Clinical trial NCT05180708: A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita
Official title: A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Pachyonychia Congenita
Lead sponsor: Palvella Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2022-01-06
Enrollment: 87
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: Patient Global Assessment of Activities Difficulty Scale
timeFrame: 6 months
Secondary outcomes:
measure: Pain at its worst as assessed by numerical rating scale
timeFrame: 6 months
measure: PROMIS physical function
timeFrame: 6 months
measure: Patient global impression of severity (PGI-S)
timeFrame: 6 months
measure: Patient global impression of change (PGI-C)
timeFrame: 6 months
measure: Clinician global impression of severity (CGI-S)
timeFrame: 6 months
measure: Clinician global impression of change (CGI-C)
timeFrame: 6 months
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Orange Park
country: United States
facility: Park Avenue Dermatology
state: Florida
city: Fridley
country: United States
facility: Minnesota Clinical Study Center
state: Minnesota
city: Portland
country: United States
facility: Oregon Health and Sciences University
state: Oregon
city: Austin
country: United States
facility: DermResearch, Inc.
state: Texas
city: Murray
country: United States
facility: University of Utah
state: Utah
city: London
country: United Kingdom
facility: Royal London Hospital
state: Whitechapel
Study officials:
affiliation: Northwestern University
name: Amy Paller, MD
role: STUDY_CHAIR
affiliation: Stanford University
name: Joyce Teng, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Utah
name: David Hansen, MD
role: PRINCIPAL_INVESTIGATOR
References: