Clinical trial NCT03920228: Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita
Official title: A Multicenter, Four-Part, Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in the Treatment of Adults With Moderate to Severe Pachyonychia Congenita
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Pachyonychia Congenita
Keywords: PC
Lead sponsor: Palvella Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2019-04-18
Enrollment: 73
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: This study consists of 2 treatment periods:

* Open-Label
* Randomized, Double-Blind, Placebo controlled
Primary purpose: TREATMENT

Brief summary: This study evaluates the safety and efficacy of PTX-022, topical rapamycin, in the treatment of adults with moderate to severe Pachyonychia Congenita.

This study includes four-parts, and if a participant completes all parts, the participant will have received at least 3-months of PTX-022 treatment.


Eligibility criteria: Inclusion Criteria:

* Adult patients, 18 years or older
* Diagnosed Pachyonychia Congenita (PC), genetically confirmed
* Moderate to Severe PC
* Able and willing to comply with all protocol-required activities
* Willing and able to provide written informed consent

Exclusion Criteria:

* Any significant concurrent condition (including involving the inferior to the ankle) that could adversely affect participation.
* Any intentional changes in the patient's daily activities (associated with standing, walking and keeping balance), not resulting from an improvement in the patient's condition due to treatment.
* Patient's deemed by the investigator as unwilling or unable to remain compliant with all tests and procedures, including adherence to study drug administration and other protocol-required activities.

Primary outcomes:
measure: Patient Global Assessment of Activities Scale
timeFrame: 6 months
Secondary outcomes:
measure: Clinician Global Impression of Change Scale
timeFrame: 6 months
measure: Pain at its worst as assessed by numerical rating scale
timeFrame: 6 months
measure: Number of steps taken as assessed by activity monitor
timeFrame: 6 months
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Arizona Research Center
state: Arizona
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Miami
country: United States
facility: International Dermatology Research
state: Florida
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Fridley
country: United States
facility: Minnesota Clinical Study Center
state: Minnesota
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Philadelphia
country: United States
facility: Paddington Testing Co.
state: Pennsylvania
city: Murray
country: United States
facility: University of Utah
state: Utah
Study officials:
affiliation: University of Utah
name: David Hansen, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Stanford University
name: Joyce Teng, MD
role: PRINCIPAL_INVESTIGATOR
References: