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Basal Cell Nevus SyndromePhase 3NCT03703310

Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Basal Cell Nevus Syndrome (Gorlin Syndrome)

A Phase 3 study testing Patidegib Topical Gel, 2% in people with Basal Cell Nevus Syndrome, run by Sol-Gel Technologies, Ltd. at 49 sites in 10 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This is a global, 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 Gorlin syndrome. Participants will be required to apply the investigational product for 12 months. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Basal Cell Nevus Syndrome
What is being tested?
Patidegib Topical Gel, 2%
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
174 participants
Where
49 sitesin 10 countries
Study dates
19 Feb 2019to 28 Dec 2020
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 participant must be age at least 18 years of age at the Screening Visit.
  • The participant must provide written informed consent prior to any study procedures.
  • The participant must meet diagnostic criteria for the basal cell nevus (Gorlin) syndrome including major criterion #3a plus 1 additional major criterion or plus 2 additional minor criteria listed below.
  • Major criteria:
  • >2 histologically confirmed BCCs or 1 for participant under age 20.
  • Odontogenic keratocysts of the jaw confirmed histologically.
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  • ≥3 palmar and/or plantar pits seen at the Screening Visit.
  • Bilamellar calcification of the falx cerebri present at less than 20 years of age.
  • Fused, bifid, or markedly splayed ribs.
  • First degree relative with Gorlin syndrome.
  • Patched protein 1 (PTCH1) mutation predicted to be of functional significance in normal tissue.
  • Minor criteria:
  • Macrocephaly.
  • Congenital malformations including frontal bossing, cleft lip or palate, "coarse face", moderate to severe hypertelorism.
  • Skeletal abnormalities detectable clinically: Sprengel deformity, marked pectus deformity, or marked finger syndactyly.
  • Skeletal abnormalities detectable radiographically: bridging of the sella turcica; vertebral abnormalities such as hemivertebrae, fusion or elongation of the vertebral bodies; modeling defects of the hands and feet; flame shaped lucencies of the hands or feet.
  • Ovarian fibroma.
  • Medulloblastoma (Modification of criteria of V Kimonis et al Am J Med Genet 69: 299-308, 1997).
  • The participant must have 10 (with at least 3 on the face) clinically typical BCCs present within 24 months prior to Randomization (Baseline/Day 1). Additionally, the subject must have at least 2 BCCs with longest diameter <5 mm present on the face prior to Randomization (Baseline/Day 1).
  • The participant is willing to have blood collected to measure circulating drug levels.
  • The participant is 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. Moisturizers and emollients are allowed. Participant will be encouraged to use their preferred sunscreen with a sun protector factor (SPF) of at least 30 daily on all exposed skin sites.
  • If the participant is a woman of childbearing potential (WOCBP), she must be willing to use complete abstinence from sexual intercourse and/or she and her partner must be willing to use at least 2 highly-effective forms of birth control starting prior to Baseline, through the duration of the study, and for 12 months after last application of IP.
  • If the participant is a male with a female sex partner who is a WOCBP, the participant must be willing to use condoms, even after a vasectomy, starting prior to Baseline, through the duration of the study, and for at least 8 months after the last application of IP.
  • The participant is willing for all facial BCCs to be evaluated and treatment recommendations made only by the Investigator.
  • The participant is willing to forego treatment of facial BCCs with anything other than the study IP except when the Investigator believes that delay of treatment of a facial BCC potentially might compromise the health of the subject. During the trial the only allowed form of treatment is surgical. Non-facial BCCs may be removed at the discretion of the Investigator or Primary Skin Care Physician (PSCP).

Who cannot join

  • The subject has previously participated in a clinical trial evaluating patidegib topical gel.
  • The participant has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study IP. Among these are use of the following:
  • 5-fluorouracil, imiquimod, diclofenac, or Ingenol mebutate (except as topical treatment to discrete non-facial BCCs) systemically or topically to the skin within the 2 months prior to the Screening Visit.
  • Systemic chemotherapy within 1 year prior to the Screening Visit.
  • Known inhibitors of the Hedgehog signaling pathway (such as vismodegib, sonidegib, itraconazole) topically or systemically within 3 months prior to the Screening Visit.
  • Photodynamic therapy (PDT) except to localized non-facial, individual BCCs within 2 months prior to the Screening Visit.
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  • The participant is known to have a hypersensitivity to any of the ingredients in the study medication formulation.
  • The participant is unable or unwilling to make a good faith effort to return to the study site for all study visits and tests.
  • The participant has uncontrolled systemic disease.
  • The participant 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.
  • The participant has current, recent (within five half-lives of the experimental drug or if half-life not known, within the past 6 months prior to the Screening Visit), or planned participation in an experimental drug study while enrolled in this study.
  • The participant is a WOCBP who is unwilling or unable to comply with pregnancy prevention measures.
  • The participant is pregnant or breastfeeding.
  • The participant has any condition or situation which, in the Investigator's opinion, may put the subject at significant risk, could confound the study results, or could interfere significantly with the subject's participation in the study. This may include a history of other skin conditions (such as severe facial eczema) or diseases, metabolic dysfunction, physical examination findings, or clinical laboratory findings giving reasonable suspicion of a disease or condition that contraindicates use of an investigational drug or that might affect interpretation of the results of the study or render the participant at high risk from treatment complications.

Trial locations and contacts

49 sites in 10 countries.

49 sites in 10 countries

United States22 sites

  • University of Michigan, Dept of Dermatology

    Ann Arbor, Michigan, 48109-5314

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

  • Laser & Skin Surgery Center of Indiana

    Carmel, Indiana, 46032

  • The University of Chicago

    Chicago, Illinois, 60637

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195

  • Duke University

    Durham, North Carolina, 27710

  • Penn State Health Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033

  • MD Anderson Cancer Center

    Houston, Texas, 77030

  • Dartmouth-Hitchcock Medical Center

    Lebanon, New Hampshire, 03756

  • University of Miami Miller School of Medicine

    Miami, Florida, 33136

  • University of Minnesota, Department of Dermatology

    Minneapolis, Minnesota, 55455

  • University of Utah Midvalley Dermatology

    Murray, Utah, 84107

  • Yale School of Medicine

    New Haven, Connecticut, 06519

  • Columbia University Medical Center

    New York, New York, 10032

  • Dermatology Center of Newport

    Newport Beach, California, 92660

  • Leavitt Medical Associates of Florida

    Ormond Beach, Florida, 32174

  • Hospital of the University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

  • Mayo Clinical Cancer Center

    Phoenix, Arizona, 85054

  • Oregon Health and Science University

    Portland, Oregon, 97239

  • Stanford University, Department of Dermatology

    Redwood City, California, 94063

  • University of California, San Francisco

    San Francisco, California, 94115

  • Saint Louis University Dermatology

    St Louis, Missouri, 63104

Other trials in United States

France6 sites

  • Hopital Saint-Andre - CHU Bordeaux

    Bordeaux, 33075

  • CHRU de Lille - Hopital Claude HURIEZ

    Lille, 59037

  • CHU La Timone

    Marseille, 13395

  • PellePharm Investigative Site

    Nantes, 44093

  • Hopital Saint-Louis

    Paris, 75010

  • PellePharm Investigative Site

    Pierre-Bénite, 69310

Other trials in France

Germany4 sites

  • Charite - Universitatsmedizin Berlin

    Berlin, 10117

  • Klinik und Poliklinik fur Dermatologie und Allergologie

    Munich, 80337

  • Universitatsklinikum Münster

    Münster, 48149

  • Universitats Hautklinik

    Tübingen, 72076

Other trials in Germany

Italy4 sites

  • Ospedale Vanvitelli-University della Campania-Dermatologia Edificio 9

    Naples, 80131

  • Catholic University of the Sacred Heart

    Roma, 00168

  • Humanitas University Milan

    Rozzano, 20089

  • Ospedale San Bortolo

    Vicenza, 36100

Other trials in Italy

United Kingdom4 sites

  • NHS Greater Glasgow and Clyde

    Glasgow, G38SJ

  • Royal London Hospital

    London, E1 1BB

  • Oxford University Hospitals NHS Foundation Trust

    Oxford, OX37LE

  • Salford Royal Hospital

    Salford, M68HD

Other trials in United Kingdom

Spain3 sites

  • Hospital Clinic I Provincial

    Barcelona, 08036

  • Hospital Universitario Ramon y Cajal

    Madrid, 28034

  • Hospital Universitiario Virgen de la Macarena

    Seville, 41009

Other trials in Spain

Belgium2 sites

  • PellePharm Investigative Site

    Brussels, 1200

  • PellePharm Investigative Site

    Leuven, 3000

Other trials in Belgium

Canada2 sites

  • Pellepharm Investigative Site

    Toronto, M5S1B2

  • Clinical Trials Unit - The Skin Care Centre

    Vancouver, British Columbia

Other trials in Canada

Denmark1 site

  • PellePharm Investigative Site

    Copenhagen

Other trials in Denmark

Netherlands1 site

  • Maastricht University Medical Center - Dept of Dermatology

    Maastricht, 6202

Other trials in Netherlands

About this study

Official title: A Multicenter, Randomized, Double-blind, Vehicle-controlled, Phase 3 Efficacy and Safety Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Basal Cell Nevus Syndrome

This is a global, 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 Gorlin syndrome. Participants will be required to apply the investigational product for 12 months. The primary endpoint is a comparison between the two treatment arms of the number of new BCCs that develop over the 12 month period.

Read the detailed description

An open-label, extension safety and tolerability study is planned for at least 12 months duration following the end of this study. All participants who complete the Month 12 Exit Visit having demonstrated adequate compliance with application of the Investigational Product (IP) without major Protocol Deviations (PDs) during the study will be eligible for participation in the extension study.

All participants will be contacted by phone approximately 30 days following the Exit or Discontinuation Visit to determine if the participant has experienced any new adverse events (AEs)/serious AEs (SAEs) since discontinuation/completion of study treatment.

Being tested
Patidegib Topical Gel, 2%, Patidegib Topical Gel, Vehicle
Keywords
Gorlin syndrome, Basal cell nevus syndrome BCNS, Nevoid basal cell carcinoma syndrome, Basal cell carcinoma, Hedgehog, Surgically eligible basal cell carcinomas, Patidegib
Medical subject headings
Basal Cell Nevus Syndrome, Carcinoma, Basal Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Patidegib Topical Gel, 2%Drug

    Also called IP

    Patidegib Topical Gel, 2%

  • Patidegib Topical Gel, VehicleDrug

    Also called IP, Vehicle

    Patidegib Topical Gel, Vehicle

Study groups in NCT03703310
Study groupRoleWhat they receive
Patidegib Topical Gel, 2%,ExperimentalParticipants will be randomized to receive Patidegib Topical Gel, 2%. The Patidegib Topical Gel, 2% will be dispensed to participants at each study visit and applied topically twice daily to the face.
Receives: Drug: Patidegib Topical Gel, 2%
Patidegib Topical Gel, VehiclePlacebo comparatorParticipants will be randomized to receive Vehicle. The Patidegib Topical Gel, Vehicle will be dispensed to participants at each study visit and applied topically twice daily to the face.
Receives: Drug: Patidegib Topical Gel, Vehicle

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of New BCCs Per Participant

    Measured: Month 12

Secondary outcomes (6)

  1. Number of New Surgically Eligible BCCs (nSEBs) Per Participant

    Measured: Month 12

  2. Percentage of Participants Developing >=2 Facial New BCCs

    Measured: Month 12

  3. Percentage of Participants Developing >=1 Facial New BCCs

    Measured: Month 12

  4. Number of New BCCs Per Participant

    Measured: Month 9

  5. Number of New BCCs Per Participant

    Measured: Month 6

  6. Change in Advanced Basal Cell Carcinoma Index (aBCCdex) Lesion Symptom Score Scale Score

    The Advanced Basal Cell Carcinoma Index (aBCCdex) measures the impact of basal cell carcinoma on patients' quality of life. The index consists of several subscales, each subscale with its own range of scores for each question

    Lesion Symptoms Scale: Scores range from 1 to 7 and it includes 6 questions. Therefore the total score range is from 6 (minimum) to 42 (maximum), where a higher score correlates with a poorer quality of life.

    Measured: Month 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Oct 2018
  2. Study start19 Feb 2019
  3. Primary completion28 Dec 2020
  4. Study completion28 Dec 2020

Resources

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
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Participants will be randomized 1:1 to receive Patidegib Topical Gel, 2%, or Vehicle (IP)
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
174 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03703310
Sponsor's protocol ID
Pelle-926-301
Status last verified
Mar 2021
First submitted
9 Oct 2018
Last updated
3 Jul 2024
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Patidegib Topical Gel, 2% in people with Basal Cell Nevus Syndrome, run by Sol-Gel Technologies, Ltd. at 49 sites in 10 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 25 inclusion and 14 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 3 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 49 sites in United States, France, Germany, Italy, United Kingdom and 5 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?

Yes. Results are posted in the registry (first posted 3 Jul 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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 Per Participant (time frame: Month 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03703310.

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 3 Jul 2024.

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