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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

73 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-04-01

Study Completion Date

2020-10-19

Brief Summary

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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.

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Detailed Description

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Conditions

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Pachyonychia Congenita

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

This study consists of 2 treatment periods:

* Open-Label
* Randomized, Double-Blind, Placebo controlled

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

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

Study Groups

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Open label period

Group Type EXPERIMENTAL

PTX-022

Intervention Type DRUG

PTX-022 QTORIN

Randomized period - Dosing A

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo topical

Randomized period - Dosing B

Group Type EXPERIMENTAL

PTX-022

Intervention Type DRUG

PTX-022 QTORIN

Placebo

Intervention Type DRUG

Placebo topical

Randomized period - Dosing C

Group Type EXPERIMENTAL

PTX-022

Intervention Type DRUG

PTX-022 QTORIN

Interventions

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PTX-022

PTX-022 QTORIN

Intervention Type DRUG

Placebo

Placebo topical

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No