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)

Palvella Therapeutics

Status and phase: Enrolling; Phase 3

Conditions: Pachyonychia Congenita

Treatments: Drug: Vehicle; Drug: QTORIN 3.9% rapamycin anhydrous gel

Study type: Interventional

Funder types: Industry

Identifiers

NCT05180708

PALV-05

Details and patient eligibility

About

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.

Enrollment: 80 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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80 participants in 2 patient groups, including a placebo group

Active

Active Comparator group

Treatment:

Drug: QTORIN 3.9% rapamycin anhydrous gel

Vehicle

Placebo Comparator group

Treatment:

Drug: Vehicle