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

Palvella Therapeutics

Status and phase: Enrolling; Phase 2

Conditions: BCCs in Gorlin Syndrome Patients

Treatments: Drug: Vehicle comparator; Drug: PTX-022

Study type: Interventional

Funder types: Industry

Identifiers

NCT04893486

PALV-04

Details and patient eligibility

About

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.

Enrollment: 60 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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60 participants in 2 patient groups, including a placebo group

Active

Experimental group

Treatment:

Drug: PTX-022

Vehicle

Placebo Comparator group

Treatment:

Drug: Vehicle comparator