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

Status

Completed

Phases

Phase 2Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03920228

Enrollment

Registered

2019-04-18

Start date

2019-04-01

Completion date

2020-10-19

Last updated

2024-08-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pachyonychia Congenita

Keywords

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.

Interventions

DRUGPTX-022

PTX-022 QTORIN

DRUGPlacebo

Placebo topical

Study design

Allocation

RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

DOUBLE (Subject, Investigator)

Masking description

During the open-label period, there is no masking. During the randomized, double-blind, placebo-controlled period the participant and Investigator are masked.

Intervention model description

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

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame
Patient Global Assessment of Activities Scale	6 months

Secondary

Measure	Time frame
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

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026