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VALO-2: PTX-022 in Pachyonychia Congenita
NCT04520750Phase 3
Key facts
- Status
- Completed
- Start
- Sep 2020
- Primary completion
- Jun 2021
- Countries
- 1
- Sponsor
- Palvella Therapeutics
- Modality
- Polymer-based Nanoparticles, Transdermal Patches, Hydrogel Systems
- Drugs
- QTORIN rapamycin
- Conditions
- pachyonychia congenita (PC)
01
Overview
Phase 3, open-label, single-arm trial of QTORIN rapamycin in pachyonychia congenita (PC), sponsored by Palvella Therapeutics. Primary endpoints: Incidence of Treatment-Emergent Adverse Events at 18 months, Changes in vital sign measurements at 18 months and 2 more. Started Sep 2020; status: completed.
02
Design
1 arm- Study design
- Non-randomizedOpen-label
Open label treatment arm
PTX-022 QTORIN
03
Who it enrols
Adult patients who previously completed the VALO study (NCT03920228) or VALO-2 sub-study and received meaningful benefit from PTX-022. Patients must have genotyped keratin mutations.
04
Endpoints
Primary
Incidence of Treatment-Emergent Adverse Events
Changes in vital sign measurements
Changes in weight measurements
Changes in clinical laboratory measurements
Secondary
Patient Global Assessment of Activities Scale
Pain at its worst as assessed by NRS
PROMIS Pain Interference Short 6A
PROMIS Physical Function
Patient Global Impression of Severity
Clinician Global Impression of Severity
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