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

Stopped early · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Venous Malformation, Lymphatic Malformation, Venolymphatic Malformation

In brief

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.

Key facts

Study ID

NCT04409145

Run by

Venthera, Inc., a BridgeBio company

People needed

15

Starts

2020-10-01

Expected to finish

2022-04-13

Last updated by the study team

2022-08-04

Who can join

Age: 18 and older, up to 60. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Have signed the current approved informed consent form
• Have a clinically or phenotypically defined VM, LM, or mixed VLM affecting the skin
• Lesion genotyping confirms either PIK3CA or TEK mutations, known to be pathogenic
• Agrees to use contraception if of childbearing potential
• Be willing and able to comply with the protocol and be available for the entire study
• Be at least 18 to 60 years of age
• Lesion must be amenable to defining a contiguous study treatment area of 140 cm2

You may not qualify if…

• Lesion to be treated is on the face or involves mucosa
• Presence of ulcerations on the target-treatment lesion
• Known systemic hypersensitivity to the VT30 drug substance, its inactive ingredients, or the vehicle
• Uncontrolled diabetes mellitus
• Hyperlipidemia that is poorly controlled on current treatment
• Pregnant or nursing, planning to become pregnant, or planning to father a child during the study
• History of malignancy except successfully treated nonmetastatic cutaneous squamous cell or basal cell carcinoma and/or localized carcinoma in situ of the cervix
• Major surgery within 8 weeks of Screening, or a surgical, laser or other procedure involving the target lesion within 8 weeks of Screening, or planned to occur during the study
• Any other medical or personal condition that, in the opinion of the Investigator, may potentially compromise the safety or compliance of the subject, or may preclude the subject's successful completion of the clinical study
• Medically significant infection (eg, cellulitis or abscess, or a systemic infection) within 8 weeks of Screening
• Ongoing therapy with another topical treatment or any medication that inhibits PI3K, Akt pathway, or the mTOR pathway, or in the opinion of the Investigator, the subject requires systemic therapy for their vascular malformation condition
• Use of a biologic or systemic immunosuppressive agent within 3 months of Screening
• Systemic use of corticosteroids, within 30 days of Screening
• Treatment with a small molecule investigational product within 30 days of Screening, or with any investigational biologic products within 3 months of Screening
• Positive for hepatitis C antibody, hepatitis B surface antigen, hepatitis B core antibody, or human immunodeficiency virus
• Alanine transaminase or aspartate transaminase laboratory values in excess of 1.5X the upper limit of normal at Screening
• Hemoglobin A1c is >8%
• Any other clinically significant laboratory or testing abnormality that, in the opinion of the Investigator, might confound the study, interfere with the subject's ability to complete the study, or represent a meaningful safety risk upon study enrollment

Where it is running

• Phoenix Children's Hospital — Phoenix, Arizona, United States
• Arkansas Children's Hospital/UAMS — Little Rock, Arkansas, United States
• Stanford University Medical Center — Palo Alto, California, United States
• Dermatology Cosmetic Laser Medical Associates of La Jolla — San Diego, California, United States
• Children's Hospital of Colorado — Aurora, Colorado, United States
• Indiana University Health (Riley Children's Hospital and University Hospital) — Indianapolis, Indiana, United States
• Mayo Clinic — Rochester, Minnesota, United States
• Duke University — Raleigh, North Carolina, United States
• Cincinnatti Children's Hospital — Cincinnati, Ohio, United States
• University Hospitals- Cleveland Medical Center — Cleveland, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Texas Children's Hospital — Houston, Texas, United States
• Texas Dermatology and Laser Specialists — San Antonio, Texas, United States
• University of Virginia Department of Dermatology — Charlottesville, Virginia, United States
• University of Wisconsin-Madison — Madison, Wisconsin, United States

Full record on ClinicalTrials.gov

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