Actively Recruiting

Phase 3
Age: 16Years +
All Genders
ID05643872

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

Led by Palvella Therapeutics, Inc. · Updated on 2022-12-09

45

Participants Needed

1

Research Sites

4 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and pharmacokinetics of QTORIN rapamycin 3.9% anhydrous gel, called PTX-022, in adults with Pachyonychia Congenita (PC) who have specific keratin gene mutations and who previously participated in the PALV-05 (VAPAUS) trial. This open-label treatment study is conducted at multiple centers to better understand long-term exposure effects of the gel in this patient group. Participants receive the study drug PTX-022 applied as a gel, with the research focusing on monitoring safety and how the drug moves through and is processed by the body over time. The study does not have a placebo group or masking, and treatment details are based on prior enrollment and dosing established in earlier trials. Participants are monitored for treatment-emergent adverse events over six months, with pharmacokinetic measurements taken before dosing and at 12 and 24 hours after application. The trial assesses drug safety and how the body absorbs and metabolizes the gel during this period, helping researchers understand long-term use implications.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 16Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Completed the PALV-05 (VAPAUS) study
  • Agree to contraceptive use
Not Eligible

You will not qualify if you...

  • 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

AI-Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

Treatment

Duration - 6 months

Participants apply QTORIN rapamycin 3.9% anhydrous gel as part of the study treatment.

Visits prior to dose, and at 12 hours and 24 hours after dosing for pharmacokinetic assessments

Trial Site Locations

Total: 1 location

1

Minnesota Clinical Study Center

New Brighton, Minnesota, United States, 55112

Actively Recruiting

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Research Team

E

Emily Cook

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

Similar Trials

International Pachyonychia Congenita Research Registry

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