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

Pachyonychia Congenita Clinical Trial

— 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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05180708
• Other study ID #: PALV-05
• Status: Recruiting
• Phase: Phase 3
• Start date: November 29, 2021
• Est. completion date: June 30, 2023

Study information

• Verified date: August 2022
• Source: Palvella Therapeutics, Inc.
• Contact: Emily Cook
• Phone: 484-253-1463
• Email: emily.cook[@]palvellatx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 80
• Est. completion date: June 30, 2023
• Est. primary completion date: June 30, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Pachyonychia Congenita

Intervention

Drug:
• QTORIN 3.9% rapamycin anhydrous gel
QTORIN 3.9% rapamycin anhydrous gel
• Vehicle
Matching vehicle (QTORIN anyhydrous gel)

Locations

Country	Name	City	State
United Kingdom	Royal London Hospital	London	Whitechapel
United States	DermResearch, Inc.	Austin	Texas
United States	Minnesota Clinical Study Center	Fridley	Minnesota
United States	University of Utah	Murray	Utah
United States	Yale University	New Haven	Connecticut
United States	Park Avenue Dermatology	Orange Park	Florida
United States	Stanford University	Palo Alto	California
United States	Oregon Health and Sciences University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Palvella Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Patient Global Assessment of Activities Difficulty Scale		6 months
Secondary	Pain at its worst as assessed by numerical rating scale		6 months
Secondary	PROMIS physical function		6 months
Secondary	Patient global impression of severity (PGI-S)		6 months
Secondary	Patient global impression of change (PGI-C)		6 months
Secondary	Clinician global impression of severity (CGI-S)		6 months
Secondary	Clinician global impression of change (CGI-C)		6 months

External Links

•   Palvella Therapeutics, Inc.
•   PC Project