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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

Primary Purpose

BCCs in Gorlin Syndrome Patients

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
PTX-022
Vehicle comparator
Sponsored by
Palvella Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for BCCs in Gorlin Syndrome Patients focused on measuring Gorlin Syndrome, Basal Cell Carcinoma, Nevoid basal cell carcinoma syndrome, Basal cell nevus syndrome BCNS, Gorlin Syndrome Alliance, Prevention, Topical, Sirolimus

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Mayo ClinicRecruiting
  • Burke Pharmaceuticals ResearchRecruiting
  • Stanford UniveristyRecruiting
  • Park Avenue DermatologyRecruiting
  • Ameriderm ResearchRecruiting
  • Gwinett Clinical Research CenterRecruiting
  • Clinical Coordinating Center (Minnesota)Recruiting
  • Mayo Clinic - MinnesotaRecruiting
  • St. Louis UniversityRecruiting
  • Duke UniversityRecruiting
  • Penn State Hershey Medical CenterRecruiting
  • DermResearch IncRecruiting
  • University of UtahRecruiting
  • Virginia Clinical Research, Inc.Recruiting
  • St. Pancras Clinical ResearchRecruiting
  • Salford Foundation TrustRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Active

Vehicle

Arm Description

Outcomes

Primary Outcome Measures

Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms
Incidence of dermatological, treatment emergent adverse events after treatment with active

Secondary Outcome Measures

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

Full Information

First Posted
May 14, 2021
Last Updated
August 26, 2022
Sponsor
Palvella Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04893486
Brief Title
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

2. Study Status

Record Verification Date
August 2022
Overall Recruitment Status
Recruiting
Study Start Date
May 6, 2021 (Actual)
Primary Completion Date
March 3, 2023 (Anticipated)
Study Completion Date
March 31, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Palvella Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
60 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Active
Arm Type
Experimental
Arm Title
Vehicle
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
PTX-022
Intervention Description
QTORIN 3.9 % Sirolimus Topical Gel
Intervention Type
Drug
Intervention Name(s)
Vehicle comparator
Intervention Description
Vehicle comparator topical gel
Primary Outcome Measure Information:
Title
Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms
Time Frame
Month 6
Title
Incidence of dermatological, treatment emergent adverse events after treatment with active
Time Frame
Month 6
Secondary Outcome Measure Information:
Title
Percentage of participants developing 2 or more new biopsy confirmed BCCs on the face.
Time Frame
Month 6
Title
Percentage of participants developing 1 or more new biopsy confirmed BCCs on the face.
Time Frame
Month 6

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Kathy Goin
Phone
484-612-2335
Email
kathy.goin@palvellatx.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
David Bickers, MD
Organizational Affiliation
Columbia University
Official's Role
Study Chair
First Name & Middle Initial & Last Name & Degree
Joyce Teng, MD
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Mayo Clinic
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85259
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Study Coordinator
Facility Name
Burke Pharmaceuticals Research
City
Hot Springs
State/Province
Arkansas
ZIP/Postal Code
71913
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Tim Dugan
Phone
501-620-4449
Email
tdugan@burkepharmaceutical.com
Facility Name
Stanford Univeristy
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Elidia Tafoya
Facility Name
Park Avenue Dermatology
City
Orange Park
State/Province
Florida
ZIP/Postal Code
32073
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Dea Kemlage
Phone
904-458-0411
Email
dkemlage@parkavedermatology.com
Facility Name
Ameriderm Research
City
Ormond Beach
State/Province
Florida
ZIP/Postal Code
32174
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sandra Warrington
Phone
386-523-0768
Email
swarrington@leavittmgt.com
Facility Name
Gwinett Clinical Research Center
City
Snellville
State/Province
Georgia
ZIP/Postal Code
30078
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Colleen Ethridge
Phone
770-972-2241
Email
gcrcce@gmail.com
Facility Name
Clinical Coordinating Center (Minnesota)
City
New Brighton
State/Province
Minnesota
ZIP/Postal Code
55112
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Patient Navigator
Phone
763-571-4200
Email
Gorlin.Codytrial@associatedskincare.com
Facility Name
Mayo Clinic - Minnesota
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Addison Demer
Facility Name
St. Louis University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rhonda Lebbing
Phone
314-977-1794
Email
Rhonda.Lebbing@health.slu.edu
Facility Name
Duke University
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Connor Whatley
Phone
919-681-8368
Email
connor.whatley@duke.edu
Facility Name
Penn State Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Study Coordinator
Facility Name
DermResearch Inc
City
Austin
State/Province
Texas
ZIP/Postal Code
78759
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Karina Garcia
Phone
512-349-9889 x105
Email
karina@driresearchsite.com
Facility Name
University of Utah
City
Murray
State/Province
Utah
ZIP/Postal Code
84107
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sean Wetland
Phone
801-213-3427
Email
sean.wentland@hsc.utah.edu
Facility Name
Virginia Clinical Research, Inc.
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23502
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Erin Rivera
Phone
757-625-0151
Email
erivera@vcrinc.org
Facility Name
St. Pancras Clinical Research
City
London
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Stuart Ratcliff, MD
Phone
0203 865 1142
Email
stuartratcliffe@stpancrasclinical.com
Facility Name
Salford Foundation Trust
City
Salford
ZIP/Postal Code
M6 8HD
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Study Coordinator

12. IPD Sharing Statement

Links:
URL
http://gorlinsyndrome.org/
Description
Gorlin Syndrome Alliance

Learn more about this trial

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

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