Back to resultsA Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
Primary Purpose
Pachyonychia Congenita
Status
Recruiting
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
PTX-022
Sponsored by
About this trial
This is an interventional treatment trial for Pachyonychia Congenita
Eligibility 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
Sites / Locations
- Minnesota Clinical Study CenterRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
PTX-022
Arm Description
Outcomes
Primary Outcome Measures
Treatment emergent adverse events
Secondary Outcome Measures
Pharmacokinetic parameters
AUC area under the sirolimus blood concentration-time profile
Full Information
NCT ID
NCT05643872
First Posted
November 8, 2022
Last Updated
December 1, 2022
Sponsor
Palvella Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT05643872
Brief Title
A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of 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
Study Type
Interventional
2. Study Status
Record Verification Date
December 2022
Overall Recruitment Status
Recruiting
Study Start Date
November 15, 2022 (Actual)
Primary Completion Date
October 2023 (Anticipated)
Study Completion Date
November 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Palvella Therapeutics, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. 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".
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pachyonychia Congenita
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
45 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
PTX-022
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
PTX-022
Intervention Description
QTORIN rapamycin 3.9% anhydrous gel
Primary Outcome Measure Information:
Title
Treatment emergent adverse events
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Pharmacokinetic parameters
Description
AUC area under the sirolimus blood concentration-time profile
Time Frame
Prior to dose, 12 hours and 24 hours
10. Eligibility
Sex
All
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Emily Cook
Phone
267-738-6366
Email
emily.cook@palvellatx.com
Facility Information:
Facility Name
Minnesota Clinical Study Center
City
New Brighton
State/Province
Minnesota
ZIP/Postal Code
55112
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Study Coordinator
Phone
763-502-2929
First Name & Middle Initial & Last Name & Degree
Steven E Kempers, MD, FAAD
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
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