First in Human Trial of Topical VT30 in Pts With Venous/Lymphatic Malformations Assoc With PIK3CA or TEK Gene Mutations
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ClinicalTrials.gov Identifier: NCT04409145 |
Recruitment Status :
Recruiting
First Posted : June 1, 2020
Last Update Posted : August 27, 2021
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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.
Condition or disease | Intervention/treatment | Phase |
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Venous Malformation Lymphatic Malformation Venolymphatic Malformation | Drug: VT30 | Phase 1 Phase 2 |

Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 51 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Intervention Model Description: | Part 1: Open-label, 4-sequence, escalating repeat-application cohort study, with intra-subject and inter-cohort dose escalation to determine safety and tolerability, and maximum feasible dose/maximum tolerable dose Part 2: Randomized, placebo-controlled, double-blind, safety and exploratory efficacy study |
Masking: | None (Open Label) |
Masking Description: | Part 1: Open label Part 2: Double blind |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | October 1, 2020 |
Estimated Primary Completion Date : | June 2022 |
Estimated Study Completion Date : | June 2022 |

Arm | Intervention/treatment |
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Experimental: VT30
VT30 is a PI3K-inhibitor prodrug, formulated as a topical gel and dispensed from a metered dose pump; administration is once or twice daily, applied to target-treatment area(s) on the skin. One pump action dispenses 250 µL of gel, intended to treat an area of 140 cm2.
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Drug: VT30
VT30 gel is intended as a topical treatment of cutaneous VMs, LMs, or VLMs that driven by inappropriate PI3K activation. In the skin, VT30 is rapidly metabolized to VT10, an active drug form, and is intended to sufficiently permeate the stratum corneum and achieve target engagement. It is expected that VT30 will lead to amelioration of the signs and symptoms of cutaneous VMs, LMs and/or VLMs. |
- Evaluation of safety and tolerability [ Time Frame: From pre-treatment to 4 weeks of treatment ]Composite of adverse events and changes in physical exam findings, vital signs, lab tests, and electrocardiogram evaluations
- Maximum feasible dose / maximum tolerable dose [ Time Frame: From pre-treatment to 4 weeks ]The MTD or MFD strength will be determined based on results from Part 1, and will inform the dose strength to be used in Part 2
- Tissue and serum drug levels [ Time Frame: From pre-treatment to 4 weeks ]Plasma levels of VT30 and VT10 will be assessed following topical administration of VT30 to determine level of systemic exposure
- Maximum tissue concentration of study drug [ Time Frame: From pre-treatment to 4 weeks ]As assessed by treated lesion tissue levels of phosphoproteins, as an indicator of local target engagement
- Changes in Pain [ Time Frame: From pre-treatment to 4 weeks ]Assessed by subjects' self-reporting their pain, related to the treated lesion, on a Numerical Rating Scale
- Changes in lesion [ Time Frame: From pre-treatment to 4 weeks ]Assessed by change in appearance of the treated lesion
- Changes in management of lesion bleeding, oozing, or discharge [ Time Frame: From pre-treatment to 4 weeks ]Assessed by subject-reported difficulty managing bleeding, oozing, or discharge from the treated lesion

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Ages Eligible for Study: | 18 Years to 60 Years (Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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
- 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
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 (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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04409145
Contact: Lisa Pugliese | 650-825-4080 | lp@venthera.com |
United States, Arizona | |
Phoenix Children's Hospital | Recruiting |
Phoenix, Arizona, United States, 85016 | |
Contact: Kellie Badger, RN 602-933-2053 kbadger@phoenixchildrens.com | |
Principal Investigator: Harper Price, MD | |
United States, Arkansas | |
Arkansas Children's Hospital/UAMS | Recruiting |
Little Rock, Arkansas, United States, 72202 | |
Contact: D'Ann Pierce 501-364-4440 piercecarold@uams.edu | |
Principal Investigator: Shelley Crary,, MD | |
United States, California | |
Stanford University Medical Center | Recruiting |
Palo Alto, California, United States, 94304 | |
Contact: Elidia Tafoya, RN 650-724-1982 etafoya@stanford.edu | |
Principal Investigator: Joyce Teng, MD | |
Dermatology Cosmetic Laser Medical Associates of La Jolla | Recruiting |
San Diego, California, United States, 92121 | |
Contact: Leslie Aguilar 858-657-1004 laguilar@clderm.com | |
Principal Investigator: - Mitchel Goldman, MD | |
United States, Colorado | |
Children's Hospital of Colorado | Recruiting |
Aurora, Colorado, United States, 80045 | |
Contact: Wendy Moore, BSN 720-777-6353 Wendy.moore2@childrenscolorado.org | |
Principal Investigator: Taizo Nakano, MD | |
United States, Indiana | |
Indiana University Health (Riley Children's Hospital and University Hospital) | Recruiting |
Indianapolis, Indiana, United States, 46202 | |
Contact: Flossy Lincoln, RN 317-274-8750 fnjinimb@iupui.edu | |
Principal Investigator: Anita Haggstrom,, MD | |
United States, Minnesota | |
Mayo Clinic | Recruiting |
Rochester, Minnesota, United States, 55905 | |
Contact: Alison Gilbertson 507-422-6964 Langer.alison@mayo.edu | |
Principal Investigator: Megha Tollefson, MD Tollefson, MD | |
United States, North Carolina | |
Duke University | Recruiting |
Raleigh, North Carolina, United States, 27513 | |
Contact: Hai Hai Huang, RN 919-684-3401 h.hai@duke.edu | |
Principal Investigator: Kristy Pahl, MD | |
United States, Ohio | |
Cincinnatti Children's Hospital | Recruiting |
Cincinnati, Ohio, United States, 45229 | |
Contact: Megan Metcalf, RN 513-803-2606 Megan.Metcalf@cchmc.org | |
Principal Investigator: Adrienne Hammill, MD | |
University Hospitals- Cleveland Medical Center | Recruiting |
Cleveland, Ohio, United States, 44106 | |
Contact: Amanda Rivera, RN 216-844-4908 Amanda.Rivera@UHhospitals.org | |
Principal Investigator: Irina Pateva, MD | |
United States, Pennsylvania | |
Children's Hospital of Philadelphia | Recruiting |
Philadelphia, Pennsylvania, United States, 19104 | |
Contact: Meghan Martin, MS, CCRP 215-590-6625 KILLENM1@chop.edu | |
Contact: Bede Nriagu, MBBS, MPH 800-879-2467 NRIAGUB@chop.edu | |
Principal Investigator: Kristin Snyder, MD | |
United States, Texas | |
Texas Children's Hospital | Recruiting |
Houston, Texas, United States, 77030 | |
Contact: Stanley Oseghale, RN 832-822-3565 sxosegha@texaschildrens.org | |
Principal Investigator: Denise Metry, MD | |
Texas Dermatology and Laser Specialists | Recruiting |
San Antonio, Texas, United States, 78218 | |
Contact: Jenna Encina Encina 210-852-2779 jenna@texasdls.com | |
Principal Investigator: John Browning, MD | |
United States, Virginia | |
University of Virginia Department of Dermatology | Recruiting |
Charlottesville, Virginia, United States, 44106 | |
Contact: Ashton Leone, RN 434-243-5737 AML7Q@virginia.edu | |
Principal Investigator: Richard Flowers, MF | |
United States, Wisconsin | |
University of Wisconsin-Madison | Recruiting |
Madison, Wisconsin, United States, 53715 | |
Contact: Christina Sheehan 608-287-2006 csheehan@dermatology.wisc.edu | |
Principal Investigator: Beth Ann Drolet |
Study Director: | Ken Truitt, MD | Venthera, Inc., a BridgeBio company |
Responsible Party: | Venthera, Inc., a BridgeBio company |
ClinicalTrials.gov Identifier: | NCT04409145 |
Other Study ID Numbers: |
VT30-101 |
First Posted: | June 1, 2020 Key Record Dates |
Last Update Posted: | August 27, 2021 |
Last Verified: | July 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Lymphangioma Lymphatic Abnormalities Congenital Abnormalities Lymphatic Vessel Tumors |
Neoplasms by Histologic Type Neoplasms Lymphatic Diseases |