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

Study Overview
=================
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.

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

Conditions
-----------------
Pachyonychia Congenita

Intervention / Treatment
-----------------
* Drug: PTX-022
* Drug: Placebo


Participation Criteria
=================
Eligibility Criteria
-----------------
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.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Sequential Assignment
Interventional Model Description: This study consists of 2 treatment periods: Open-Label Randomized, Double-Blind, Placebo controlled
Masking: Double


Arms and Interventions

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

What is the study measuring?
-----------------
Primary Outcome Measures

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

Secondary Outcome Measures

| Outcome Measure | Measure Description | 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 | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
PC