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

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 Overview

• Status: Recruiting
• Conditions: Pachyonychia Congenita
• Intervention / Treatment: Drug: PTX-022

• Study Type: Interventional
• Enrollment (Anticipated): 45
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Emily Cook; Phone Number: 267-738-6366; Email: emily.cook@palvellatx.com

Study Locations

• United States
  • Minnesota
    • New Brighton, Minnesota, United States, 55112
      • Recruiting
      • Minnesota Clinical Study Center
      • Contact: Study Coordinator; Phone Number: 763-502-2929
      • Principal Investigator: Steven E Kempers, MD, FAAD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PTX-022	Drug: PTX-022; QTORIN rapamycin 3.9% anhydrous gel

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Treatment emergent adverse events	6 months

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Palvella Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): November 15, 2022
• Primary Completion (Anticipated): October 1, 2023
• Study Completion (Anticipated): November 1, 2023

Study Registration Dates

• First Submitted: November 8, 2022
• First Submitted That Met QC Criteria: December 1, 2022
• First Posted (Estimate): December 9, 2022

Study Record Updates

• Last Update Posted (Estimate): December 9, 2022
• Last Update Submitted That Met QC Criteria: December 1, 2022
• Last Verified: December 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Pathological Conditions, Anatomical; Skin Diseases, Genetic; Nail Diseases; Skin Abnormalities; Abnormalities, Multiple; Ectodermal Dysplasia; Pachyonychia Congenita; Nails, Malformed
• Other Study ID Numbers: PALV-08

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No