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

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

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04520750

Acronym

VALO-2

Enrollment

Registered

2020-08-20

Start date

2020-09-01

Completion date

2022-12-15

Last updated

2024-08-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pachyonychia Congenita

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.

Interventions

DRUGPTX-022

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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Intervention model description

Open label

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

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

Design outcomes

Primary

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

Measure	Time frame
Patient Global Impression of Severity as assessed by likert scale	18 months
Patient Global Assessment of Activities Scale	18 Months
Clinician Global Impression of Severity as assessed by likert 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

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026