First in Human Trial of Topical VT30 in Pts With Venous/Lymphatic Malformations Assoc With PIK3CA or TEK Gene Mutations
Trial ID or NCT#
Status
NOT RECRUITING
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
Eligibility Criteria
- 1. Have signed the current approved informed consent form2. Have a clinically or phenotypically defined VM, LM, or mixed VLM affecting the skin3. Lesion genotyping confirms either PIK3CA or TEK mutations, known to be pathogenic4. Agrees to use contraception if of childbearing potential5. Be willing and able to comply with the protocol and be available for the entire study6. Be at least 18 to 60 years of age7. Lesion must be amenable to defining a contiguous study treatment area of 140 cm2
- 1. Lesion to be treated is on the face or involves mucosa2. Presence of ulcerations on the target-treatment lesion3. Known systemic hypersensitivity to the VT30 drug substance, its inactive ingredients, or the vehicle4. Uncontrolled diabetes mellitus5. Hyperlipidemia that is poorly controlled on current treatment6. Pregnant or nursing, planning to become pregnant, or planning to father a child during the study7. History of malignancy except successfully treated nonmetastatic cutaneous squamous cell or basal cell carcinoma and/or localized carcinoma in situ of the cervix8. 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 study9. 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 study10. Medically significant infection (eg, cellulitis or abscess, or a systemic infection) within 8 weeks of Screening11. 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 condition12. Use of a biologic or systemic immunosuppressive agent within 3 months of Screening13. Systemic use of corticosteroids, within 30 days of Screening14. Treatment with a small molecule investigational product within 30 days of Screening, or with any investigational biologic products within 3 months of Screening15. Positive for hepatitis C antibody, hepatitis B surface antigen, hepatitis B core antibody, or human immunodeficiency virus16. Alanine transaminase or aspartate transaminase laboratory values in excess of 1.5X the upper limit of normal at Screening17. 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
Investigator(s)
Contact us to find out if this trial is right for you.
Contact
Pediatric Dermatology Research
650-723-0636
View on ClinicalTrials.gov
