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

Purpose

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".

Condition

  • Pachyonychia Congenita

Eligibility

Eligible Ages
Over 16 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Completed the PALV-05 (VAPAUS) study - Agree to contraceptive use

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 Design

Phase
Phase 3
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

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

Recruiting Locations

Minnesota Clinical Study Center
New Brighton, Minnesota 55112
Contact:
Study Coordinator
763-502-2929

More Details

Status
Recruiting
Sponsor
Palvella Therapeutics, Inc.

Study Contact

Emily Cook
267-738-6366
emily.cook@palvellatx.com