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First in Human Trial of Topical VT30 in Pts With Venous/Lymphatic Malformations Assoc With PIK3CA or TEK Gene Mutations
Not Recruiting
Trial ID: NCT04409145
Purpose
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.
Official Title
Open-Label, Intra Subject, Dose Escalation (Part 1) Followed by Randomized, Double Blind, Placebo Controlled (Part 2) Trial of Topical VT30 in Pts With Venous, Lymphatic or Mixed Malformations Associated With PIK3CA or TEK Genetic Mutations
Stanford Investigator(s)
Joyce Teng, MD, PhD
Professor of Dermatology and, by courtesy, of Pediatrics
Eligibility
Inclusion Criteria:
1. Have signed the current approved informed consent form
2. Have a clinically or phenotypically defined VM, LM, or mixed VLM affecting the skin
3. Lesion genotyping confirms either PIK3CA or TEK mutations, known to be pathogenic
4. Agrees to use contraception if of childbearing potential
5. Be willing and able to comply with the protocol and be available for the entire study
6. Be at least 18 to 60 years of age
7. Lesion must be amenable to defining a contiguous study treatment area of 140 cm2
Exclusion Criteria:
1. Lesion to be treated is on the face or involves mucosa
2. Presence of ulcerations on the target-treatment lesion
3. Known systemic hypersensitivity to the VT30 drug substance, its inactive ingredients,
or the vehicle
4. Uncontrolled diabetes mellitus
5. Hyperlipidemia that is poorly controlled on current treatment
6. Pregnant or nursing, planning to become pregnant, or planning to father a child during
the study
7. History of malignancy except successfully treated nonmetastatic cutaneous squamous
cell or basal cell carcinoma and/or localized carcinoma in situ of the cervix
8. 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
9. 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
10. Medically significant infection (eg, cellulitis or abscess, or a systemic infection)
within 8 weeks of Screening
11. 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
12. Use of a biologic or systemic immunosuppressive agent within 3 months of Screening
13. Systemic use of corticosteroids, within 30 days of Screening
14. Treatment with a small molecule investigational product within 30 days of Screening,
or with any investigational biologic products within 3 months of Screening
15. Positive for hepatitis C antibody, hepatitis B surface antigen, hepatitis B core
antibody, or human immunodeficiency virus
16. Alanine transaminase or aspartate transaminase laboratory values in excess of 1.5X the
upper limit of normal at Screening
17. Hemoglobin A1c is >8%
18. 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
Intervention(s):
drug: VT30
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Pediatric Dermatology Research
650-723-0636