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

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 Overview

• Status: Completed
• Conditions: Pachyonychia Congenita
• Intervention / Treatment: Drug: PTX-022; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 73
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85053
      • Arizona Research Center
  • California
    • Palo Alto, California, United States, 94304
      • Stanford University
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale University
  • Florida
    • Miami, Florida, United States, 33144
      • International Dermatology Research
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern University
  • Minnesota
    • Fridley, Minnesota, United States, 55432
      • Minnesota Clinical Study Center
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health and Science University
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19103
      • Paddington Testing Co.
  • Utah
    • Murray, Utah, United States, 84107
      • University of Utah

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Double

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Open label period	Drug: PTX-022; PTX-022 QTORIN
Placebo Comparator: Randomized period - Dosing A	Drug: Placebo; Placebo topical
Experimental: Randomized period - Dosing B	Drug: PTX-022; PTX-022 QTORIN; Drug: Placebo; Placebo topical
Experimental: Randomized period - Dosing C	Drug: PTX-022; PTX-022 QTORIN

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Patient Global Assessment of Activities Scale	6 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Pain at its worst as assessed by numerical rating scale	6 months
Clinician Global Impression of Change Scale	6 months
Number of steps taken as assessed by activity monitor	6 months

Collaborators and Investigators

• Sponsor: Palvella Therapeutics, Inc.
• Investigators: Principal Investigator: Joyce Teng, MD, Stanford University; Principal Investigator: David Hansen, MD, University of Utah

Publications and helpful links

Helpful Links

• PC Project
• Palvella Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): April 1, 2019
• Primary Completion (Actual): September 19, 2020
• Study Completion (Actual): October 19, 2020

Study Registration Dates

• First Submitted: April 2, 2019
• First Submitted That Met QC Criteria: April 15, 2019
• First Posted (Actual): April 18, 2019

Study Record Updates

• Last Update Posted (Actual): August 29, 2024
• Last Update Submitted That Met QC Criteria: August 28, 2024
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Keywords: PC
• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Pathological Conditions, Anatomical; Skin Diseases, Genetic; Nail Diseases; Skin Abnormalities; Abnormalities, Multiple; Ectodermal Dysplasia; Pachyonychia Congenita; Nails, Malformed
• Other Study ID Numbers: PALV-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No