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
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Palvella Therapeutics, Inc.
- Enrollment
- 36
- Locations
- 4
- Primary Endpoint
- Incidence of Treatment-Emergent Adverse Events
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Exclusion Criteria
- •Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications or to PTX-022
Arms & Interventions
Open label treatment arm
PTX-022 QTORIN
Intervention: PTX-022 (Drug)
Outcomes
Primary Outcomes
Incidence of Treatment-Emergent Adverse Events
Time Frame: 18 Months
Changes in vital sign measurements from baseline
Time Frame: 18 months
Changes in weight measurements from baseline
Time Frame: 18 months
Changes in clinical laboratory measurements from baseline
Time Frame: 18 months
Secondary Outcomes
- Pain at its worst as assessed by NRS(18 months)
- PROMIS Physical Function as assessed by likert scale(18 months)
- Clinician Global Impression of Severity as assessed by likert scale(18 months)
- Patient Global Assessment of Activities Scale(18 Months)
- PROMIS Pain Interference Short 6A as assessed by likert scale(18 months)
- Patient Global Impression of Severity as assessed by likert scale(18 months)