A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: PTX-022.
- Who it may be relevant to
- Registry conditions: Pachyonychia Congenita. Basic parameters: from 16 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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
Overview
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".
Interventions
- Drug PTX-022
QTORIN rapamycin 3.9% anhydrous gel
Primary outcome measures
- Treatment emergent adverse events [Time frame: 6 months]
Secondary outcome measures (1)
- Pharmacokinetic parameters [Time frame: Prior to dose, 12 hours and 24 hours]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 1 center
- Minnesota Clinical Study Center — New Brighton
Identifiers
NCT: NCT05643872 · PALV-08