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

• Status: Active, not recruiting
• Trial end date: 2020-10-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Palvella Therapeutics, Inc.

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.