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

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 Overview

• Status: Completed
• Conditions: Pachyonychia Congenita
• Intervention / Treatment: Drug: PTX-022

• Study Type: Interventional
• Enrollment (Actual): 36
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85053
      • Arizona Research Center
  • California
    • Palo Alto, California, United States, 94304
      • Stanford University
  • Minnesota
    • Fridley, Minnesota, United States, 55432
      • Minnesota Clinical Study Center
  • Utah
    • Murray, Utah, United States, 84107
      • University of Utah

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Open label treatment arm; PTX-022 QTORIN	Drug: PTX-022; Safety and Efficacy of PTX-022 in the Treatment of Adults with Pachyonychia Congenita

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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

Outcome Measure	Time Frame
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
Clinician Global Impression of Severity as assessed by likert scale	18 months

Collaborators and Investigators

• Sponsor: Palvella Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2020
• Primary Completion (Actual): June 30, 2021
• Study Completion (Actual): December 15, 2022

Study Registration Dates

• First Submitted: March 6, 2020
• First Submitted That Met QC Criteria: August 18, 2020
• First Posted (Actual): August 20, 2020

Study Record Updates

• Last Update Posted (Actual): August 29, 2024
• Last Update Submitted That Met QC Criteria: August 28, 2024
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Pathological Conditions, Anatomical; Skin Diseases, Genetic; Nail Diseases; Skin Abnormalities; Abnormalities, Multiple; Ectodermal Dysplasia; Pachyonychia Congenita; Nails, Malformed
• Other Study ID Numbers: PALV-03

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No