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

Palvella Therapeutics

Status and phase: Enrolling; Phase 3

Conditions: Pachyonychia Congenita

Treatments: Drug: PTX-022

Study type: Interventional

Funder types: Industry

Identifiers

NCT05643872

PALV-08

Details and patient eligibility

About

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".

Enrollment: 45 estimated patients

Sex: All

Ages: 16+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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45 participants in 1 patient group

PTX-022

Experimental group

Treatment:

Drug: PTX-022