A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
- Registration Number
- NCT05643872
- Lead Sponsor
- Palvella Therapeutics, Inc.
- Brief Summary
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
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 45
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description PTX-022 PTX-022 -
- Primary Outcome Measures
Name Time Method Treatment emergent adverse events 6 months
- Secondary Outcome Measures
Name Time Method Pharmacokinetic parameters Prior to dose, 12 hours and 24 hours AUC area under the sirolimus blood concentration-time profile
Trial Locations
- Locations (1)
Minnesota Clinical Study Center
🇺🇸New Brighton, Minnesota, United States