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

Primary Purpose

Pachyonychia Congenita

Status
Unknown status
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
PTX-022
Placebo
Sponsored by
Palvella Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pachyonychia Congenita focused on measuring PC

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

  • Arizona Research Center
  • Stanford University
  • Yale University
  • International Dermatology Research
  • Northwestern University
  • Minnesota Clinical Study Center
  • Oregon Health and Science University
  • Paddington Testing Co.
  • University of Utah

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Placebo Comparator

Experimental

Experimental

Arm Label

Open label period

Randomized period - Dosing A

Randomized period - Dosing B

Randomized period - Dosing C

Arm Description

Outcomes

Primary Outcome Measures

Patient Global Assessment of Activities Scale

Secondary Outcome Measures

Clinician Global Impression of Change Scale
Pain at its worst as assessed by numerical rating scale
Number of steps taken as assessed by activity monitor

Full Information

First Posted
April 2, 2019
Last Updated
August 18, 2020
Sponsor
Palvella Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03920228
Brief Title
Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2020
Overall Recruitment Status
Unknown status
Study Start Date
April 1, 2019 (Actual)
Primary Completion Date
August 2020 (Anticipated)
Study Completion Date
October 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Palvella Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pachyonychia Congenita
Keywords
PC

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Sequential Assignment
Model Description
This study consists of 2 treatment periods: Open-Label Randomized, Double-Blind, Placebo controlled
Masking
ParticipantInvestigator
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.
Allocation
Randomized
Enrollment
73 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Open label period
Arm Type
Experimental
Arm Title
Randomized period - Dosing A
Arm Type
Placebo Comparator
Arm Title
Randomized period - Dosing B
Arm Type
Experimental
Arm Title
Randomized period - Dosing C
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
PTX-022
Intervention Description
PTX-022 QTORIN
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo topical
Primary Outcome Measure Information:
Title
Patient Global Assessment of Activities Scale
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Clinician Global Impression of Change Scale
Time Frame
6 months
Title
Pain at its worst as assessed by numerical rating scale
Time Frame
6 months
Title
Number of steps taken as assessed by activity monitor
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
David Hansen, MD
Organizational Affiliation
University of Utah
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Joyce Teng, MD
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Arizona Research Center
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85053
Country
United States
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
Yale University
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06519
Country
United States
Facility Name
International Dermatology Research
City
Miami
State/Province
Florida
ZIP/Postal Code
33144
Country
United States
Facility Name
Northwestern University
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Minnesota Clinical Study Center
City
Fridley
State/Province
Minnesota
ZIP/Postal Code
55432
Country
United States
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Paddington Testing Co.
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19103
Country
United States
Facility Name
University of Utah
City
Murray
State/Province
Utah
ZIP/Postal Code
84107
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Links:
URL
https://www.pachyonychia.org/
Description
PC Project
URL
http://www.palvellatx.com
Description
Palvella Therapeutics, Inc.

Learn more about this trial

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

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