A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita

• Status: Recruiting
• Trial end date: 2023-11-01
• Target enrollment: 0
• Participant gender: All

Summary

PALV-08 is a multicenter, open-label treatment (OLT) study enrolling adults with Pachyonychia Congenita (PC) with genotyped keratin mutations KRT6A, KRT6B, KRT6C or KRT16 who were previously enrolled in the PALV-05 (VAPAUS) trial.

The purpose of this OLT study is to investigate the safety of long term exposure and pharmacokinetics (PK) of QTORIN rapamycin 3.9% anhydrous gel or "PTX-022".

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

Details

• Lead Sponsor: Palvella Therapeutics, Inc.

Criteria

Key Inclusion Criteria:

• Completed the PALV-05 (VAPAUS) study

• Agree to contraceptive use

Key Exclusion Criteria:

• Females who are pregnant or breastfeeding

• Concomitant use of sirolimus or any sirolimus-containing medications (systemic or topical)

• Any significant concurrent condition that could adversely affect participation and/or the assessment of the safety and efficacy in the study

• Prior or planned treatment, including surgery or other medically necessary intervention deemed by the investigator that could adversely affect participation and/or the assessment of the safety and efficacy in the study