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

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

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05643872

Enrollment

Registered

2022-12-09

Start date

2022-11-15

Completion date

2023-11-30

Last updated

2022-12-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pachyonychia Congenita

Brief 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.

Interventions

DRUGPTX-022

QTORIN rapamycin 3.9% anhydrous gel

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

16 Years to No maximum

Healthy volunteers

Inclusion 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

Design outcomes

Primary

Measure	Time frame
Treatment emergent adverse events	6 months

Secondary

Measure	Time frame	Description
Pharmacokinetic parameters	Prior to dose, 12 hours and 24 hours	AUC area under the sirolimus blood concentration-time profile

Countries

United States

Contacts

Primary ContactEmily Cook

emily.cook@palvellatx.com267-738-6366

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026