CompletedPhase 3

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

United States36 participantsStarted 2020-09-01

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Incidence of Treatment-Emergent Adverse Events

Timeframe: 18 Months

Changes in vital sign measurements from baseline

Timeframe: 18 months

Changes in weight measurements from baseline

Timeframe: 18 months

Changes in clinical laboratory measurements from baseline

Timeframe: 18 months

Trial details

NCT IDNCT04520750

SponsorPalvella Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-06-30

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