VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita
VALO-2
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
1 other identifier
interventional
36
1 country
4
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Sep 2020
4 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 6, 2020
CompletedFirst Posted
Study publicly available on registry
August 20, 2020
CompletedStudy Start
First participant enrolled
September 1, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
December 15, 2022
CompletedAugust 29, 2024
August 1, 2024
10 months
March 6, 2020
August 28, 2024
Conditions
Outcome Measures
Primary Outcomes (4)
Incidence of Treatment-Emergent Adverse Events
18 Months
Changes in vital sign measurements from baseline
18 months
Changes in weight measurements from baseline
18 months
Changes in clinical laboratory measurements from baseline
18 months
Secondary Outcomes (6)
Patient Global Assessment of Activities Scale
18 Months
Pain at its worst as assessed by NRS
18 months
PROMIS Pain Interference Short 6A as assessed by likert scale
18 months
PROMIS Physical Function as assessed by likert scale
18 months
Patient Global Impression of Severity as assessed by likert scale
18 months
- +1 more secondary outcomes
Study Arms (1)
Open label treatment arm
EXPERIMENTALPTX-022 QTORIN
Interventions
Safety and Efficacy of PTX-022 in the Treatment of Adults with Pachyonychia Congenita
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications or to PTX-022
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (4)
Arizona Research Center
Phoenix, Arizona, 85053, United States
Stanford University
Palo Alto, California, 94304, United States
Minnesota Clinical Study Center
Fridley, Minnesota, 55432, United States
University of Utah
Murray, Utah, 84107, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 6, 2020
First Posted
August 20, 2020
Study Start
September 1, 2020
Primary Completion
June 30, 2021
Study Completion
December 15, 2022
Last Updated
August 29, 2024
Record last verified: 2024-08