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Completed

NCT Number: NCT04893486

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

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.

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

St. Pancras Clinical Research, London, United Kingdom

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

PTX-022

Drug

QTORIN 3.9 % Sirolimus Topical Gel

Vehicle comparator

Drug

Vehicle comparator topical gel

Primary outcomes

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

    Time frame: Month 6

  2. Incidence of dermatological, treatment emergent adverse events after treatment with active

    Time frame: Month 6

Secondary outcomes

  1. Percentage of participants developing 2 or more new biopsy confirmed BCCs on the face.

    Time frame: Month 6

  2. Percentage of participants developing 1 or more new biopsy confirmed BCCs on the face.

    Time frame: Month 6

Sponsors and collaborators

Lead sponsor

Palvella Therapeutics, Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2021
Primary completion
2023
Study completion
2023
First posted
May 19, 2021
Registry last updated
Aug 29, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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