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A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita (VAPAUS)

Primary Purpose

Pachyonychia Congenita

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
QTORIN 3.9% rapamycin anhydrous gel
Vehicle
Sponsored by
Palvella Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pachyonychia Congenita

Eligibility Criteria

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

Key Inclusion Criteria:

  • Adult patients, 18 years or older (16 years or older in the UK)
  • Diagnosed with Pachyonychia Congenita (PC), genetically confirmed

Key Exclusion Criteria:

  • Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications.
  • Any significant concurrent condition (including conditions involving or inferior to the ankle) that could adversely affect the patient's participation and/or the assessment of the safety and efficacy in the study.
  • Participation in any other interventional clinical research trial within 30 days prior to the screening visit or during the patient's participation in this study

Sites / Locations

  • Stanford UniversityRecruiting
  • Yale UniversityRecruiting
  • Park Avenue DermatologyRecruiting
  • Minnesota Clinical Study CenterRecruiting
  • Oregon Health and Sciences UniversityRecruiting
  • DermResearch, Inc.Recruiting
  • University of UtahRecruiting
  • Royal London HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Active

Vehicle

Arm Description

Outcomes

Primary Outcome Measures

Patient Global Assessment of Activities Difficulty Scale

Secondary Outcome Measures

Pain at its worst as assessed by numerical rating scale
PROMIS physical function
Patient global impression of severity (PGI-S)
Patient global impression of change (PGI-C)
Clinician global impression of severity (CGI-S)
Clinician global impression of change (CGI-C)

Full Information

First Posted
December 20, 2021
Last Updated
August 26, 2022
Sponsor
Palvella Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05180708
Brief Title
A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita
Acronym
VAPAUS
Official Title
A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita
Study Type
Interventional

2. Study Status

Record Verification Date
August 2022
Overall Recruitment Status
Recruiting
Study Start Date
November 29, 2021 (Actual)
Primary Completion Date
June 30, 2023 (Anticipated)
Study Completion Date
June 30, 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

5. Study Description

Brief Summary
This study evaluates the safety and efficacy of QTORIN 3.9% rapamycin anhydrous gel in the treatment of adults with Pachyonychia Congenita. This study includes a screening period, baseline period and 6-month treatment period.

6. Conditions and Keywords

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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
80 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Active
Arm Type
Active Comparator
Arm Title
Vehicle
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
QTORIN 3.9% rapamycin anhydrous gel
Intervention Description
QTORIN 3.9% rapamycin anhydrous gel
Intervention Type
Drug
Intervention Name(s)
Vehicle
Intervention Description
Matching vehicle (QTORIN anyhydrous gel)
Primary Outcome Measure Information:
Title
Patient Global Assessment of Activities Difficulty Scale
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Pain at its worst as assessed by numerical rating scale
Time Frame
6 months
Title
PROMIS physical function
Time Frame
6 months
Title
Patient global impression of severity (PGI-S)
Time Frame
6 months
Title
Patient global impression of change (PGI-C)
Time Frame
6 months
Title
Clinician global impression of severity (CGI-S)
Time Frame
6 months
Title
Clinician global impression of change (CGI-C)
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Adult patients, 18 years or older (16 years or older in the UK) Diagnosed with Pachyonychia Congenita (PC), genetically confirmed Key Exclusion Criteria: Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications. Any significant concurrent condition (including conditions involving or inferior to the ankle) that could adversely affect the patient's participation and/or the assessment of the safety and efficacy in the study. Participation in any other interventional clinical research trial within 30 days prior to the screening visit or during the patient's participation in this study
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Emily Cook
Phone
484-253-1463
Email
emily.cook@palvellatx.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Amy Paller, MD
Organizational Affiliation
Northwestern 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
First Name & Middle Initial & Last Name & Degree
David Hansen, MD
Organizational Affiliation
University of Utah
Official's Role
Principal Investigator
Facility Information:
Facility Name
Stanford University
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
Email
etafoya@stanford.edu
First Name & Middle Initial & Last Name & Degree
Joyce Teng, MD
Facility Name
Yale University
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06519
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nicole Olszweksi
Email
nicole.olszewski@yale.edu
First Name & Middle Initial & Last Name & Degree
Christopher Bunick, MD
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
Rochelle Carver
Email
rcarver@parkavedermatology.com
First Name & Middle Initial & Last Name & Degree
George Schmieder, DO
Facility Name
Minnesota Clinical Study Center
City
Fridley
State/Province
Minnesota
ZIP/Postal Code
55432
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Alissa Prochniak
Email
Aprochniak@associatedskincare.com
First Name & Middle Initial & Last Name & Degree
Steven Kempers, MD
Facility Name
Oregon Health and Sciences University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Amanda Mundy
Email
mundya@ohsu.edu
First Name & Middle Initial & Last Name & Degree
Tracy Funk, MD
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
Email
karina@driresearchsite.com
First Name & Middle Initial & Last Name & Degree
Janet DuBois, MD
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 Wentland
Email
u0156987@utah.edu
First Name & Middle Initial & Last Name & Degree
David Hansen, MD
Facility Name
Royal London Hospital
City
London
State/Province
Whitechapel
ZIP/Postal Code
E11FR
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Edel O'Toole, MB, PhD, FRCP

12. IPD Sharing Statement

Links:
URL
http://www.pachyonychia.org/
Description
PC Project
URL
http://palvellatx.com
Description
Palvella Therapeutics, Inc.

Learn more about this trial

A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita

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