Clinical Trials /

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

NCT04893486

Description:

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.

Related Conditions:
  • Basal Cell Carcinoma
Recruiting Status:

Recruiting

Phase:

Phase 2

URL:

https://clinicaltrials.gov/show/NCT04893486

Trial Eligibility

Document

Title

  • Brief Title: 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
  • 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

Clinical Trial IDs

  • ORG STUDY ID: PALV-04
  • NCT ID: NCT04893486

Conditions

  • BCCs in Gorlin Syndrome Patients

Interventions

DrugSynonymsArms
PTX-022Active
Vehicle comparatorVehicle

Purpose

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.

Trial Arms

NameTypeDescriptionInterventions
ActiveExperimental
  • PTX-022
VehiclePlacebo Comparator
  • Vehicle comparator

Eligibility Criteria

        Key Inclusion Criteria:

          -  Adults, 18 years or older

          -  Live in the United States

          -  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.
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms
Time Frame:Month 6
Safety Issue:
Description:

Secondary Outcome Measures

Measure:Percentage of participants developing 2 or more new biopsy confirmed BCCs on the face.
Time Frame:Month 6
Safety Issue:
Description:
Measure:Percentage of participants developing 1 or more new biopsy confirmed BCCs on the face.
Time Frame:Month 6
Safety Issue:
Description:

Details

Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Palvella Therapeutics, Inc.

Trial Keywords

  • Gorlin Syndrome
  • Basal Cell Carcinoma
  • Nevoid basal cell carcinoma syndrome
  • Basal cell nevus syndrome BCNS
  • Gorlin Syndrome Alliance
  • Prevention
  • Topical
  • Sirolimus

Last Updated

May 20, 2021