A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita

NCT ID: NCT05643872

Last Updated: 2022-12-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

45 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-11-15

Study Completion Date

2023-11-30

Brief Summary

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PALV-08 is a multicenter, open-label treatment (OLT) study enrolling adults with Pachyonychia Congenita (PC) with genotyped keratin mutations KRT6A, KRT6B, KRT6C or KRT16 who were previously enrolled in the PALV-05 (VAPAUS) trial.

The purpose of this OLT study is to investigate the safety of long term exposure and pharmacokinetics (PK) of QTORIN rapamycin 3.9% anhydrous gel or "PTX-022".

Detailed Description

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Conditions

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Pachyonychia Congenita

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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PTX-022

Group Type EXPERIMENTAL

PTX-022

Intervention Type DRUG

QTORIN rapamycin 3.9% anhydrous gel

Interventions

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PTX-022

QTORIN rapamycin 3.9% anhydrous gel

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Completed the PALV-05 (VAPAUS) study
* Agree to contraceptive use

Exclusion Criteria

* Females who are pregnant or breastfeeding
* Concomitant use of sirolimus or any sirolimus-containing medications (systemic or topical)
* Any significant concurrent condition that could adversely affect participation and/or the assessment of the safety and efficacy in the study
* Prior or planned treatment, including surgery or other medically necessary intervention deemed by the investigator that could adversely affect participation and/or the assessment of the safety and efficacy in the study
Minimum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Palvella Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Minnesota Clinical Study Center

New Brighton, Minnesota, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Emily Cook

Role: CONTACT

Phone: 267-738-6366

Email: [email protected]

Facility Contacts

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Study Coordinator

Role: primary

Other Identifiers

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PALV-08

Identifier Type: -

Identifier Source: org_study_id