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

Pachyonychia Congenita Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05643872
• Other study ID #: PALV-08
• Status: Recruiting
• Phase: Phase 3
• Start date: November 15, 2022
• Est. completion date: November 2023

Study information

• Verified date: December 2022
• Source: Palvella Therapeutics, Inc.
• Contact: Emily Cook
• Phone: 267-738-6366
• Email: emily.cook[@]palvellatx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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".

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 45
• Est. completion date: November 2023
• Est. primary completion date: October 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Pachyonychia Congenita

Intervention

Drug:
• PTX-022
QTORIN rapamycin 3.9% anhydrous gel

Locations

Country	Name	City	State
United States	Minnesota Clinical Study Center	New Brighton	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Palvella Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Treatment emergent adverse events		6 months
Secondary	Pharmacokinetic parameters	AUC area under the sirolimus blood concentration-time profile	Prior to dose, 12 hours and 24 hours