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
- Conditions
- BCCs in Gorlin Syndrome Patients
- Interventions
- Drug: Vehicle comparator
- Registration Number
- NCT04893486
- Lead Sponsor
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 73
- 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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Active PTX-022 - Vehicle Vehicle comparator -
- Primary Outcome Measures
Name Time Method Incidence of dermatological, treatment emergent adverse events after treatment with active Month 6 Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms Month 6
- Secondary Outcome Measures
Name Time Method Percentage of participants developing 2 or more new biopsy confirmed BCCs on the face. Month 6 Percentage of participants developing 1 or more new biopsy confirmed BCCs on the face. Month 6
Trial Locations
- Locations (16)
DermResearch Inc
🇺🇸Austin, Texas, United States
Mayo Clinic
🇺🇸Scottsdale, Arizona, United States
Virginia Clinical Research, Inc.
🇺🇸Norfolk, Virginia, United States
Clinical Coordinating Center (Minnesota)
🇺🇸New Brighton, Minnesota, United States
Penn State Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
St. Louis University
🇺🇸Saint Louis, Missouri, United States
Park Avenue Dermatology
🇺🇸Orange Park, Florida, United States
Burke Pharmaceuticals Research
🇺🇸Hot Springs, Arkansas, United States
Salford Foundation Trust
🇬🇧Salford, United Kingdom
University of Utah
🇺🇸Murray, Utah, United States
Stanford Univeristy
🇺🇸Palo Alto, California, United States
Ameriderm Research
🇺🇸Ormond Beach, Florida, United States
St. Pancras Clinical Research
🇬🇧London, United Kingdom
Mayo Clinic - Minnesota
🇺🇸Rochester, Minnesota, United States
Duke University
🇺🇸Durham, North Carolina, United States
Gwinett Clinical Research Center
🇺🇸Snellville, Georgia, United States