CompletedPhase 2/3

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

United States73 participantsStarted 2019-04-01

Plain-language summary

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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

Patient Global Assessment of Activities Scale

Timeframe: 6 months

Trial details

NCT IDNCT03920228

SponsorPalvella Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-09-19

View on ClinicalTrials.gov More Pachyonychia Congenita trials