Back to resultsVALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita (VALO-2)
Primary Purpose
Pachyonychia Congenita
Status
Active
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
PTX-022
Sponsored by
About this trial
This is an interventional treatment trial for Pachyonychia Congenita
Eligibility Criteria
Key Inclusion Criteria:
- Adult patients 18 years or older,
- Previously completed the VALO protocol, or VALO-2 K6C/17 sub-study, and received meaningful benefit from investigational PTX-022 as determined by the clinician.
Key Exclusion Criteria:
- Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications or to PTX-022
Sites / Locations
- Arizona Research Center
- Stanford University
- Minnesota Clinical Study Center
- University of Utah
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Open label treatment arm
Arm Description
PTX-022 QTORIN
Outcomes
Primary Outcome Measures
Incidence of Treatment-Emergent Adverse Events
Changes in vital sign measurements from baseline
Changes in weight measurements from baseline
Changes in clinical laboratory measurements from baseline
Secondary Outcome Measures
Patient Global Assessment of Activities Scale
Pain at its worst as assessed by NRS
PROMIS Pain Interference Short 6A as assessed by likert scale
PROMIS Physical Function as assessed by likert scale
Patient Global Impression of Severity as assessed by likert scale
Clinician Global Impression of Severity as assessed by likert scale
Full Information
NCT ID
NCT04520750
First Posted
March 6, 2020
Last Updated
December 2, 2021
Sponsor
Palvella Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT04520750
Brief Title
VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita
Acronym
VALO-2
Official Title
VALO-2: A Multicenter, Phase 3b, Open-Label Treatment Extension Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita
Study Type
Interventional
2. Study Status
Record Verification Date
December 2021
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 1, 2020 (Actual)
Primary Completion Date
June 30, 2021 (Actual)
Study Completion Date
January 2022 (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
Data Monitoring Committee
No
5. Study Description
Brief Summary
VALO-2 is a multicenter, open-label extension (OLE) study enrolling patients with genotyped keratin mutations KRT6A, KRT6B and KRT16 who were previously treated with investigational PTX-022 during the VALO study. The purpose of the OLE study is to investigate long term exposure to investigational PTX-022 and evaluate safety and efficacy data. A sub-study is included to evaluate safety and efficacy of patients with genotyped keratin mutations of KRT6C and KRT17 not previously treated with investigational PTX-022.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pachyonychia Congenita
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
Open label
Masking
None (Open Label)
Allocation
N/A
Enrollment
36 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Open label treatment arm
Arm Type
Experimental
Arm Description
PTX-022 QTORIN
Intervention Type
Drug
Intervention Name(s)
PTX-022
Intervention Description
Safety and Efficacy of PTX-022 in the Treatment of Adults with Pachyonychia Congenita
Primary Outcome Measure Information:
Title
Incidence of Treatment-Emergent Adverse Events
Time Frame
18 Months
Title
Changes in vital sign measurements from baseline
Time Frame
18 months
Title
Changes in weight measurements from baseline
Time Frame
18 months
Title
Changes in clinical laboratory measurements from baseline
Time Frame
18 months
Secondary Outcome Measure Information:
Title
Patient Global Assessment of Activities Scale
Time Frame
18 Months
Title
Pain at its worst as assessed by NRS
Time Frame
18 months
Title
PROMIS Pain Interference Short 6A as assessed by likert scale
Time Frame
18 months
Title
PROMIS Physical Function as assessed by likert scale
Time Frame
18 months
Title
Patient Global Impression of Severity as assessed by likert scale
Time Frame
18 months
Title
Clinician Global Impression of Severity as assessed by likert scale
Time Frame
18 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Adult patients 18 years or older,
Previously completed the VALO protocol, or VALO-2 K6C/17 sub-study, and received meaningful benefit from investigational PTX-022 as determined by the clinician.
Key Exclusion Criteria:
Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications or to PTX-022
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
Minnesota Clinical Study Center
City
Fridley
State/Province
Minnesota
ZIP/Postal Code
55432
Country
United States
Facility Name
University of Utah
City
Murray
State/Province
Utah
ZIP/Postal Code
84107
Country
United States
12. IPD Sharing Statement
Learn more about this trial
VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita
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