Gorlin SyndromePhase 3NCT06050122
Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome
A Phase 3 study testing Patidegib Topical Gel and Patidegib Topical Gel with no active patidegib in people with Gorlin Syndrome, run by Sol-Gel Technologies, Ltd. at 40 sites in 7 countries.
Trial summary
The aim of this clinical study is to find out how well Patidegib Gel 2% works in preventing new basal cell carcinomas (BCCs) developing on the face of adults with Gorlin syndrome, and how safe Patidegib Gel 2% is to use. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gorlin Syndrome
- What is being tested?
- Patidegib Topical Gel and Patidegib Topical Gel with no active patidegib
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 113 participants
- Where
- 40 sitesin 7 countries
- Study dates
- 17 Mar 2024to 14 Sep 2026
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- The subject must be at least 18 years old at the Screening Visit.
- The subject must be confirmed to have a PTCH1 mutation.
- The subject must have at least 10 BCCs on the face at Randomization (Baseline/Day 1).
- The subject must be willing to abstain from application of any non-study topical medication (prescription or over the counter) to their facial skin for the duration of the trial, except as prescribed by the investigator.
Who cannot join
- The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study Investigational Product (IP).
- The subject is known to have hypersensitivity to any of the ingredients in the IP formulation.
- The subject has uncontrolled systemic disease.
- The subject has been treated for invasive cancer within the past 5 years excluding chronic lymphocytic leukemia Stage 0, non-melanoma skin cancer, successfully treated melanoma in situ and Stage I melanoma, Stage I cervical cancer, or ductal carcinoma in situ of the breast.
- Inefficacy of previous Hedgehog inhibitor therapy.
Trial locations and contacts
40 sites in 7 countries.
40 sites in 7 countries
United States24 sites
University of Michigan Health - Michigan Medicine - University Hospital
Ann Arbor, Michigan, 48109
The Johns Hopkins Hospital
Baltimore, Maryland, 21287-0006
Apex Clinical Research Center
Canton, Ohio, 44718
The University of Chicago Medicine
Chicago, Illinois, 60637
Cleveland Clinic - Main Campus
Cleveland, Ohio, 44195
Duke University Health System
Durham, North Carolina, 27710
Center for Dermatology Clinical Research
Fremont, California, 94538
North Texas Center for Clinical Research
Frisco, Texas, 75034
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033
The University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
University of California Los Angeles (UCLA) - Center for Inflammatory Bowel Diseases - Westwood Location
Los Angeles, California, 90024
Apex Clinical Research Center
Mayfield Heights, Ohio, 44124
Minnesota Clinical Study Center
New Brighton, Minnesota, 55112
Yale University
New Haven, Connecticut, 06519
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
The Dermatology Center of Newport
Newport Beach, California, 92660
SSM Health Dermatology
Oklahoma City, Oklahoma, 73102
Oregon Health and Science University
Portland, Oregon, 97239
Stanford University - Lucille Packard's Children's Hospital
Redwood City, California, 94063
Northwest Arkansas Clinical Trials Center
Rogers, Arkansas, 72758
MediSearch Clinical Trials
Saint Joseph, Missouri, 64506
Dermatology Associates of Tallahassee
Tallahassee, Florida, 32308
University of South Florida Health
Tampa, Florida, 33612
Schweiger Dermatology P.C.
Verona, New Jersey, 07044
France3 sites
Hopital Avicenne
Bobigny
Centre Hospitalier Universitaire De Nantes
Nantes
Centre Hospitalier Lyon Sud
Pierre-Bénite
Germany3 sites
Charité Universitätsmedizin Berlin - Campus Charité Mitte
Berlin
DRK Gemeinnützige Krankenhaus GmbH Sachsen
Chemnitz
Ludwig-Maximilians-Universitaet Muenchen (LMU) - Klinik und Poliklinik fuer Dermatologie und Allergologie
Munich, Bavaria
Italy3 sites
ASST Spedali Civili di Brescia
Brescia
AOU Maggiore della Carita
Novara
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome
Spain3 sites
Hospital Clinic Barcelona
Barcelona
Hospital de la Santa Creu i Sant Pau
Barcelona
Hospital Universitartio 12 De Octubre
Madrid
United Kingdom3 sites
The University of Birmingham
Birmingham
Barts Health NHS Trust - The Royal London Hospital
London
Northern Care Alliance NHS Foundation Trust
Salford
About this study
Official title: A Multicenter, Randomized, Double Blind, Vehicle-controlled, Phase 3 Efficacy and Safety Study of Patidegib Gel 2% for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Gorlin Syndrome
The aim of this clinical study is to find out how well Patidegib Gel 2% works in preventing new basal cell carcinomas (BCCs) developing on the face of adults with Gorlin syndrome, and how safe Patidegib Gel 2% is to use.
People who take part will apply either Patidegib Gel 2% or a Vehicle Gel (with no active drug substance) to their face twice a day for a year (12 months). The number of new BCCs on the face will be compared between those who used Patidegib Gel 2% or Vehicle Gel after 12 months.
Read the detailed description
This is a global, multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Gel 2%, applied topically twice daily to the face of adult subjects with Gorlin syndrome.
Subjects will be randomized (1:1) to receive either Patidegib Gel 2% or Vehicle Gel for 12 months.
The assignment of subjects to the 2 groups will be stratified by sex assigned at birth, age (≥60 or <60 years), and number of BCC lesions at the treatment area (face) (10-15, 16-30 or >30) at Baseline.
All suspicious lesions will be imaged and tracked consistently throughout the study so that new BCCs that arise can be readily identified. New BCCs will be confirmed by dermoscopic analysis through the study. The BCC images will be reviewed and assessed by a Central Photo Review Board (CPRB).
- Conditions
- Gorlin Syndrome
- Being tested
- Patidegib Topical Gel, Patidegib Topical Gel with no active patidegib
- Medical subject headings
- Basal Cell Nevus Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Patidegib Topical GelDrug
Also called Patidegib, IPI-926, Saridegib, SGT-610
Patidegib Gel 2% is a smooth, clear, colorless-to-yellow, viscous, hydro-alcoholic gel for topical administration.
Patidegib Topical Gel with no active patidegibDrug
Also called Vehicle Gel, Placebo
Vehicle Gel consists of the excipients of Patidegib Gel with no active patidegib. It is a smooth viscous gel for topical administration.
| Study group | Role | What they receive |
|---|---|---|
| Patidegib Gel 2% | Experimental | Patidegib Gel 2% (w/w), applied topically to the face twice daily for 12 months Receives: Drug: Patidegib Topical Gel |
| Vehicle Gel | Placebo comparator | Vehicle Gel, applied topically to the face twice daily for 12 months Receives: Drug: Patidegib Topical Gel with no active patidegib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of new BCCs on the face at Month 12
New BCCs at Month 12 in the Patidegib Gel 2% and Vehicle Gel groups, as assessed by the CPRB
Measured: Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2023
- Study start17 Mar 2024
- Primary completion20 Aug 2026
- Study completion14 Sep 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gorlin SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 113 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sol-Gel Technologies, Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06050122
- Sponsor's protocol ID
- SGT-610-01
- Other IDs
- 2023-507528-21-00, 2023
- Status last verified
- Sep 2026
- First submitted
- 15 Sep 2023
- Last updated
- 18 Sep 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Double Blind, Vehicle-controlled, Phase 3 Efficacy and Safety Study of Patidegib Gel 2% for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Gorlin Syndrome
- ClinicalTrials.gov ID
- NCT06050122
- Acronym / sponsor ID
- SGT-610-01
- Conditions
- Gorlin Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 113 participants (actual)
- Lead sponsor
- Sol-Gel Technologies, Ltd.Industry (pharma, biotech, devices)
- Study start
- 17 Mar 2024 (actual)
- Primary completion
- 20 Aug 2026 (actual)
- Study completion
- 14 Sep 2026 (actual)
- First posted
- 22 Sep 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this clinical study is to find out how well Patidegib Gel 2% works in preventing new basal cell carcinomas (BCCs) developing on the face of adults with Gorlin syndrome, and how safe Patidegib Gel 2% is to use.
People who take part will apply either Patidegib Gel 2% or a Vehicle Gel (with no active drug substance) to their face twice a day for a year (12 months). The number of new BCCs on the face will be compared between those who used Patidegib Gel 2% or Vehicle Gel after 12 months.
Detailed description
This is a global, multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Gel 2%, applied topically twice daily to the face of adult subjects with Gorlin syndrome.
Subjects will be randomized (1:1) to receive either Patidegib Gel 2% or Vehicle Gel for 12 months.
The assignment of subjects to the 2 groups will be stratified by sex assigned at birth, age (≥60 or <60 years), and number of BCC lesions at the treatment area (face) (10-15, 16-30 or >30) at Baseline.
All suspicious lesions will be imaged and tracked consistently throughout the study so that new BCCs that arise can be readily identified. New BCCs will be confirmed by dermoscopic analysis through the study. The BCC images will be reviewed and assessed by a Central Photo Review Board (CPRB).
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patidegib Gel 2% | Experimental | Drug: Patidegib Topical GelPatidegib Gel 2% (w/w), applied topically to the face twice daily for 12 months |
| Vehicle Gel | Placebo comparator | Drug: Patidegib Topical Gel with no active patidegibVehicle Gel, applied topically to the face twice daily for 12 months |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- The subject must be at least 18 years old at the Screening Visit.
- The subject must be confirmed to have a PTCH1 mutation.
- The subject must have at least 10 BCCs on the face at Randomization (Baseline/Day 1).
- The subject must be willing to abstain from application of any non-study topical medication (prescription or over the counter) to their facial skin for the duration of the trial, except as prescribed by the investigator.
Key exclusion criteria (5 listed)
- The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study Investigational Product (IP).
- The subject is known to have hypersensitivity to any of the ingredients in the IP formulation.
- The subject has uncontrolled systemic disease.
- The subject has been treated for invasive cancer within the past 5 years excluding chronic lymphocytic leukemia Stage 0, non-melanoma skin cancer, successfully treated melanoma in situ and Stage I melanoma, Stage I cervical cancer, or ductal carcinoma in situ of the breast.
- Inefficacy of previous Hedgehog inhibitor therapy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Patidegib Topical GelAlso: Patidegib, IPI-926, Saridegib, SGT-610Patidegib Gel 2% is a smooth, clear, colorless-to-yellow, viscous, hydro-alcoholic gel for topical administration. | Drug | Patidegib Gel 2% |
| Patidegib Topical Gel with no active patidegibAlso: Vehicle Gel, PlaceboVehicle Gel consists of the excipients of Patidegib Gel with no active patidegib. It is a smooth viscous gel for topical administration. | Drug | Vehicle Gel |
Primary outcome
Number of new BCCs on the face at Month 12
Time frame: Month 12
New BCCs at Month 12 in the Patidegib Gel 2% and Vehicle Gel groups, as assessed by the CPRB
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
40 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Patidegib Topical Gel and Patidegib Topical Gel with no active patidegib in people with Gorlin Syndrome, run by Sol-Gel Technologies, Ltd. at 40 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 40 sites in United States, France, Germany, Italy, Spain and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of new BCCs on the face at Month 12 (time frame: Month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2026.