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NCT Number: NCT05643872

A 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".

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Key information

Conditions

Pachyonychia Congenita Abnormalities, Multiple Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Ectodermal Dysplasia Genetic Diseases, Inborn Nail Diseases Skin Abnormalities Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases

Age range

16 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Minnesota Clinical Study Center

New Brighton, Minnesota, 55112, United States

Location status: Recruiting

Location contact

Steven E Kempers, MD, FAAD

PRINCIPAL_INVESTIGATOR

Study Coordinator

CONTACT

763-502-2929

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

PTX-022

Drug

QTORIN rapamycin 3.9% anhydrous gel

Primary outcomes

  1. Treatment emergent adverse events

    Time frame: 6 months

Secondary outcomes

  1. Pharmacokinetic parameters

    Time frame: Prior to dose, 12 hours and 24 hours

    AUC area under the sirolimus blood concentration-time profile

Study contacts

Contact information is provided by the study sponsor or research team.

Emily Cook

CONTACT

[email protected]

267-738-6366

Sponsors and collaborators

Lead sponsor

Palvella Therapeutics, Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2022
Primary completion
2023
Study completion
2023
First posted
Dec 9, 2022
Registry last updated
Dec 9, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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