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CODY: A Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel For The Prevention Of Basal Cell Carcinomas (BCCs) In Patients With Gorlin 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: NCT04893486
Recruitment Status : Recruiting
First Posted : May 19, 2021
Last Update Posted : May 6, 2022
Sponsor:
Information provided by (Responsible Party):
Palvella Therapeutics, Inc.

Brief Summary:
A Phase 2b study looks at the safety and efficacy of a treatment that is being investigated for people with certain diseases. This study will be conducted at multiple centers in the United States where participants with Gorlin Syndrome, also known as basal cell nevus syndrome, will be randomly placed into two groups; one group will receive the active topical gel, the other a topical vehicle gel, also know as placebo. Participants will apply this topical product to their face once a day for 6 months. The study will be looking at the number of new BCCs that develop on the faces of all the participants during this time.

Condition or disease Intervention/treatment Phase
BCCs in Gorlin Syndrome Patients Drug: PTX-022 Drug: Vehicle comparator Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
Official Title: A Multicenter, Phase 2B, Randomized, Double-Blind, Stratified, Vehicle-Controlled Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel In Prevention Of BCCs In Patients With Gorlin Syndrome
Actual Study Start Date : May 6, 2021
Estimated Primary Completion Date : August 30, 2022
Estimated Study Completion Date : September 30, 2022


Arm Intervention/treatment
Experimental: Active Drug: PTX-022
QTORIN 3.9 % Sirolimus Topical Gel

Placebo Comparator: Vehicle Drug: Vehicle comparator
Vehicle comparator topical gel




Primary Outcome Measures :
  1. Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms [ Time Frame: Month 6 ]
  2. Incidence of dermatological, treatment emergent adverse events after treatment with active [ Time Frame: Month 6 ]

Secondary Outcome Measures :
  1. Percentage of participants developing 2 or more new biopsy confirmed BCCs on the face. [ Time Frame: Month 6 ]
  2. Percentage of participants developing 1 or more new biopsy confirmed BCCs on the face. [ Time Frame: Month 6 ]


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

Key Inclusion Criteria:

  • Adults, 18 years or older
  • Meet diagnostic criteria for Gorlin Syndrome
  • Willing to avoid application of a non-study topical medication to the face (prescription or over the counter) during the study.
  • Willing to forego treatment of BCCs with anything other than the study medication except when the Investigator believes that delay of treatment of a BCC potentially might compromise the health of the subject. During the trial, the only allowed form of BCC treatment is surgical.
  • Participant will be encouraged to use their preferred sunscreen with a sun protector factor (SPF) of at least 30 daily

Key Exclusion Criteria:

  • Recently participated in a clinical trial evaluating an investigational product for the treatment of BCCs or Gorlin Syndrome within 3 months prior to to starting study medication. Participant can start the study screening period prior to completing the 3 month washout.
  • Recently used topical or systemic (oral) treatment that might interfere with the evaluation of the study medication.

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): NCT04893486


Contacts
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Contact: Kathy Goin 484-612-2335 kathy.goin@palvellatx.com

Locations
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United States, Arizona
Mayo Clinic Not yet recruiting
Scottsdale, Arizona, United States, 85259
Contact: Study Coordinator         
United States, Arkansas
Burke Pharmaceuticals Research Recruiting
Hot Springs, Arkansas, United States, 71913
Contact: Tim Dugan    501-620-4449    tdugan@burkepharmaceutical.com   
United States, California
Stanford Univeristy Not yet recruiting
Palo Alto, California, United States, 94304
Contact: Elidia Tafoya         
United States, Florida
Park Avenue Dermatology Recruiting
Orange Park, Florida, United States, 32073
Contact: Dea Kemlage    904-458-0411    dkemlage@parkavedermatology.com   
Ameriderm Research Recruiting
Ormond Beach, Florida, United States, 32174
Contact: Sandra Warrington    386-523-0768    swarrington@leavittmgt.com   
United States, Georgia
Gwinett Clinical Research Center Recruiting
Snellville, Georgia, United States, 30078
Contact: Colleen Ethridge    770-972-2241    gcrcce@gmail.com   
United States, Minnesota
Clinical Coordinating Center (Minnesota) Recruiting
New Brighton, Minnesota, United States, 55112
Contact: Patient Navigator    763-571-4200    Gorlin.Codytrial@associatedskincare.com   
Mayo Clinic - Minnesota Not yet recruiting
Rochester, Minnesota, United States, 55905
Contact: Addison Demer         
United States, Missouri
St. Louis University Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Rhonda Lebbing    314-977-1794    Rhonda.Lebbing@health.slu.edu   
United States, North Carolina
Duke University Recruiting
Durham, North Carolina, United States, 27705
Contact: Connor Whatley    919-681-8368    connor.whatley@duke.edu   
United States, Pennsylvania
Penn State Hershey Medical Center Not yet recruiting
Hershey, Pennsylvania, United States, 17033
Contact: Study Coordinator         
United States, Texas
DermResearch Inc Recruiting
Austin, Texas, United States, 78759
Contact: Karina Garcia    512-349-9889 x105    karina@driresearchsite.com   
United States, Utah
University of Utah Recruiting
Murray, Utah, United States, 84107
Contact: Sean Wetland    801-213-3427    sean.wentland@hsc.utah.edu   
United States, Virginia
Virginia Clinical Research, Inc. Recruiting
Norfolk, Virginia, United States, 23502
Contact: Erin Rivera    757-625-0151    erivera@vcrinc.org   
United Kingdom
St. Pancras Clinical Research Recruiting
London, United Kingdom
Contact: Stuart Ratcliff, MD    0203 865 1142    stuartratcliffe@stpancrasclinical.com   
Salford Foundation Trust Recruiting
Salford, United Kingdom, M6 8HD
Contact: Study Coordinator         
Sponsors and Collaborators
Palvella Therapeutics, Inc.
Investigators
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Study Chair: David Bickers, MD Columbia University
Principal Investigator: Joyce Teng, MD Stanford University
Additional Information:
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Responsible Party: Palvella Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04893486    
Other Study ID Numbers: PALV-04
First Posted: May 19, 2021    Key Record Dates
Last Update Posted: May 6, 2022
Last Verified: May 2022

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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 Palvella Therapeutics, Inc.:
Gorlin Syndrome
Basal Cell Carcinoma
Nevoid basal cell carcinoma syndrome
Basal cell nevus syndrome BCNS
Gorlin Syndrome Alliance
Prevention
Topical
Sirolimus
Additional relevant MeSH terms:
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Carcinoma, Basal Cell
Basal Cell Nevus Syndrome
Syndrome
Disease
Pathologic Processes
Carcinoma
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Basal Cell
Odontogenic Cysts
Jaw Cysts
Bone Cysts
Cysts
Neoplastic Syndromes, Hereditary
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Jaw Diseases
Stomatognathic Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn