Recruiting
Phase 3

QTORIN Rapamycin

Sponsor:

Palvella Therapeutics, Inc.

Code:

NCT05643872

Conditions

Pachyonychia Congenita

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

Interventions

PTX-022

Study Details

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".

Conditions

Pachyonychia Congenita

Study ID

NCT05643872

Start date

Nov 15, 2022

Status verified date

Dec, 2022

Completion date

Nov, 2023

Anticipated

Primary completion date

Oct, 2023

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

45 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: PTX-022

Interventions

PTX-022

QTORIN rapamycin 3.9% anhydrous gel

Primary outcome measure

  • Treatment emergent adverse events [ Time Frame: 6 months ]

Central Contacts and Locations

Central contacts

Emily Cook

267-738-6366emily.cook@palvellatx.com

Locations

Minnesota Clinical Study Center

Recruiting

New Brighton, Minnesota, United States, 55112

Contacts

Study Coordinator

763-502-2929

Principal Investigator:

Steven E Kempers, MD, FAAD

More Information

Sponsor

Palvella Therapeutics, Inc.

Last update posted

Dec 9, 2022

Last verified

Dec, 2022

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Palvella Therapeutics, Inc. on 2022-12-09.