VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita

Phase 3

Completed

• Conditions: Pachyonychia Congenita
• Interventions: Drug: PTX-022

• Registration Number: NCT04520750
• Lead Sponsor: Palvella Therapeutics, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-03-06
• Study First Submitted QC: 2020-08-18
• Study First Posted: 2020-08-20
• Study Start: 2020-09-01
• Primary Completion: 2021-06-30
• Study Completion: 2022-12-15
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-28
• Last Update Posted: 2024-08-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 36

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open label treatment arm	PTX-022	PTX-022 QTORIN

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
Pain at its worst as assessed by NRS	18 months
Patient Global Assessment of Activities Scale	18 Months
PROMIS Physical Function as assessed by likert scale	18 months
Clinician Global Impression of Severity as assessed by likert 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

Trial Locations

Locations (4)

Arizona Research Center; 🇺🇸 Phoenix, Arizona, United States

Minnesota Clinical Study Center; 🇺🇸 Fridley, Minnesota, United States

Stanford University; 🇺🇸 Palo Alto, California, United States

University of Utah; 🇺🇸 Murray, Utah, United States