ClinicalTrials.gg
Sign In
CompletedNCT03920228Updated Aug 29, 2024

Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita

A Phase 2/3 interventional study of PTX-022 and Placebo in Pachyonychia Congenita, sponsored by Palvella Therapeutics, Inc.. Completed at 9 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-08-29.

Sponsored by Palvella Therapeutics, Inc. · Phase 2/3, Interventional, and Treatment

From the registry’s dates

  • Primary completion was Sep 2020, 6 years 1 month ago, and no results have been posted to the registry.
Phase
Phase 2/3
Study type
Interventional
Enrollment
73
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study 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.

02

Conditions studied

  • Pachyonychia Congenita

Keywords

  • PC
03

In context

Lead sponsor

Palvella Therapeutics, Inc. is the lead sponsor of 9 studies on the registry; 2 are open to participants now.

Of its 5 completed or terminated interventional studies of FDA-regulated products, 1 (20%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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

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.
05

Study design

Phase
Phase 2 / Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Sequential assignment
Masking
Double (Participant, Investigator)
Enrollment
73 participants (actual)

Study arms

  • Experimental
    Open label period

    Drug: PTX-022

  • Placebo comparator
    Randomized period - Dosing A

    Drug: Placebo

  • Experimental
    Randomized period - Dosing B

    Drug: PTX-022 · Drug: Placebo

  • Experimental
    Randomized period - Dosing C

    Drug: PTX-022

Interventions

  • DrugPTX-022

    PTX-022 QTORIN

  • DrugPlacebo

    Placebo topical

06

What researchers measure

Primary outcomes

  1. Patient Global Assessment of Activities Scale

    Time frame: 6 months

Secondary outcomes

  1. Clinician Global Impression of Change Scale

    Time frame: 6 months

  2. Pain at its worst as assessed by numerical rating scale

    Time frame: 6 months

  3. Number of steps taken as assessed by activity monitor

    Time frame: 6 months

07

Study locations

9 sites
  • Arizona Research Center
    Phoenix, Arizona 85053, United States
  • Stanford University
    Palo Alto, California 94304, United States
  • Yale University
    New Haven, Connecticut 06519, United States
  • International Dermatology Research
    Miami, Florida 33144, United States
  • Northwestern University
    Chicago, Illinois 60611, United States
  • Minnesota Clinical Study Center
    Fridley, Minnesota 55432, United States
  • Oregon Health and Science University
    Portland, Oregon 97239, United States
  • Paddington Testing Co.
    Philadelphia, Pennsylvania 19103, United States
  • University of Utah
    Murray, Utah 84107, United States
08

References and documents

Individual participant data

Plan to share: No

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Apr 2019 to primary completion in Sep 2020, 17 months.

How these figures are worked out, and which studies are left out of them.

Registered
Apr 2, 2019
First posted
Apr 18, 2019
StartActual
Apr 1, 2019
Primary completionActual
Sep 19, 2020
CompletionActual
Oct 19, 2020
Last update
Aug 29, 2024
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Aug 29, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT03920228
Lead sponsor
Palvella Therapeutics, Inc.
Responsible party
Sponsor
First posted
Apr 18, 2019
Start date
Apr 1, 2019
Primary completion
Sep 19, 2020
Completion
Oct 19, 2020
Last update
Aug 29, 2024

Study contacts

David Hansen, MD
principal investigator · University of Utah
Joyce Teng, MD
principal investigator · Stanford University

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Aug 2024. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Studies of the drug tested here: Sirolimus Topical Gel PTX-022

Other studies in this condition

Pachyonychia Congenita

Other studies from this sponsor

Palvella Therapeutics, Inc.

Start the discussion