Clinical trial • Phase III • Rare Disease|Dermatology|Oncology
PATIDEGIB HYDROCHLORIDE ISOPROPANOLATE for Gorlin syndrome|Basal cell carcinoma
Phase III trial of PATIDEGIB HYDROCHLORIDE ISOPROPANOLATE for Gorlin syndrome|Basal cell carcinoma.
Overview
- Trial Therapeutic Area
- Rare Disease|Dermatology|Oncology
- Trial Disease
- Gorlin syndrome|Basal cell carcinoma
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
Key dates
- Initial CTIS Submission Date
- 05-12-2023
- First CTIS Authorization Date
- 11-04-2024
Trial design
Randomised, vehicle gel (placebo), applied twice daily to the face (vehicle-controlled arm). Phase III trial across 12 sites in Poland, Netherlands, Germany and others.
- Randomised
- Yes
- Comparator
- Vehicle Gel (placebo), applied twice daily to the face (vehicle-controlled arm).
- Target Sample Size
- 96
- Trial Duration For Participant
- 395
Eligibility
Recruits 96 Vulnerable population is selected in the CTIS record. Informed consent is obtained using Subject Information Sheets and Informed Consent Forms (SIS and ICF) (multiple ICF documents present including 'L1 SIS and ICF' and 'Pregnant Partner' forms). A pre-ICF telephone data consent procedure is documented. Patient-facing documents and ICF materials are available in multiple languages..
- Vulnerable Population
- Vulnerable population is selected in the CTIS record. Informed consent is obtained using Subject Information Sheets and Informed Consent Forms (SIS and ICF) (multiple ICF documents present including 'L1 SIS and ICF' and 'Pregnant Partner' forms). A pre-ICF telephone data consent procedure is documented. Patient-facing documents and ICF materials are available in multiple languages.
Inclusion criteria
- {"criterion_text":"- The subject must be at least 18 years old at the Screening Visit\n- The subject must be confirmed to have a PTCH1 mutation\n- The subject must have at least 10 BCCs on the face at Randomization (Baseline/Day 1)\n- 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"}
Exclusion criteria
- {"criterion_text":"- The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study Investigational Product (IP)\n- The subject is known to have hypersensitivity to any of the ingredients in the IP formulation\n- The subject has uncontrolled systemic disease\n- The subject has been treated for invasive cancer within the past 5 years excluding chronic lymphocytic leukemia Stage 0 and non-melanoma skin cancer, Stage I cervical cancer, or ductal carcinoma in situ of the breast\n- Previous Hedgehog inhibitor therapy was considered to have failed for reasons other than unfavorable side effect(s)"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Number of new BCCs at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) when applied twice daily to the face of subjects with Gorlin syndrome as assessed by the CPRB","definition_or_measurement_approach":"Count of new basal cell carcinomas at Month 12 comparing Patidegib Gel 2% vs vehicle; assessment performed by the Central Photo Review Board (CPRB)."}
Secondary endpoints
- {"endpoint_text":"- Number of New Surgically Eligible BCCs (nSEBs) at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) when applied twice daily to the face of subjects with Gorlin syndrome\n- Change from Baseline to Month 12 in Advanced Basal Cell Carcinoma Index (aBCCdex) lesion symptoms scale score\n- Change from Baseline to Month 12 in confirmed BCCs lesion diameter\n- Proportion of Surgically Eligible BCCs (SEBs) and nSEBs assessed as resolved by Month 12\n- Proportion of SEBs and nSEBs per subject that have regressed by Month 12\n- Proportion of subjects developing ≥2 facial new BCCs at Month 12\n- Change in total number of confirmed BCCs from Baseline to Month 12\n- Change from Baseline to Month 12 in the Skin Cancer Index quality of life questionnaire score\n- Adverse events (AEs), including serious adverse events (SAEs) occurring at any time during the study\n- Investigator Dermal Safety and Local Tolerability Assessments score at any time during the study\n- Change from Baseline to Month 12 in Patient Global Impression in disease Severity (PGI-S)\n- Change in Patient Global Impression of Change in clinical status (PGI C) at Month 12\n- Number of new BCCs at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) when applied twice daily to the face of subjects with Gorlin syndrome\n- Number of nSEBs at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) when applied twice daily to the face of subjects with Gorlin syndrome\n- Time to first new BCC\n- Skin pain and peak pruritus scores as reported by the subject on a numeric rating scale","definition_or_measurement_approach":"Endpoints assessed by lesion counts at Month 12, investigator assessments (Investigator Dermal Safety and Local Tolerability), central photo review (for lesion counts), patient-reported outcome scales (aBCCdex, Skin Cancer Index, PGI-S, PGI-C), time-to-event analysis for first new BCC, and standard AE/SAE reporting throughout study."}
Recruitment
- Digital Remote Recruitment
- Yes
- Planned Sample Size
- 96
- Recruitment Window Months
- 22
- Consent Approach
- Informed consent obtained from participants (subjects must be ≥18 years). Subject Information Sheets and Informed Consent Forms (L1 SIS and ICF and related documents) are provided; pre-ICF telephone data consent is documented. Patient-facing materials and ICFs are available in multiple languages (English, French, Spanish, Italian, Dutch, Polish, German as indicated by document translations). There is a 'Pregnant Partner' ICF variant documented.
Methods
- K1/K2 recruitment documents present (titles include 'K1_ Recruitment arrangements' and 'K2_ Recruitment material_Study Referral Letter') indicating site referral and clinician/GP referral materials.
- GP/primary care recruitment channel: 'K2_Recruitment material_GP Letter' document present (study referral via GPs/clinicians).
- Site-based recruitment via participating dermatology clinics (trial sites listed in each country).
Geography
- Total Number Of Sites
- 12
- Total Number Of Participants
- 60
Poland
- Earliest CTIS Part Ii Submission Date
- 29-03-2024
- Latest Decision Or Authorization Date
- 30-07-2024
- Processing Time Days
- 123
- Number Of Sites
- 1
- Number Of Participants
- 10
Sites
- Site Name
- Dermoklinika-Centrum Medyczne s.c. M. Kierstan J. Narbutt A. Lesiak
- Principal Investigator Name
- Aleksandra Lesiak
- Principal Investigator Email
- lesiak_ola@interia.pl
- Contact Person Name
- Aleksandra Lesiak
- Contact Person Email
- lesiak_ola@interia.pl
Netherlands
- Earliest CTIS Part Ii Submission Date
- 15-03-2024
- Latest Decision Or Authorization Date
- 10-04-2025
- Processing Time Days
- 391
- Number Of Sites
- 1
- Number Of Participants
- 10
Sites
- Site Name
- University Hospital Maastricht
- Department Name
- Dermatology
- Principal Investigator Name
- Klara Mosterd
- Principal Investigator Email
- K.mosterd@mumc.nl
- Contact Person Name
- Klara Mosterd
- Contact Person Email
- K.mosterd@mumc.nl
Germany
- Earliest CTIS Part Ii Submission Date
- 15-03-2024
- Latest Decision Or Authorization Date
- 06-08-2025
- Processing Time Days
- 509
- Number Of Sites
- 3
- Number Of Participants
- 10
Sites
- Site Name
- Deutsches Rotes Kreuz Gemeinnuetzige Krankenhaus Sachsen GmbH
- Department Name
- Clinic of Dermatology
- Principal Investigator Name
- Martin Kaatz
- Principal Investigator Email
- kaatz.martin@drk-khs.de
- Contact Person Name
- Martin Kaatz
- Contact Person Email
- kaatz.martin@drk-khs.de
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Dept. of Dermatology, Venerology and Allergology
- Principal Investigator Name
- Thomas Eigentler
- Principal Investigator Email
- thomas.eigentler@charite.de
- Contact Person Name
- Thomas Eigentler
- Contact Person Email
- thomas.eigentler@charite.de
- Site Name
- Klinikum der Universitaet Muenchen AöR
- Department Name
- Department of Dermatology
- Principal Investigator Name
- Lucie Heinzerling
- Principal Investigator Email
- Lucie.Heinzerling@med.uni-muenchen.de
- Contact Person Name
- Lucie Heinzerling
- Contact Person Email
- Lucie.Heinzerling@med.uni-muenchen.de
France
- Earliest CTIS Part Ii Submission Date
- 04-04-2024
- Latest Decision Or Authorization Date
- 12-03-2026
- Processing Time Days
- 707
- Number Of Sites
- 3
- Number Of Participants
- 10
Sites
- Site Name
- Hopital Avicenne
- Department Name
- Dermatology
- Principal Investigator Name
- Eve Maubec
- Principal Investigator Email
- eve.maubec@aphp.fr
- Contact Person Name
- Eve Maubec
- Contact Person Email
- eve.maubec@aphp.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- Dermatology
- Principal Investigator Name
- Stéphane DALLE
- Principal Investigator Email
- stephane.dalle@chu-lyon.fr
- Contact Person Name
- Stéphane DALLE
- Contact Person Email
- stephane.dalle@chu-lyon.fr
- Site Name
- Hospital Hotel Dieu
- Department Name
- Oncodermatology
- Principal Investigator Name
- Gaëlle Quereux
- Principal Investigator Email
- gaelle.quereux@chu-nantes.fr
- Contact Person Name
- Gaëlle Quereux
- Contact Person Email
- gaelle.quereux@chu-nantes.fr
Italy
- Earliest CTIS Part Ii Submission Date
- 14-02-2024
- Latest Decision Or Authorization Date
- 12-03-2026
- Processing Time Days
- 757
- Number Of Sites
- 3
- Number Of Participants
- 10
Sites
- Site Name
- Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
- Department Name
- Dermatology
- Principal Investigator Name
- Piergiacomo Calzavara Pinton
- Principal Investigator Email
- piergiacomo.calzavarapinton@unibs.it
- Contact Person Name
- Piergiacomo Calzavara Pinton
- Contact Person Email
- piergiacomo.calzavarapinton@unibs.it
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- Dermatology
- Principal Investigator Name
- Ketty Peris
- Principal Investigator Email
- ketty.peris@unicatt.it
- Contact Person Name
- Ketty Peris
- Contact Person Email
- ketty.peris@unicatt.it
- Site Name
- Azienda Ospedaliero-Universitaria Maggiore Della Carita
- Department Name
- Dermatology
- Principal Investigator Name
- Paola Savoia
- Principal Investigator Email
- paola.savoia@maggioreosp.novara.it
- Contact Person Name
- Paola Savoia
- Contact Person Email
- paola.savoia@maggioreosp.novara.it
Spain
- Earliest CTIS Part Ii Submission Date
- 19-03-2024
- Latest Decision Or Authorization Date
- 12-03-2026
- Processing Time Days
- 723
- Number Of Sites
- 3
- Number Of Participants
- 10
Sites
- Site Name
- Hospital Universitario 12 De Octubre
- Department Name
- Dermatology
- Principal Investigator Name
- Pablo Luis Ortiz Romero
- Principal Investigator Email
- pablo.ortiz@salud.madrid.org
- Contact Person Name
- Pablo Luis Ortiz Romero
- Contact Person Email
- pablo.ortiz@salud.madrid.org
- Site Name
- Hospital Clinic De Barcelona
- Department Name
- Dermatology
- Principal Investigator Name
- Susana Puig Sarda
- Principal Investigator Email
- spuig@clinic.cat
- Contact Person Name
- Susana Puig Sarda
- Contact Person Email
- spuig@clinic.cat
- Site Name
- Hospital De La Santa Creu I Sant Pau
- Department Name
- Dermatology
- Principal Investigator Name
- Veronica Ruiz Salas
- Principal Investigator Email
- vruizs@santpau.cat
- Contact Person Name
- Veronica Ruiz Salas
- Contact Person Email
- vruizs@santpau.cat
Sponsor
Primary sponsor
- Full Name
- Sol-Gel Technologies Ltd.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Israel
Contract research organisations
- Name
- Premier Research Group Limited
- Responsibilities
- codes: 1,10,11,12,13,2,3,5,6,8
- Name
- Medidata Solutions Inc.
- Responsibilities
- codes: 7
- Name
- Acm Global Central Laboratory Limited
- Responsibilities
- codes: 4
Third parties
- {"country":"United States","full_name":"Meeting Protocol Worldwide LP","duties_or_roles":"Patient travel reimbursement","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Canfield Scientific Inc.","duties_or_roles":"Central photography reader","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
- {"country":"France","full_name":"Banook Services","duties_or_roles":"Central ECG reader","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United Kingdom","full_name":"Acm Global Central Laboratory Limited","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Netherlands","full_name":"Xerimis B.V.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Premier Research Group Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Patidegib Gel
- Active Substance
- PATIDEGIB HYDROCHLORIDE ISOPROPANOLATE
- Modality
- Small molecule
- Routes Of Administration
- Topical (facial application)
- Route
- Topical (face)
- Starting Dose
- Patidegib Gel 2% applied twice daily to the face
- Frequency
- Twice daily
- Maximum Dose
- 1 g per day
- Investigational Product Name
- Vehicle Gel
- Modality
- Other
- Routes Of Administration
- Topical (facial application)
- Route
- Topical (face)
- Frequency
- Twice daily