Evaluating a gel treatment for adults with Pachyonychia Congenita
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
Run by Palvella Therapeutics, Inc. · NCT05643872 · Data updated 2026-11-12
This study is testing a new gel treatment for adults with Pachyonychia Congenita to see how safe it is and how it works in the body over time.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Ages | 16 Years and up |
| Sex | All |
| Enrollment | 45 (estimated) |
| Sponsor | Palvella Therapeutics, Inc. Industry-sponsored |
| Locations | 1 site (New Brighton, Minnesota) |
| Status | Recruiting |
| Started | November 15, 2022 |
| Main results expected | October 2023 |
| Study ends | November 2023 |
| Registry record updated | December 9, 2022 |
| Trial ID | NCT05643872 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are adults with Pachyonychia Congenita who have completed the PALV-05 study and have specific keratin mutations.
✕Probably not a fit
Patients who are pregnant, breastfeeding, or using sirolimus-containing medications will not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This study investigates the safety and pharmacokinetics of QTORIN rapamycin 3.9% anhydrous gel in adults diagnosed with Pachyonychia Congenita who have specific keratin mutations. It is a multicenter, open-label treatment study that enrolls participants who have previously completed the PALV-05 trial. The focus is on understanding the long-term effects of the gel and how it is processed in the body.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for managing symptoms of Pachyonychia Congenita.
How similar studies have gone: While this approach is novel for Pachyonychia Congenita, similar studies evaluating topical treatments for genetic skin conditions have shown promise.
Who can join: the full rules
Show full inclusion / exclusion 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
Where it's running
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Minnesota Clinical Study Center
New Brighton, Minnesota, United States Recruiting
Study contacts
- Study coordinator: Emily Cook
- Email: emily.cook@palvellatx.com
- Phone: 267-738-6366
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Registry for patients with Pachyonychia Congenita
This study is collecting information from people with Pachyonychia Congenita to better understand the condition and help with future research.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.