Proof of Concept Study to Access Superficial Basal Cell Carcinoma in Adults

NCT05713760 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: AICR-BCC-01
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Phase 2, Single-Arm, Open-Label, Single Center, Proof of Concept Study to Assess Efficacy and Safety of Tirbanibulin Ointment 1% in Adult Subjects with superficial basal cell carcinoma (sBCC) on the neck, trunk, or extremities (excluding, axilla, anogenital, and face/scalp).

Conditions

Superficial Basal Cell Carcinoma

Outcome Measures

Primary Outcomes (1)

• Complete clearance rate of sBCC lesion

  The primary efficacy endpoint will be the percent of subjects with both clinical and pathological clearance of sBCC lesion for subjects who completed 1 course of treatment and subjects who completed 2 courses of treatment.

  26 days for subjects who completed 1 course of treatment and 51 days for subjects who completed 2 courses of treatment

Study Arms (2)

5 Day treatment course 1 with Tirbanibulin Ointment 1%

EXPERIMENTAL

10 Subjects will be given one study kit containing single-dose packets of Tirbanibulin Ointment 1% and instructed to topically apply to the treatment area once daily for 5 consecutive days.

Drug: Tirbanibulin Ointment 1%

5 Day treatment course 2 with Tirbanibulin Ointment 1%

EXPERIMENTAL

Subjects with unresolved lesion will be given an additional study kit containing single-dose packets of Tirbanibulin Ointment 1% and instructed to topically apply to the treatment area once daily for 5 consecutive days.

Drug: Tirbanibulin Ointment 1%

Interventions

Tirbanibulin Ointment 1% DRUG

Klisyri® ointment is a microtubule inhibitor for topical use and is currently on the market for the treatment of actinic keratosis of the face or scalp. The chemical name of tirbanibulin is N-benzyl-2-(5-(4-(2- morpholinoethoxy)phenyl)pyridin-2-yl) acetamide. Tirbanibulin ointment 1% contains 10 mg tirbanibulin per gram of white to off-white ointment containing mono- and di-glycerides and propylene glycol and is supplied in packets containing 250 mg of tirbanibulin ointment 1%. Each packet includes single-dose packets (2.5 mg tirbanibulin in 250 mg) to be applied to evenly cover up to 25 cm² of the treatment field once daily for 5 consecutive days using 1 single-dose packet per application.

Also known as: Klisyri (tirbanibulin ointment 1%)

5 Day treatment course 1 with Tirbanibulin Ointment 1%; 5 Day treatment course 2 with Tirbanibulin Ointment 1%

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males and females ≥ 18 years old.
• Willingness and ability to sign the informed consent form and Health Insurance Portability and Accountability Act (HIPAA) form. A study-specific informed consent form and a HIPAA form must be obtained in writing for all subjects prior to starting any study procedures.
• Present of 1 naïve sBCC on the neck, trunk, or extremities (excluding, axilla, anogenital, and face/scalp) that:
• is between 6 and 20mm in greatest diameter
• \>2cm from a hairline
• Must lack any evidence of aggressive growth patterns (e.g., severe squamous metaplasia, infiltrative/desmoplastic features, or basosquamous features)
• sBCC assessed as non-naïve (e.g., previously treated, or recurrent) or non-eligible by biopsy and at least 5cm away from an eligible lesion should be excised or treated with Electrodesiccation and Curettage (ED\&C) in a timely manner.
• Free of significant physical abnormalities (e.g., tattoos, dermatoses) within the potential treatment area plus a 5cm radius surrounding the lesion as they may interfere with examination or final evaluation, in the opinion of the investigator.
• Willingness to stop the use of moisturizers, sunscreens, and any other cosmetics within the treatment area plus a 5cm radius from screening until final the visit.
• Willingness to abstain from sunbathing or the use of artificial tanning from screening until the final visit. Subjects with a sunburn in the treatment area cannot be included until fully recovered.
• Subjects must be healthy or have a medical condition that is stable in the opinion of the investigator.
• Females must be postmenopausal (\>45 years of age with at least 12 months of amenorrhea), surgically sterile (by hysterectomy, bilateral oophorectomy, or tubal ligation); or, if of childbearing potential, must be using highly effective contraception for at least 30 days or 1 menstrual cycle, whichever is longer, prior to study treatment and must agree to continue to use highly effective contraception for at least 30 days following their last dose of study treatment. Highly effective contraception includes oral hormonal contraceptives, hormonal contraceptive implant, injection or patch, intrauterine device, or complete abstinence from sexual intercourse.
• Sexually active males who have not had a vasectomy, and whose partner is reproductively capable, must agree to use barrier contraception from Screening through 90 days after their last dose of study treatment.

You may not qualify if:

• Location of the treatment area is:
• On face, scalp, anogenital, axillae
• Within 5 cm of an incompletely healed wound
• Within 2 cm of hairline
• Anticipated need for in-patient hospitalization or in-patient surgery from Day 1 to Day 57.
• Treatment with 5-fluorouracil (5-FU), imiquimod, ingenol mebutate, diclofenac, photodynamic therapy, or other treatments for Actinic Keratosis (AK) within the treatment area or within 2 cm of the treatment area, within 8 weeks prior to the Screening visit.
• Use of the following therapies and/or medications within 2 weeks prior to the Screening visit:
• Cosmetic or therapeutic procedures (e.g., use of liquid nitrogen, surgical excision, curettage, dermabrasion, medium or greater depth chemical peel, laser resurfacing) within the treatment area or within 2 cm of the selected treatment area
• Acid-containing therapeutic products (e.g., salicylic acid or fruit acids, such as alpha and beta-hydroxyl acids and glycolic acids), topical retinoids, or light chemical peels within the treatment area or within 2 cm of the selected treatment area
• Topical salves or topical steroids within the treatment area or within 2 cm of the selected treatment area
• Artificial tanners within the treatment area or within 5 cm of the selected treatment area
• Use of the following therapies and/or medications within 4 weeks prior to the Screening visit:
• Treatment with immunomodulators (e.g., azathioprine), cytotoxic drugs (e.g., cyclophosphamide, vinblastine, chlorambucil, methotrexate) or interferons/interferon inducers
• Treatment with systemic medications that suppress the immune system (e.g., cyclosporine, prednisone, methotrexate, alefacept, infliximab)
• Use of systemic retinoids (e.g., isotretinoin, acitretin, bexarotene) within 6 months prior to the Screening visit

Sponsors & Collaborators

• Austin Institute for Clinical Research: lead

Study Sites (1)

Austin Institute for Clinical Research, Inc.

Pflugerville, Texas, 78660, United States

Location

Related Publications (2)

• Blauvelt A, Kempers S, Lain E, Schlesinger T, Tyring S, Forman S, Ablon G, Martin G, Wang H, Cutler DL, Fang J, Kwan MR; Phase 3 Tirbanibulin for Actinic Keratosis Group. Phase 3 Trials of Tirbanibulin Ointment for Actinic Keratosis. N Engl J Med. 2021 Feb 11;384(6):512-520. doi: 10.1056/NEJMoa2024040.

  PMID: 33567191 BACKGROUND

• Hong JB, Wu PY, Qin A, Huang YW, Tseng KC, Lai CY, Chan WK, Fang J, Cutler DL, Tsai TF. Topical Tirbanibulin, a Dual Src Kinase and Tubulin Polymerization Inhibitor, for the Treatment of Plaque-Type Psoriasis: Phase I Results. Pharmaceutics. 2022 Oct 11;14(10):2159. doi: 10.3390/pharmaceutics14102159.

  PMID: 36297594 RESULT

MeSH Terms

Interventions

tirbanibulin

Study Officials

• Edward Lain, MD, MBA

  Austin Institute for Clinical Research

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Model Details: Open Label Study

Study Record Dates

• First Submitted: January 16, 2023
• First Posted: February 6, 2023
• Study Start: April 17, 2023
• Primary Completion: March 7, 2024
• Study Completion: March 7, 2024
• Last Updated: July 19, 2024

Record last verified: 2024-07

Locations

US (1)