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

August 28, 2024 updated by: Palvella Therapeutics, Inc.

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

73

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • London, United Kingdom
        • St. Pancras Clinical Research
      • Salford, United Kingdom, M6 8HD
        • Salford Foundation Trust
  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85259
        • Mayo Clinic
    • Arkansas
      • Hot Springs, Arkansas, United States, 71913
        • Burke Pharmaceuticals Research
    • California
      • Palo Alto, California, United States, 94304
        • Stanford Univeristy
    • Florida
      • Orange Park, Florida, United States, 32073
        • Park Avenue Dermatology
      • Ormond Beach, Florida, United States, 32174
        • Ameriderm Research
    • Georgia
      • Snellville, Georgia, United States, 30078
        • Gwinett Clinical Research Center
    • Minnesota
      • New Brighton, Minnesota, United States, 55112
        • Clinical Coordinating Center (Minnesota)
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic - Minnesota
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • St. Louis University
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke University
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Penn State Hershey Medical Center
    • Texas
      • Austin, Texas, United States, 78759
        • DermResearch Inc
    • Utah
      • Murray, Utah, United States, 84107
        • University of Utah
    • Virginia
      • Norfolk, Virginia, United States, 23502
        • Virginia Clinical Research, Inc.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Vehicle
Drug: Vehicle comparator
Vehicle comparator topical gel
Experimental: Active
Drug: PTX-022
QTORIN 3.9 % Sirolimus Topical Gel

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Palvella Therapeutics, Inc.

Investigators

  • Principal Investigator: Joyce Teng, MD, Stanford University
  • Study Chair: David Bickers, MD, Columbia University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 6, 2021

Primary Completion (Actual)

March 13, 2023

Study Completion (Actual)

March 13, 2023

Study Registration Dates

First Submitted

May 14, 2021

First Submitted That Met QC Criteria

May 14, 2021

First Posted (Actual)

May 19, 2021

Study Record Updates

Last Update Posted (Actual)

August 29, 2024

Last Update Submitted That Met QC Criteria

August 28, 2024

Last Verified

August 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PALV-04

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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