NCT03920228CompletedPhase 2
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
Palvella Therapeutics, Inc.9 sites in 1 country73 target enrollmentStarted: April 1, 2019Last updated:
Conditions
Interventions
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Palvella Therapeutics, Inc.
- Enrollment
- 73
- Locations
- 9
- Primary Endpoint
- Patient Global Assessment of Activities Scale
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Investigator)
Masking Description
During the open-label period, there is no masking. During the randomized, double-blind, placebo-controlled period the participant and Investigator are masked.
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
Open label period
Experimental
Intervention: PTX-022 (Drug)
Randomized period - Dosing A
Placebo Comparator
Intervention: Placebo (Drug)
Randomized period - Dosing B
Experimental
Intervention: PTX-022 (Drug)
Randomized period - Dosing B
Experimental
Intervention: Placebo (Drug)
Randomized period - Dosing C
Experimental
Intervention: PTX-022 (Drug)
Outcomes
Primary Outcomes
Patient Global Assessment of Activities Scale
Time Frame: 6 months
Secondary Outcomes
- Clinician Global Impression of Change Scale(6 months)
- Pain at its worst as assessed by numerical rating scale(6 months)
- Number of steps taken as assessed by activity monitor(6 months)
Investigators
Study Sites (9)
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