Clinical trial 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
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
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
Lead sponsor: Palvella Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2021-05-19
Enrollment: 73
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

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.


Eligibility 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.

Primary outcomes:
measure: Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms
timeFrame: Month 6
measure: Incidence of dermatological, treatment emergent adverse events after treatment with active
timeFrame: Month 6
Secondary outcomes:
measure: Percentage of participants developing 2 or more new biopsy confirmed BCCs on the face.
timeFrame: Month 6
measure: Percentage of participants developing 1 or more new biopsy confirmed BCCs on the face.
timeFrame: Month 6
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Mayo Clinic
state: Arizona
city: Hot Springs
country: United States
facility: Burke Pharmaceuticals Research
state: Arkansas
city: Palo Alto
country: United States
facility: Stanford Univeristy
state: California
city: Orange Park
country: United States
facility: Park Avenue Dermatology
state: Florida
city: Ormond Beach
country: United States
facility: Ameriderm Research
state: Florida
city: Snellville
country: United States
facility: Gwinett Clinical Research Center
state: Georgia
city: New Brighton
country: United States
facility: Clinical Coordinating Center (Minnesota)
state: Minnesota
city: Rochester
country: United States
facility: Mayo Clinic - Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: St. Louis University
state: Missouri
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Hershey
country: United States
facility: Penn State Hershey Medical Center
state: Pennsylvania
city: Austin
country: United States
facility: DermResearch Inc
state: Texas
city: Murray
country: United States
facility: University of Utah
state: Utah
city: Norfolk
country: United States
facility: Virginia Clinical Research, Inc.
state: Virginia
city: London
country: United Kingdom
facility: St. Pancras Clinical Research
city: Salford
country: United Kingdom
facility: Salford Foundation Trust
Study officials:
affiliation: Columbia University
name: David Bickers, MD
role: STUDY_CHAIR
affiliation: Stanford University
name: Joyce Teng, MD
role: PRINCIPAL_INVESTIGATOR
References: