Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 2 of 5 for:    Recruiting, Not yet recruiting Studies | Gorlin Syndrome

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03703310
Recruitment Status : Recruiting
First Posted : October 11, 2018
Last Update Posted : June 17, 2019
Sponsor:
Information provided by (Responsible Party):
PellePharm, Inc.

Tracking Information
First Submitted Date  ICMJE October 9, 2018
First Posted Date  ICMJE October 11, 2018
Last Update Posted Date June 17, 2019
Actual Study Start Date  ICMJE February 20, 2019
Estimated Primary Completion Date May 1, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 9, 2018)
Number of new surgically eligible BCCs (nSEBs) [ Time Frame: Month 12 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03703310 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 17, 2018)
  • Percentage of participants developing >=2 facial nSEBs [ Time Frame: Baseline, Month 12 ]
  • Percentage of participants developing >=1 facial nSEBs [ Time Frame: Baseline, Month 12 ]
  • Number of nSEBs per participant [ Time Frame: Month 9 ]
  • Number of nSEBs per participant [ Time Frame: Month 6 ]
  • Number of new BCCs [ Time Frame: Baseline, Month 12 ]
  • Change in Advanced Basal Cell Carcinoma Index (aBCCdex) lesion score [ Time Frame: Baseline, Month 12 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: October 9, 2018)
  • Percentage of participants developing >=2 facial nSEBs [ Time Frame: Baseline, Month 12 ]
  • Percentage of participants developing >=1 facial nSEBs [ Time Frame: Baseline, Month 12 ]
  • Number of nSEBs per participant [ Time Frame: Month 9 ]
  • Number of nSEBs per participant [ Time Frame: Month 6 ]
  • Number of new BCCs [ Time Frame: Baseline, Month 12 ]
  • Change in aBCCdex lesion score [ Time Frame: Baseline, Month 12 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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
Official Title  ICMJE 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
Brief 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 Basal Cell Nevus Syndrome (BCNS; Gorlin Syndrome). Subjects 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 surgically eligible BCCs (nSEBs) that develop over the 12 month period.
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
Partcipants will be randomized 1:1 to receive Patidegib Topical Gel, 2%, or Vehicle (IP)
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:
As a double-blinded study, the Investigators, the site staff, Sponsor, and the Clinical Monitor(s) will be blinded to the treatment assigned to individual participants.
Primary Purpose: Treatment
Condition  ICMJE Basal Cell Nevus Syndrome
Intervention  ICMJE
  • Drug: Patidegib Topical Gel, 2%
    Patidegib Topical Gel, 2%
    Other Name: IP
  • Drug: Patidegib Topical Gel, Vehicle
    Patidegib Topical Gel, Vehicle
    Other Name: IP, Vehicle
Study Arms  ICMJE
  • Experimental: Patidegib Topical Gel, 2%,
    Participants 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.
    Intervention: Drug: Patidegib Topical Gel, 2%
  • Placebo Comparator: Patidegib Topical Gel, Vehicle
    Participants 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.
    Intervention: Drug: Patidegib Topical Gel, Vehicle
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 9, 2018)
150
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 1, 2020
Estimated Primary Completion Date May 1, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. The participant must be age at least 18 years of age at the Screening Visit.
  2. The participant must provide written informed consent prior to any study procedures.
  3. 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:

    1. >2 histologically confirmed BCCs or 1 for participant under age 20.
    2. Odontogenic keratocysts of the jaw confirmed histologically.
    3. ≥3 palmar and/or plantar pits seen at the Screening Visit.
    4. Bilamellar calcification of the falx cerebri present at less than 20 years of age.
    5. Fused, bifid, or markedly splayed ribs.
    6. First degree relative with BCNS.
    7. Patched proteine 1 (PTCH1) mutation predicted to be of functional significance in normal tissue.

    Minor criteria:

    1. Macrocephaly.
    2. Congenital malformations including frontal bossing, cleft lip or palate, "coarse face", moderate to severe hypertelorism.
    3. Skeletal abnormalities detectable clinically: Sprengel deformity, marked pectus deformity, or marked finger syndactyly.
    4. 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.
    5. Ovarian fibroma.
    6. Medulloblastoma (Modification of criteria of V Kimonis et al Am J Med Genet 69: 299-308, 1997).
  4. 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).
  5. The participant is willing to have blood collected to measure circulating drug levels.
  6. 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 an sun protector factor (SPF) of at least 30 daily on all exposed skin sites.
  7. 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.
  8. 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.
  9. The participant is willing for all facial BCCs to be evaluated and treatment recommendations made only by the Investigator.
  10. 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).

Exclusion Criteria:

  1. The subject has previously participated in a clinical trial evaluating patidegib topical gel.
  2. 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:

    1. Topical glucocorticoids of potency greater than 2.5% hydrocortisone or desonide within the 30 days prior to the Screening Visit.
    2. Retinoids (such as etretinate, isotretinoin, acetretin, tazarotene, tretinoic acid, adapalene) systemically or topically, or >5% concentration of an alpha hydroxy acid (such as glycolic acid, lactic acid) within the 3 months prior to the Screening Visit.
    3. 5-fluorouracil, imiquimod, diclofenac, or Ingenol mebutate (except as topical treatment to discrete non-facial BCCs) systemically or topically to the skin within the 3 months prior to the Screening Visit.
    4. Systemic chemotherapy within 1 year prior to the Screening Visit.
    5. Known inhibitors of the Hedgehog signaling pathway (such as vismodegib, sonidegib, itraconazole) topically or systemically within 3 months prior to the Screening Visit.
    6. Photodynamic therapy (PDT) except to localized non-facial, individual BCCs within 3 months prior to the Screening Visit.
    7. High-dose nicotinamide orally within 3 months prior to the Screening Visit.
  3. The participant is known to have a hypersensitivity to any of the ingredients in the study medication formulation.
  4. The participant is unable or unwilling to make a good faith effort to return to the study site for all study visits and tests.
  5. The participant has uncontrolled systemic disease.
  6. 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.
  7. 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.
  8. The participant is a WOCBP who is unwilling or unable to comply with pregnancy prevention measures.
  9. The participant is pregnant or breastfeeding.
  10. 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Sr. Clinical Operations Manager 844-332-5161 clinical@pellepharm.com
Listed Location Countries  ICMJE France,   Netherlands,   Spain,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03703310
Other Study ID Numbers  ICMJE Pelle-926-301
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party PellePharm, Inc.
Study Sponsor  ICMJE PellePharm, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: VP, Clinical Operations PellePharm, Inc.
PRS Account PellePharm, Inc.
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP