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OpenTrials
Completed

NCT Number: NCT05180708

A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita

This study evaluates the safety and efficacy of QTORIN 3.9% rapamycin anhydrous gel in the treatment of adults with Pachyonychia Congenita. This study includes a screening period, baseline period and 6-month treatment period.

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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

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Royal London Hospital, London, Whitechapel, United Kingdom

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

  • Adult patients, 18 years or older (16 years or older in the UK)
  • Diagnosed with Pachyonychia Congenita (PC), genetically confirmed

Key Exclusion Criteria:

  • Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications.
  • Any significant concurrent condition (including conditions involving or inferior to the ankle) that could adversely affect the patient's participation and/or the assessment of the safety and efficacy in the study.
  • Participation in any other interventional clinical research trial within 30 days prior to the screening visit or during the patient's participation in this study

Treatment and study plan

QTORIN 3.9% rapamycin anhydrous gel

Drug

QTORIN 3.9% rapamycin anhydrous gel

Vehicle

Drug

Matching vehicle (QTORIN anyhydrous gel)

Primary outcomes

  1. Patient Global Assessment of Activities Difficulty Scale

    Time frame: 6 months

Secondary outcomes

  1. Pain at its worst as assessed by numerical rating scale

    Time frame: 6 months

  2. PROMIS physical function

    Time frame: 6 months

  3. Patient global impression of severity (PGI-S)

    Time frame: 6 months

  4. Patient global impression of change (PGI-C)

    Time frame: 6 months

  5. Clinician global impression of severity (CGI-S)

    Time frame: 6 months

  6. Clinician global impression of change (CGI-C)

    Time frame: 6 months

Sponsors and collaborators

Lead sponsor

Palvella Therapeutics, Inc.

Industry

Registry information

Acronym: VAPAUS

Important dates

Study start
2021
Primary completion
2023
Study completion
2023
First posted
Jan 6, 2022
Registry last updated
Aug 29, 2024

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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