Recurrent Basal Cell Carcinoma
Conditions
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
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.
Interventions
Patidegib Topical Gel, 2%
Patidegib Topical Gel, Vehicle
Sponsors
Sol-Gel Technologies, Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Masking description
As a double-blinded study, the Investigators, the site staff, PellePharm, and the Clinical Monitor(s) will be blinded to the treatment assigned to individual subjects. Delegated staff members at the study site will dispense the investigational product (IP) and will collect and weigh all used and unused IP tubes.
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.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants With New Surgically Eligible Basal Cell Carcinomas (nSEBs) | Up to 9 months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Up to 9 months | TEAEs were defined as AEs that started on or after the first dose of study drug. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'. |
Countries
United States
Contacts
STUDY_DIRECTORVP, Clinical Operations
PellePharm, Inc.
Participant flow
Recruitment details
The study was terminated early by the previous responsible party (PellePharm Inc.) on 15-Jun-2021 due to low blinded event rate. Data presented in this results posting reflect the data collected prior to study termination by the previous responsible party, PellePharm Inc., and are reported by the current responsible party, Sol-Gel Technologies Ltd.
Pre-assignment details
The study was originally planned as a 2-arm study. However, participant-level data were collected and reported only in aggregate for the overall study, rather than by treatment arm for participant flow, study outcome measures, all-cause mortality, and adverse event data. The current Sponsor (Sol-Gel) prepared a Note to File for the Statistical Analysis Plan (SAP) to document how the data were collected.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 20 Participants |
| Age, Categorical Between 18 and 65 years | 11 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 24 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 47 Participants |
| Sex: Female, Male Female | 15 Participants |
| Sex: Female, Male Male | 16 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 47 |
| other Total, other adverse events | 0 / 47 |
| serious Total, serious adverse events | 2 / 47 |
Outcome results
None listed