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Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04308395
Recruitment Status : Enrolling by invitation
First Posted : March 16, 2020
Last Update Posted : October 19, 2020
Sponsor:
Information provided by (Responsible Party):
PellePharm, Inc.

Brief Summary:
This is a multicenter, open label extension study evaluating the safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult subjects with Gorlin syndrome.

Condition or disease Intervention/treatment Phase
Basal Cell Nevus Syndrome Drug: Patidegib Topical Gel, 2% Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 191 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: All patients will receive Patidegib Topical Gel, 2%,
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Multicenter, Open-Label Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)
Actual Study Start Date : June 3, 2020
Estimated Primary Completion Date : November 30, 2021
Estimated Study Completion Date : November 30, 2021


Arm Intervention/treatment
Experimental: Patidegib Topical Gel, 2%
Patidegib Topical Gel, 2%
Drug: Patidegib Topical Gel, 2%
Patidegib Topical Gel, 2%




Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events (TEAEs) [ Time Frame: 12 months ]

Secondary Outcome Measures :
  1. Change in number of facial BCCs removed by surgery [ Time Frame: 12 months ]
  2. Change in number of new lesions suspicious for BCC [ Time Frame: 12 months ]
  3. Change in Advanced Basal Cell Carcinoma Index (aBCCdex) lesion score [ Time Frame: 12 months ]
  4. Change in Dermatology Life Quality Index (DLQI) [ Time Frame: 12 months ]
  5. Change in EuroQol Group 5-level EQ 5D (EQ 5D 5L) [ Time Frame: 12 months ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. The subject must have completed PellePharm Study Pelle-926-201 or Pelle-926-301.
  2. Study Pelle-926-301 subjects must have completed the End of Treatment Visit in Study 301, prior to the Screening Visit in this study. They must also complete all Study 301 related procedures prior to the Baseline Visit of this study.
  3. 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. Moisturizers and emollients are allowed. Subjects will be encouraged to use their preferred sunscreen with a sunscreen protection factor (SPF) of at least 30 daily on all exposed skin sites.
  4. Female subjects must have a negative pregnancy test. For Study 301 subjects a negative serum pregnancy test result from Study 301 is acceptable if the test was done within 7 days of the Screening Visit of this study.
  5. If the subject is a woman of child bearing potential (WOCBP), she must be willing to use birth control methods which may be considered highly effective. Hormonal contraception must be supplemented with a barrier method (preferably condom). Birth control must start prior to Baseline, continue through the duration of the study, and for 30 days after last application of investigational product (IP).
  6. If the subject is a male with a female sex partner who is a WOCBP, the subject must be willing to use condoms, even after a vasectomy, starting prior to Baseline, through the duration of the study, and for at least 3 months after the last application of IP.
  7. The subject is willing for all facial BCCs to be evaluated and follow treatment recommendations made only by the Investigator.
  8. The subject 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 BCC potentially might compromise the health of the subject. In such instances, the only other allowed form of treatment is surgical.

Exclusion Criteria:

  1. The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study IP.
  2. The subject 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 (while enrolled in this study) participation in an experimental drug study (excluding Study 301).
  3. The subject is a WOCBP who is unwilling or unable to comply with pregnancy prevention measures.
  4. The subject is pregnant or breastfeeding.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04308395


Locations
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United States, California
PellePharm Investigative Site
Fremont, California, United States, 94538
United States, Connecticut
Yale University
New Haven, Connecticut, United States, 06519
United States, Florida
PellePharm Investigative Site
Ormond Beach, Florida, United States, 32174
United States, Indiana
Laser & Skin Surgery Center of Indiana
Indianapolis, Indiana, United States, 46260
United States, Missouri
Saint Louis University
Saint Louis, Missouri, United States, 63104
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
United States, Utah
University of Utah, Medvalley Dermatology
Murray, Utah, United States, 84107
France
Hopital Saint Louis
Paris, France, 75010
Netherlands
PellePharm Investigative Site
Maastricht, Netherlands, 6229 HX
Spain
PellePharm Investigative Site
Barcelona, Spain, 08036
PellePharm Investigative Site
Madrid, Spain, 28023
Sponsors and Collaborators
PellePharm, Inc.
Investigators
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Study Director: Study Director VP, Clinical Operations PellePharm, Inc.
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Responsible Party: PellePharm, Inc.
ClinicalTrials.gov Identifier: NCT04308395    
Other Study ID Numbers: Pelle-926-301E
First Posted: March 16, 2020    Key Record Dates
Last Update Posted: October 19, 2020
Last Verified: October 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by PellePharm, Inc.:
Gorlin syndrome
Patidegib
Basal cell nevus syndrome
Nevoid basal cell carcinoma syndrome
Basal cell carcinoma
Hedgehog
Surgically eligible basal cell carcinomas
BCC
BCNS
Additional relevant MeSH terms:
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Nevus
Nevus, Pigmented
Basal Cell Nevus Syndrome
Syndrome
Disease
Pathologic Processes
Nevi and Melanomas
Neoplasms by Histologic Type
Neoplasms
Odontogenic Cysts
Jaw Cysts
Bone Cysts
Cysts
Carcinoma, Basal Cell
Carcinoma
Neoplasms, Glandular and Epithelial
Neoplasms, Basal Cell
Neoplastic Syndromes, Hereditary
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Jaw Diseases
Stomatognathic Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn