Trial | NCT03920228  Report issue

Title

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

  • Phase

    Phase 2/Phase 3

  • Study Type

    Interventional

  • Status

    Unknown status

  • Intervention/Treatment

    PTX-022 ...

  • Study Participants

    73
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.
Study Started
Apr 01
2019
Primary Completion
Aug 31
2020
Anticipated
Study Completion
Oct 31
2020
Anticipated
Last Update
Aug 19
2020

Drug PTX-022

PTX-022 QTORIN

Drug Placebo

Placebo topical

Open label period Experimental

Randomized period - Dosing A Placebo Comparator

Randomized period - Dosing B Experimental

Randomized period - Dosing C Experimental

Criteria 

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.
No Results Posted