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)

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

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.

Study Overview

• Status: Completed
• Conditions: Pachyonychia Congenita
• Intervention / Treatment: Drug: QTORIN 3.9% rapamycin anhydrous gel; Drug: Vehicle

• Study Type: Interventional
• Enrollment (Actual): 87
• Phase: Phase 3

Contacts and Locations

Study Locations

• United Kingdom
  • Whitechapel
    • London, Whitechapel, United Kingdom, E11FR
      • Royal London Hospital
• United States
  • California
    • Palo Alto, California, United States, 94304
      • Stanford University
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale University
  • Florida
    • Orange Park, Florida, United States, 32073
      • Park Avenue Dermatology
  • Minnesota
    • Fridley, Minnesota, United States, 55432
      • Minnesota Clinical Study Center
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health and Sciences University
  • Texas
    • Austin, Texas, United States, 78759
      • Dermresearch, Inc.
  • Utah
    • Murray, Utah, United States, 84107
      • University of Utah

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Vehicle	Drug: Vehicle; Matching vehicle (QTORIN anyhydrous gel)
Active Comparator: Active	Drug: QTORIN 3.9% rapamycin anhydrous gel; QTORIN 3.9% rapamycin anhydrous gel

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Patient Global Assessment of Activities Difficulty Scale	6 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Pain at its worst as assessed by numerical rating scale	6 months
PROMIS physical function	6 months
Patient global impression of severity (PGI-S)	6 months
Patient global impression of change (PGI-C)	6 months
Clinician global impression of severity (CGI-S)	6 months
Clinician global impression of change (CGI-C)	6 months

Collaborators and Investigators

• Sponsor: Palvella Therapeutics, Inc.
• Investigators: Study Chair: Amy Paller, MD, Northwestern University; Principal Investigator: Joyce Teng, MD, Stanford University; Principal Investigator: David Hansen, MD, University of Utah

Publications and helpful links

Helpful Links

• PC Project
• Palvella Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): November 29, 2021
• Primary Completion (Actual): June 30, 2023
• Study Completion (Actual): June 30, 2023

Study Registration Dates

• First Submitted: December 20, 2021
• First Submitted That Met QC Criteria: January 5, 2022
• First Posted (Actual): January 6, 2022

Study Record Updates

• Last Update Posted (Actual): August 29, 2024
• Last Update Submitted That Met QC Criteria: August 28, 2024
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Pathological Conditions, Anatomical; Skin Diseases, Genetic; Nail Diseases; Skin Abnormalities; Abnormalities, Multiple; Ectodermal Dysplasia; Pachyonychia Congenita; Nails, Malformed; Physiological Effects of Drugs; Anti-Infective Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Anti-Bacterial Agents; Antibiotics, Antineoplastic; Antifungal Agents; Sirolimus
• Other Study ID Numbers: PALV-05

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No