RecruitingPhase 3NCT05643872

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


Sponsor

Palvella Therapeutics, Inc.

Enrollment

45 participants

Start Date

Nov 15, 2022

Study Type

INTERVENTIONAL

Conditions

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".


Eligibility

Min Age: 16 Years

Inclusion Criteria2

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

Exclusion Criteria4

  • 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

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Interventions

DRUGPTX-022

QTORIN rapamycin 3.9% anhydrous gel


Locations(1)

Minnesota Clinical Study Center

New Brighton, Minnesota, United States

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NCT05643872


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