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)

August 26, 2022 updated by: Palvella Therapeutics, Inc.

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

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

80

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United Kingdom
    • Whitechapel
      • London, Whitechapel, United Kingdom, E11FR
        • Recruiting
        • Royal London Hospital
        • Principal Investigator:
          • Edel O'Toole, MB, PhD, FRCP
  • United States
    • California
      • Palo Alto, California, United States, 94304
        • Recruiting
        • Stanford University
        • Contact:
        • Principal Investigator:
          • Joyce Teng, MD
    • Connecticut
      • New Haven, Connecticut, United States, 06519
        • Recruiting
        • Yale University
        • Contact:
        • Principal Investigator:
          • Christopher Bunick, MD
    • Florida
      • Orange Park, Florida, United States, 32073
    • Minnesota
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health and Sciences University
        • Contact:
        • Principal Investigator:
          • Tracy Funk, MD
    • Texas
      • Austin, Texas, United States, 78759
    • Utah
      • Murray, Utah, United States, 84107
        • Recruiting
        • University of Utah
        • Contact:
        • Principal Investigator:
          • David Hansen, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

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
Time Frame: 6 months
6 months

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

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

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 29, 2021

Primary Completion (Anticipated)

June 30, 2023

Study Completion (Anticipated)

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, 2022

Last Update Submitted That Met QC Criteria

August 26, 2022

Last Verified

August 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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