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NCT Number: NCT04520750

VALO-2: 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.

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Key information

Conditions

Pachyonychia Congenita Abnormalities, Multiple Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Ectodermal Dysplasia Genetic Diseases, Inborn Nail Diseases Skin Abnormalities Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Arizona Research Center, Phoenix, Arizona, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

PTX-022

Drug

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

Primary outcomes

  1. Incidence of Treatment-Emergent Adverse Events

    Time frame: 18 Months

  2. Changes in vital sign measurements from baseline

    Time frame: 18 months

  3. Changes in weight measurements from baseline

    Time frame: 18 months

  4. Changes in clinical laboratory measurements from baseline

    Time frame: 18 months

Secondary outcomes

  1. Patient Global Assessment of Activities Scale

    Time frame: 18 Months

  2. Pain at its worst as assessed by NRS

    Time frame: 18 months

  3. PROMIS Pain Interference Short 6A as assessed by likert scale

    Time frame: 18 months

  4. PROMIS Physical Function as assessed by likert scale

    Time frame: 18 months

  5. Patient Global Impression of Severity as assessed by likert scale

    Time frame: 18 months

  6. Clinician Global Impression of Severity as assessed by likert scale

    Time frame: 18 months

Sponsors and collaborators

Lead sponsor

Palvella Therapeutics, Inc.

Industry

Registry information

Official study 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

Acronym: VALO-2

Important dates

Study start
2020
Primary completion
2021
Study completion
2022
First posted
Aug 20, 2020
Registry last updated
Aug 29, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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