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

Pachyonychia Congenita Clinical Trial

— VALO-2  

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04520750
• Other study ID #: PALV-03
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: September 1, 2020
• Est. completion date: January 2022

Study information

• Verified date: December 2021
• Source: Palvella Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 36
• Est. completion date: January 2022
• Est. primary completion date: June 30, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Pachyonychia Congenita

Intervention

Drug:
• PTX-022
Safety and Efficacy of PTX-022 in the Treatment of Adults with Pachyonychia Congenita

Locations

Country	Name	City	State
United States	Minnesota Clinical Study Center	Fridley	Minnesota
United States	University of Utah	Murray	Utah
United States	Stanford University	Palo Alto	California
United States	Arizona Research Center	Phoenix	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
Palvella Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Treatment-Emergent Adverse Events		18 Months
Primary	Changes in vital sign measurements from baseline		18 months
Primary	Changes in weight measurements from baseline		18 months
Primary	Changes in clinical laboratory measurements from baseline		18 months
Secondary	Patient Global Assessment of Activities Scale		18 Months
Secondary	Pain at its worst as assessed by NRS		18 months
Secondary	PROMIS Pain Interference Short 6A as assessed by likert scale		18 months
Secondary	PROMIS Physical Function as assessed by likert scale		18 months
Secondary	Patient Global Impression of Severity as assessed by likert scale		18 months
Secondary	Clinician Global Impression of Severity as assessed by likert scale		18 months