugs for Venous Malformation →
• Lymphatic Malformation — all drugs for Lymphatic Malformation →
• Venolymphatic Malformation — all drugs for Venolymphatic Malformation →

Sponsor

Venthera, Inc., a BridgeBio company — full company profile →

Who can join

Adults 18 to 60, any sex, with Venous Malformation or Lymphatic Malformation. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Genetic and Molecular Determinants of Lymphatic Malformations: Potential Targets for Therapy.
   Lee SY, Loll EG, Hassan AS, Cheng M, et al · · 2022 · cited 17× · PMID 35225964 · DOI 10.3390/jdb10010011
2. Whole mount of adult ear skin as a model to study vascular malformations.
   Decker-Rockefeller B, Li Q, Pumiglia K. · · 2023 · cited 2× · PMID 37682010 · DOI 10.1002/ame2.12343
3. PI3K/AKT/mTOR axis in vascular malformations: from molecular insights to targeted clinical trials.
   Zheng YY, Hua C, Lin XX. · · 2025 · cited 1× · PMID 41430313 · DOI 10.1186/s13023-025-04115-2