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

NCT ID: NCT04893486

Last Updated: 2024-08-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

73 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-05-06

Study Completion Date

2023-03-13

Brief Summary

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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

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Conditions

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BCCs in Gorlin Syndrome Patients

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Active

Group Type EXPERIMENTAL

PTX-022

Intervention Type DRUG

QTORIN 3.9 % Sirolimus Topical Gel

Vehicle

Group Type PLACEBO_COMPARATOR

Vehicle comparator

Intervention Type DRUG

Vehicle comparator topical gel

Interventions

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PTX-022

QTORIN 3.9 % Sirolimus Topical Gel

Intervention Type DRUG

Vehicle comparator

Vehicle comparator topical gel

Intervention Type DRUG

Eligibility Criteria

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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

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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Palvella Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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David Bickers, MD

Role: STUDY_CHAIR

Columbia University

Joyce Teng, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

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Mayo Clinic

Scottsdale, Arizona, United States

Site Status

Burke Pharmaceuticals Research

Hot Springs, Arkansas, United States

Site Status

Stanford Univeristy

Palo Alto, California, United States

Site Status

Park Avenue Dermatology

Orange Park, Florida, United States

Site Status

Ameriderm Research

Ormond Beach, Florida, United States

Site Status

Gwinett Clinical Research Center

Snellville, Georgia, United States

Site Status

Clinical Coordinating Center (Minnesota)

New Brighton, Minnesota, United States

Site Status

Mayo Clinic - Minnesota

Rochester, Minnesota, United States

Site Status

St. Louis University

St Louis, Missouri, United States

Site Status

Duke University

Durham, North Carolina, United States

Site Status

Penn State Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

DermResearch Inc

Austin, Texas, United States

Site Status

University of Utah

Murray, Utah, United States

Site Status

Virginia Clinical Research, Inc.

Norfolk, Virginia, United States

Site Status

St. Pancras Clinical Research

London, , United Kingdom

Site Status

Salford Foundation Trust

Salford, , United Kingdom

Site Status

Countries

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United States United Kingdom

Related Links

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http://weblink.donorperfect.com/clinicaltrialinterest

Sign-up to be contacted about participation in the clinical trial

http://gorlinsyndrome.org/

Gorlin Syndrome Alliance

Other Identifiers

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PALV-04

Identifier Type: -

Identifier Source: org_study_id

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