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

NCT03920228 | Completed Phase 2 FDA Drug

• 1 other identifier: PALV-02
• Study Type: interventional
• Target: 73
• Locations: 1 country
• Sites: 9

Brief 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.

Conditions

Pachyonychia Congenita

Keywords

PC

Outcome Measures

Primary Outcomes (1)

• Patient Global Assessment of Activities Scale

  6 months

Secondary Outcomes (3)

• Clinician Global Impression of Change Scale

  6 months

• Pain at its worst as assessed by numerical rating scale

  6 months

• Number of steps taken as assessed by activity monitor

  6 months

Study Arms (4)

Open label period

EXPERIMENTAL

Drug: PTX-022

Randomized period - Dosing A

PLACEBO COMPARATOR

Drug: Placebo

Randomized period - Dosing B

EXPERIMENTAL

Drug: PTX-022; Drug: Placebo

Randomized period - Dosing C

EXPERIMENTAL

Drug: PTX-022

Interventions

PTX-022 DRUG

PTX-022 QTORIN

Open label period; Randomized period - Dosing B; Randomized period - Dosing C

Placebo DRUG

Placebo topical

Randomized period - Dosing A; Randomized period - Dosing B

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Palvella Therapeutics, Inc.: lead

Study Sites (9)

Arizona Research Center

Phoenix, Arizona, 85053, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

Yale University

New Haven, Connecticut, 06519, United States

Location

International Dermatology Research

Miami, Florida, 33144, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Minnesota Clinical Study Center

Fridley, Minnesota, 55432, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Paddington Testing Co.

Philadelphia, Pennsylvania, 19103, United States

Location

University of Utah

Murray, Utah, 84107, United States

Location

Related Links

• PC Project
• Palvella Therapeutics, Inc.

MeSH Terms

Conditions

Pachyonychia Congenita

Condition Hierarchy (Ancestors)

Ectodermal Dysplasia; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Nail Diseases; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• David Hansen, MD

  University of Utah

  PRINCIPAL INVESTIGATOR

• Joyce Teng, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: During the open-label period, there is no masking. During the randomized, double-blind, placebo-controlled period the participant and Investigator are masked.
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This study consists of 2 treatment periods: * Open-Label * Randomized, Double-Blind, Placebo controlled

Study Record Dates

• First Submitted: April 2, 2019
• First Posted: April 18, 2019
• Study Start: April 1, 2019
• Primary Completion: September 19, 2020
• Study Completion: October 19, 2020
• Last Updated: August 29, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (9)