Status:
TERMINATED
First in Human Trial of Topical VT30 in Pts With Venous/Lymphatic Malformations Assoc With PIK3CA or TEK Gene Mutations
Lead Sponsor:
Venthera, Inc., a BridgeBio company
Conditions:
Venous Malformation
Lymphatic Malformation
Eligibility:
All Genders
18-60 years
Phase:
PHASE1
PHASE2
Brief Summary
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 ...
Detailed Description
VT30-101 is designed as a Phase 1/2, first-in-human study of topically administered VT30 to subjects with cutaneous venous malformations (VMs), lymphatic malformations (LMs), or mixed venolymphatic ma...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
October 1 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 13 2022
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT04409145
Start Date
October 1 2020
End Date
April 13 2022
Last Update
August 4 2022
Active Locations (15)
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1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
2
Arkansas Children's Hospital/UAMS
Little Rock, Arkansas, United States, 72202
3
Stanford University Medical Center
Palo Alto, California, United States, 94304
4
Dermatology Cosmetic Laser Medical Associates of La Jolla
San Diego, California, United States, 92121