Clinical trial NCT04155190: An Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas in Patients With Non-Gorlin High Frequency BCC
Official title: A Multicenter, Randomized, Double Blind, Vehicle-controlled, Phase 2 Efficacy and Safety Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Patients With Non-Gorlin High Frequency BCC
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Recurrent Basal Cell Carcinoma
Keywords: High Frequency Basal Cell Carcinoma
non-gorlin
HF-BCC
Bazex-Dupré-Christol Syndrome
Rombo Syndrome
Oley Syndrome
Xeroderma Pigmentosum (XP) Syndrome
BCC
SEB
Hedgehog inhibitor
Basal Cell Nevus Syndrome
Basal Cell Carcinoma
Surgically Eligible Basal Cell Carcinoma
patidegib
HFBCC
HHI
Lead sponsor: Sol-Gel Technologies, Ltd.
Sponsor class: INDUSTRY
First posted: 2019-11-07
Enrollment: 47
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Participants will be randomized (1:1) to receive either Patidegib Topical Gel, 2%, or Vehicle for 9 months. Randomization will be stratified by gender.
Primary purpose: TREATMENT

Brief summary: This is a multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult participants with non-Gorlin HF-BCC (high-frequency basal cell carcinoma). Participants will be randomized (1:1) to receive either Patidegib Topical Gel, 2%, or Vehicle for 9 months. Randomization will be stratified by gender. The primary endpoint is the number of nSEB (surgically eligible basal cell carcinoma) that develop on the face over the 9 month period. The primary end point will be assessed by imaging and tracking of BCCs consistently throughout the study in order to identify nSEBs.


Eligibility criteria: Inclusion Criteria:

1. The subject must have had at least 10 (with at least 3 on the face) clinically typical BCCs present within 24 months prior to Screening and Randomization (Baseline/Day 1). Additionally, the subject must have at least 2 BCCs with longest diameter \<5 mm present on the face prior to Screening and Randomization (Baseline/Day 1).
2. The subject must be willing to abstain from application of a non-study topical medication (prescription or over the counter) to facial skin for the duration of the trial except as prescribed by the Investigator.

Exclusion Criteria:

1. The subject has been previously diagnosed with Gorlin syndrome
2. On medical history, family history or clinical examination there is a suspicion that the patient has Gorlin syndrome.
3. Patients with a family history of medulloblastoma
4. The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study IP.
5. The subject has uncontrolled systemic disease.
6. The subject has been treated for invasive cancer within the past 5 years excluding non-melanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of the breast, or chronic lymphocytic leukemia (CLL) Stage 0.

Primary outcomes:
measure: Number of new surgically eligible BCCs (nSEBs)
timeFrame: Baseline through Month 9
Secondary outcomes:
measure: Number of treatment emergent adverse events assessed with means and standard errors or proportions
timeFrame: Baseline through Month 9
Other outcomes:
Locations:
city: Apple Valley
country: United States
facility: Axiom Research, LLC
state: California
city: Colton
country: United States
facility: Axiom Research, LLC
state: California
city: Fremont
country: United States
facility: Center for Dermatology Clinical Research, Inc.
state: California
city: Los Angeles
country: United States
facility: Dermatology Research Associates
state: California
city: Newport Beach
country: United States
facility: The Dermatology Center of Newport
state: California
city: Delray Beach
country: United States
facility: Palm Beach Dermatology Research
state: Florida
city: Ormond Beach
country: United States
facility: Leavitt Medical Associates of Florida
state: Florida
city: Saint Augustine
country: United States
facility: PellePharm Investigative Site
state: Florida
city: Plainfield
country: United States
facility: The Indiana Clinical Trials Center
state: Indiana
city: Warren
country: United States
facility: Grekin Skin Institute
state: Michigan
city: Henderson
country: United States
facility: PellePharm Investigative Site
state: Nevada
city: Hackensack
country: United States
facility: Skin Laser and Surgery Specialists of NY&NJ
state: New Jersey
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Murray
country: United States
facility: University of Utah
state: Utah
Study officials:
affiliation: PellePharm, Inc.
name: VP, Clinical Operations
role: STUDY_DIRECTOR
References: