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NCT Number: NCT03920228

Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita

This study evaluates the safety and efficacy of PTX-022, topical rapamycin, in the treatment of adults with moderate to severe Pachyonychia Congenita.

This study includes four-parts, and if a participant completes all parts, the participant will have received at least 3-months of PTX-022 treatment.

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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 2 / Phase 3

Primary location

Arizona Research Center, Phoenix, Arizona, United States

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Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Adult patients, 18 years or older
  • Diagnosed Pachyonychia Congenita (PC), genetically confirmed
  • Moderate to Severe PC
  • Able and willing to comply with all protocol-required activities
  • Willing and able to provide written informed consent

Exclusion criteria

  • Any significant concurrent condition (including involving the inferior to the ankle) that could adversely affect participation.
  • Any intentional changes in the patient's daily activities (associated with standing, walking and keeping balance), not resulting from an improvement in the patient's condition due to treatment.
  • Patient's deemed by the investigator as unwilling or unable to remain compliant with all tests and procedures, including adherence to study drug administration and other protocol-required activities.

Treatment and study plan

PTX-022

Drug

PTX-022 QTORIN

Placebo

Drug

Placebo topical

Primary outcomes

  1. Patient Global Assessment of Activities Scale

    Time frame: 6 months

Secondary outcomes

  1. Clinician Global Impression of Change Scale

    Time frame: 6 months

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

    Time frame: 6 months

  3. Number of steps taken as assessed by activity monitor

    Time frame: 6 months

Sponsors and collaborators

Lead sponsor

Palvella Therapeutics, Inc.

Industry

Registry information

Official study title

A Multicenter, Four-Part, Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in the Treatment of Adults With Moderate to Severe Pachyonychia Congenita

Important dates

Study start
2019
Primary completion
2020
Study completion
2020
First posted
Apr 18, 2019
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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