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

Phase 3RecruitingNCT05643872

Palvella Therapeutics, Inc.45 enrolled

Overview

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".

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Pachyonychia Congenita

Interventions

PTX-022DRUG

QTORIN rapamycin 3.9% anhydrous gel

Eligibility

Sex: ALLMin age: 16 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Treatment emergent adverse events

Time frame: 6 months

Secondary Outcomes

Pharmacokinetic parameters

AUC area under the sirolimus blood concentration-time profile

Time frame: Prior to dose, 12 hours and 24 hours