A Study to Evaluate Topical VT30 to Treat Patients with Venous/Lymphatic Malformations Associated with PIK3CA or TEK Gene Mutations

Overview

About this study

The primary purpose of this two-part study is to evaluate the safety and tolerability of VT30.  

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 effectiveness 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. 

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • 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.
  • Willing and able to comply with the protocol and be available for the entire study.
  • Between 18 and 60 years of age.
  • Lesion must be amenable to defining a contiguous study treatment area of 140 cm^2.

Exclusion Criteria:

  • 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 (e.g., 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 1.5 x 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.

Eligibility last updated 10/12/21. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Megha Tollefson, M.D.

Open for enrollment

Contact information:

Jessica Hafeman

(507) 422-6964

Hafeman.Jessica@mayo.edu

More information

Publications

Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available

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CLS-20490342

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