Clinical trial

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

Name

PALV-08

Description

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".

Trial arms

Trial start

2022-11-15

Estimated PCD

2023-10-01

Trial end

2023-11-01

Status

Recruiting

Phase

Early phase I

Treatment

PTX-022

QTORIN rapamycin 3.9% anhydrous gel

Arms:

PTX-022

Size

Primary endpoint

Treatment emergent adverse events

6 months

Eligibility criteria

Key Inclusion Criteria: * Completed the PALV-05 (VAPAUS) study * Agree to contraceptive use Key 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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 45, 'type': 'ESTIMATED'}}

Updated at

2022-12-09

1 organization

1 product

1 indication

Organization

Palvella Therapeutics

Product

PTX-022

Indication

Pachyonychia Congenita