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

Pachyonychia Congenita Clinical Trial

Official 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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03920228
• Other study ID #: PALV-02
• Status: Active, not recruiting
• Phase: Phase 2/Phase 3
• Start date: April 1, 2019
• Est. completion date: October 2020

Study information

• Verified date: August 2020
• Source: Palvella Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 73
• Est. completion date: October 2020
• Est. primary completion date: August 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Pachyonychia Congenita

Intervention

Drug:
• PTX-022
PTX-022 QTORIN
• Placebo
Placebo topical

Locations

Country	Name	City	State
United States	Northwestern University	Chicago	Illinois
United States	Minnesota Clinical Study Center	Fridley	Minnesota
United States	International Dermatology Research	Miami	Florida
United States	University of Utah	Murray	Utah
United States	Yale University	New Haven	Connecticut
United States	Stanford University	Palo Alto	California
United States	Paddington Testing Co.	Philadelphia	Pennsylvania
United States	Arizona Research Center	Phoenix	Arizona
United States	Oregon Health and Science University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Palvella Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Patient Global Assessment of Activities Scale		6 months
Secondary	Clinician Global Impression of Change Scale		6 months
Secondary	Pain at its worst as assessed by numerical rating scale		6 months
Secondary	Number of steps taken as assessed by activity monitor		6 months

External Links

•   Palvella Therapeutics, Inc.
•   PC Project
•   Valo Study website