VALO-2: Study Evaluating the Safety and Efficacy of PTX02... | NCT04520750

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

NCT04520750

Sponsor

Palvella Therapeutics, Inc.

Status

Completed

Last Update Posted

Aug 29, 2024Actual

Enrollment

36Actual

Phase

Phase 3

Conditions

Pachyonychia Congenita

Interventions

PTX-022

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Aug 2024

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Sep 1, 2020Actual

Primary Completion Date

Jun 30, 2021Actual

Completion Date

Dec 15, 2022Actual

First Submitted Date

Mar 6, 2020

First Submission Date that Met QC Criteria

Aug 18, 2020

First Posted Date

Aug 20, 2020Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Aug 28, 2024

Last Update Posted Date

Aug 29, 2024Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

36Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Open label treatment arm	Experimental	PTX-022 QTORIN	Drug: PTX-022

Interventions

Name	Type	Description	Arm Group Labels	Other Names
PTX-022	Drug	Safety and Efficacy of PTX-022 in the Treatment of Adults with Pachyonychia Congenita	Open label treatment arm

Outcomes Module

Primary Outcomes

Measure	Description	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 Outcomes

Measure	Description	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

Other Outcomes

Not provided

Eligibility Module

Eligibility 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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Arizona Research Center		Phoenix	Arizona	85053	United States
Stanford University

Submission Tracking

Estimated Results First Submitted Date

Dec 24, 2024

First MCP Posted Date

Not provided

Submission Infos

Release Date	Unrelease Date	Unrelease Date Unknown	Reset Date	MCP Release Number
Dec 24, 2024			Jan 14, 2025	3

Condition Browse Module

MeSH Terms

ID	Term
D053549	Pachyonychia Congenita

Ancestor Terms

ID	Term
D004476	Ectodermal Dysplasia
D000015	Abnormalities, Multiple
D000013	Congenital Abnormalities
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Browse Leaves

Not provided

Browse Branches

Not provided