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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05180708
Other study ID # PALV-05
Secondary ID
Status Recruiting
Phase Phase 3
First received
Last updated
Start date November 29, 2021
Est. completion date June 30, 2023

Study information

Verified date August 2022
Source Palvella Therapeutics, Inc.
Contact Emily Cook
Phone 484-253-1463
Email emily.cook@palvellatx.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This study evaluates the safety and efficacy of QTORIN 3.9% rapamycin anhydrous gel in the treatment of adults with Pachyonychia Congenita. This study includes a screening period, baseline period and 6-month treatment period.


Recruitment information / eligibility

Status Recruiting
Enrollment 80
Est. completion date June 30, 2023
Est. primary completion date June 30, 2023
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Key Inclusion Criteria: - Adult patients, 18 years or older (16 years or older in the UK) - Diagnosed with Pachyonychia Congenita (PC), genetically confirmed Key Exclusion Criteria: - Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications. - Any significant concurrent condition (including conditions involving or inferior to the ankle) that could adversely affect the patient's participation and/or the assessment of the safety and efficacy in the study. - Participation in any other interventional clinical research trial within 30 days prior to the screening visit or during the patient's participation in this study

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
QTORIN 3.9% rapamycin anhydrous gel
QTORIN 3.9% rapamycin anhydrous gel
Vehicle
Matching vehicle (QTORIN anyhydrous gel)

Locations

Country Name City State
United Kingdom Royal London Hospital London Whitechapel
United States DermResearch, Inc. Austin Texas
United States Minnesota Clinical Study Center Fridley Minnesota
United States University of Utah Murray Utah
United States Yale University New Haven Connecticut
United States Park Avenue Dermatology Orange Park Florida
United States Stanford University Palo Alto California
United States Oregon Health and Sciences University Portland Oregon

Sponsors (1)

Lead Sponsor Collaborator
Palvella Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Patient Global Assessment of Activities Difficulty Scale 6 months
Secondary Pain at its worst as assessed by numerical rating scale 6 months
Secondary PROMIS physical function 6 months
Secondary Patient global impression of severity (PGI-S) 6 months
Secondary Patient global impression of change (PGI-C) 6 months
Secondary Clinician global impression of severity (CGI-S) 6 months
Secondary Clinician global impression of change (CGI-C) 6 months
See also
  Status Clinical Trial Phase
Completed NCT02592954 - Effect of Broccoli Sprout Extract on Keratinocyte Differentiation in Normal Skin Phase 1
Active, not recruiting NCT04520750 - VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita Phase 3
Recruiting NCT05643872 - A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita Phase 3
Recruiting NCT05956314 - Assessment of KM-001 - Safety, Tolerability, and Efficacy in Patients With PPPK1 or PC Phase 1
Not yet recruiting NCT01382511 - Simvastatin Treatment of Pachyonychia Congenita N/A
Completed NCT02152007 - Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC) Phase 1
Completed NCT00716014 - Study of TD101, a Small Interfering RNA (siRNA) Designed for Treatment of Pachyonychia Congenita Phase 1
Recruiting NCT02321423 - International Pachyonychia Congenita Research Registry
Active, not recruiting NCT03920228 - Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita Phase 2/Phase 3