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

Sponsor

Palvella Therapeutics, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05643872

Collaborator

(none)

Enrollment

Location

Arm

11.5

Anticipated Duration (Months)

3.9

Patients Per Site Per Month

Study Details

Study Description

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".

Condition or Disease	Intervention/Treatment	Phase
Pachyonychia Congenita	Drug: PTX-022	Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

45 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Nov 15, 2022

Anticipated Primary Completion Date :

Oct 1, 2023

Anticipated Study Completion Date :

Nov 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: PTX-022	Drug: PTX-022 QTORIN rapamycin 3.9% anhydrous gel

Arm

Intervention/Treatment

Experimental: PTX-022

Drug: PTX-022

QTORIN rapamycin 3.9% anhydrous gel

Outcome Measures

Primary Outcome Measures

Treatment emergent adverse events [6 months]

Secondary Outcome Measures

Pharmacokinetic parameters [Prior to dose, 12 hours and 24 hours]
AUC area under the sirolimus blood concentration-time profile

Eligibility Criteria

Criteria

Ages Eligible for Study:

16 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Minnesota Clinical Study Center	New Brighton	Minnesota	United States	55112

Sponsors and Collaborators

Palvella Therapeutics, Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Palvella Therapeutics, Inc.

ClinicalTrials.gov Identifier:

NCT05643872

Other Study ID Numbers:

PALV-08

First Posted:

Dec 9, 2022

Last Update Posted:

Dec 9, 2022

Last Verified:

Dec 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Pachyonychia Congenita

Nails, Malformed

Study Results

No Results Posted as of Dec 9, 2022