Pachyonychia CongenitaPhase 2/3NCT03920228
Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita
A Phase 2/3 study testing PTX-022 in people with Pachyonychia Congenita, run by Palvella Therapeutics, Inc. at 9 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pachyonychia Congenita
- What is being tested?
- PTX-022
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 73 participants
- Where
- 9 sitesUnited States
- Study dates
- 1 Apr 2019to 19 Oct 2020
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Northwestern University
Chicago, Illinois, 60611
Minnesota Clinical Study Center
Fridley, Minnesota, 55432
International Dermatology Research
Miami, Florida, 33144
University of Utah
Murray, Utah, 84107
Yale University
New Haven, Connecticut, 06519
Stanford University
Palo Alto, California, 94304
Paddington Testing Co.
Philadelphia, Pennsylvania, 19103
Arizona Research Center
Phoenix, Arizona, 85053
Oregon Health and Science University
Portland, Oregon, 97239
About this study
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
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.
- Conditions
- Pachyonychia Congenita
- Being tested
- PTX-022, Placebo
- Keywords
- PC
- Medical subject headings
- Pachyonychia Congenita
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PTX-022Drug
PTX-022 QTORIN
PlaceboDrug
Placebo topical
| Study group | Role | What they receive |
|---|---|---|
| Open label period | Experimental | Not described Receives: Drug: PTX-022 |
| Randomized period - Dosing A | Placebo comparator | Not described Receives: Drug: Placebo |
| Randomized period - Dosing B | Experimental | Not described Receives: Drug: PTX-022; Drug: Placebo |
| Randomized period - Dosing C | Experimental | Not described Receives: Drug: PTX-022 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Patient Global Assessment of Activities Scale
Measured: 6 months
Secondary outcomes (3)
Clinician Global Impression of Change Scale
Measured: 6 months
Pain at its worst as assessed by numerical rating scale
Measured: 6 months
Number of steps taken as assessed by activity monitor
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2019
- Study start1 Apr 2019
- Primary completion19 Sep 2020
- Study completion19 Oct 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pachyonychia CongenitaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- This study consists of 2 treatment periods: * Open-Label * Randomized, Double-Blind, Placebo controlled
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 73 participants (actual)
Sponsor and investigators
- Lead sponsor
- Palvella Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- David Hansen, MD, Principal investigator, University of UtahJoyce Teng, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03920228
- Sponsor's protocol ID
- PALV-02
- Status last verified
- Aug 2024
- First submitted
- 2 Apr 2019
- Last updated
- 29 Aug 2024
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03920228
- Acronym / sponsor ID
- PALV-02
- Conditions
- Pachyonychia Congenita
- Keywords
- PC
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 73 participants (actual)
- Lead sponsor
- Palvella Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Apr 2019 (actual)
- Primary completion
- 19 Sep 2020 (actual)
- Study completion
- 19 Oct 2020 (actual)
- First posted
- 18 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
- Model description
- This study consists of 2 treatment periods: * Open-Label * Randomized, Double-Blind, Placebo controlled
| Arm | Type | Receives |
|---|---|---|
| Open label period | Experimental | Drug: PTX-022 |
| Randomized period - Dosing A | Placebo comparator | Drug: Placebo |
| Randomized period - Dosing B | Experimental | Drug: PTX-022, Drug: Placebo |
| Randomized period - Dosing C | Experimental | Drug: PTX-022 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PTX-022PTX-022 QTORIN | Drug | Open label period, Randomized period - Dosing B, Randomized period - Dosing C |
| PlaceboPlacebo topical | Drug | Randomized period - Dosing A, Randomized period - Dosing B |
Primary outcome
Patient Global Assessment of Activities Scale
Time frame: 6 months
3 secondary outcomes
Clinician Global Impression of Change Scale
Time frame: 6 months
Pain at its worst as assessed by numerical rating scale
Time frame: 6 months
Number of steps taken as assessed by activity monitor
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Aug 2024.
- PC ProjectLink provided by the sponsor.
- Palvella Therapeutics, Inc.Link provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David Hansen, MD principal investigator
University of Utah
Joyce Teng, MD principal investigator
Stanford University
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing PTX-022 in people with Pachyonychia Congenita, run by Palvella Therapeutics, Inc. at 9 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Patient Global Assessment of Activities Scale (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Aug 2024.