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 | Completed Phase 2 FDA Drug

• 1 other identifier: PALV-04
• Study Type: interventional
• Target: 73
• Locations: 2 countries
• Sites: 16

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.

Conditions

BCCs in Gorlin Syndrome Patients

Keywords

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

Outcome Measures

Primary Outcomes (2)

• Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms

  Month 6

• Incidence of dermatological, treatment emergent adverse events after treatment with active

  Month 6

Secondary Outcomes (2)

• 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

Study Arms (2)

Active

EXPERIMENTAL

Drug: PTX-022

Vehicle

PLACEBO COMPARATOR

Drug: Vehicle comparator

Interventions

PTX-022 DRUG

QTORIN 3.9 % Sirolimus Topical Gel

Active

Vehicle comparator DRUG

Vehicle comparator topical gel

Vehicle

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Palvella Therapeutics, Inc.: lead

Study Sites (16)

Mayo Clinic

Scottsdale, Arizona, 85259, United States

Location

Burke Pharmaceuticals Research

Hot Springs, Arkansas, 71913, United States

Location

Stanford Univeristy

Palo Alto, California, 94304, United States

Location

Park Avenue Dermatology

Orange Park, Florida, 32073, United States

Location

Ameriderm Research

Ormond Beach, Florida, 32174, United States

Location

Gwinett Clinical Research Center

Snellville, Georgia, 30078, United States

Location

Clinical Coordinating Center (Minnesota)

New Brighton, Minnesota, 55112, United States

Location

Mayo Clinic - Minnesota

Rochester, Minnesota, 55905, United States

Location

St. Louis University

St Louis, Missouri, 63110, United States

Location

Duke University

Durham, North Carolina, 27705, United States

Location

Penn State Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

DermResearch Inc

Austin, Texas, 78759, United States

Location

University of Utah

Murray, Utah, 84107, United States

Location

Virginia Clinical Research, Inc.

Norfolk, Virginia, 23502, United States

Location

St. Pancras Clinical Research

London, United Kingdom

Location

Salford Foundation Trust

Salford, M6 8HD, United Kingdom

Location

Related Links

• Sign-up to be contacted about participation in the clinical trial
• Gorlin Syndrome Alliance

MeSH Terms

Conditions

Basal Cell Nevus Syndrome; Carcinoma, Basal Cell

Condition Hierarchy (Ancestors)

Odontogenic Cysts; Jaw Cysts; Bone Cysts; Cysts; Neoplasms; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms, Basal Cell; Neoplastic Syndromes, Hereditary; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Jaw Diseases; Stomatognathic Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Officials

• David Bickers, MD

  Columbia University

  STUDY CHAIR

• Joyce Teng, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 14, 2021
• First Posted: May 19, 2021
• Study Start: May 6, 2021
• Primary Completion: March 13, 2023
• Study Completion: March 13, 2023
• Last Updated: August 29, 2024

Record last verified: 2024-08

Locations

GB (2); US (14)