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

• Status: Recruiting
• Trial end date: 2023-06-30
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Palvella Therapeutics, Inc.
• Treatments: Sirolimus

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