Pachyonychia CongenitaPhase 3VAPAUSNCT05180708
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
A Phase 3 study testing QTORIN 3.9% rapamycin anhydrous gel in people with Pachyonychia Congenita, run by Palvella Therapeutics, Inc. at 8 sites in 2 countries.
Trial 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. 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?
- QTORIN 3.9% rapamycin anhydrous gel
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 87 participants
- Where
- 8 sitesin 2 countries
- Study dates
- 29 Nov 2021to 30 Jun 2023
- Phase
- Phase 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 (16 years or older in the UK)
- Diagnosed with Pachyonychia Congenita (PC), genetically confirmed
Who cannot join
- 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
Trial locations and contacts
8 sites in 2 countries.
8 sites in 2 countries
United States7 sites
DermResearch, Inc.
Austin, Texas, 78759
Minnesota Clinical Study Center
Fridley, Minnesota, 55432
University of Utah
Murray, Utah, 84107
Yale University
New Haven, Connecticut, 06519
Park Avenue Dermatology
Orange Park, Florida, 32073
Stanford University
Palo Alto, California, 94304
Oregon Health and Sciences University
Portland, Oregon, 97239
About this study
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
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.
- Conditions
- Pachyonychia Congenita
- Being tested
- QTORIN 3.9% rapamycin anhydrous gel, Vehicle
- Medical subject headings
- Pachyonychia Congenita
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QTORIN 3.9% rapamycin anhydrous gelDrug
QTORIN 3.9% rapamycin anhydrous gel
VehicleDrug
Matching vehicle (QTORIN anyhydrous gel)
| Study group | Role | What they receive |
|---|---|---|
| Active | Active comparator | Not described Receives: Drug: QTORIN 3.9% rapamycin anhydrous gel |
| Vehicle | Placebo comparator | Not described Receives: Drug: Vehicle |
What is being measured
The primary outcome is the main question the study is designed to answer.
Patient Global Assessment of Activities Difficulty Scale
Measured: 6 months
Secondary outcomes (6)
Pain at its worst as assessed by numerical rating scale
Measured: 6 months
PROMIS physical function
Measured: 6 months
Patient global impression of severity (PGI-S)
Measured: 6 months
Patient global impression of change (PGI-C)
Measured: 6 months
Clinician global impression of severity (CGI-S)
Measured: 6 months
Clinician global impression of change (CGI-C)
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 posted6 Jan 2022
- Study start29 Nov 2021
- Primary completion30 Jun 2023
- Study completion30 Jun 2023
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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 87 participants (actual)
Sponsor and investigators
- Lead sponsor
- Palvella Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Amy Paller, MD, Study chair, Northwestern UniversityJoyce Teng, MD, Principal investigator, Stanford UniversityDavid Hansen, MD, Principal investigator, University of Utah
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05180708
- Sponsor's protocol ID
- PALV-05
- Acronym
- VAPAUS
- Status last verified
- Aug 2024
- First submitted
- 20 Dec 2021
- Last updated
- 29 Aug 2024
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- 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, 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
- ClinicalTrials.gov ID
- NCT05180708
- Acronym / sponsor ID
- VAPAUS / PALV-05
- Conditions
- Pachyonychia Congenita
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 87 participants (actual)
- Lead sponsor
- Palvella Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 29 Nov 2021 (actual)
- Primary completion
- 30 Jun 2023 (actual)
- Study completion
- 30 Jun 2023 (actual)
- First posted
- 6 Jan 2022 (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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Active | Active comparator | Drug: QTORIN 3.9% rapamycin anhydrous gel |
| Vehicle | Placebo comparator | Drug: Vehicle |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Adult patients, 18 years or older (16 years or older in the UK)
- Diagnosed with Pachyonychia Congenita (PC), genetically confirmed
Key exclusion criteria (3 listed)
- 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
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 |
|---|---|---|
| QTORIN 3.9% rapamycin anhydrous gelQTORIN 3.9% rapamycin anhydrous gel | Drug | Active |
| VehicleMatching vehicle (QTORIN anyhydrous gel) | Drug | Vehicle |
Primary outcome
Patient Global Assessment of Activities Difficulty Scale
Time frame: 6 months
6 secondary outcomes
Pain at its worst as assessed by numerical rating scale
Time frame: 6 months
PROMIS physical function
Time frame: 6 months
Patient global impression of severity (PGI-S)
Time frame: 6 months
Patient global impression of change (PGI-C)
Time frame: 6 months
Clinician global impression of severity (CGI-S)
Time frame: 6 months
Clinician global impression of change (CGI-C)
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.
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A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
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RecruitingObservational study
International Pachyonychia Congenita Research Registry
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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.
Amy Paller, MD study chair
Northwestern University
Joyce Teng, MD principal investigator
Stanford University
David Hansen, MD principal investigator
University of Utah
8 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing QTORIN 3.9% rapamycin anhydrous gel in people with Pachyonychia Congenita, run by Palvella Therapeutics, Inc. at 8 sites in 2 countries.
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 2 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 8 sites in United States, United Kingdom. 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 phase mean?
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 Difficulty 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.