Pachyonychia CongenitaPhase 3NCT05643872
A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
A Phase 3 study testing PTX-022 in people with Pachyonychia Congenita, run by Palvella Therapeutics, Inc. at 1 site in United States.
Trial summary
PALV-08 is a multicenter, open-label treatment (OLT) study enrolling adults with Pachyonychia Congenita (PC) with genotyped keratin mutations KRT6A, KRT6B, KRT6C or KRT16 who were previously enrolled in the PALV-05 (VAPAUS) trial. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Pachyonychia Congenita
- What is being tested?
- PTX-022
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Nov 2022to Nov 2023
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completed the PALV-05 (VAPAUS) study
- Agree to contraceptive use
Who cannot join
- Females who are pregnant or breastfeeding
- Concomitant use of sirolimus or any sirolimus-containing medications (systemic or topical)
- Any significant concurrent condition that could adversely affect participation and/or the assessment of the safety and efficacy in the study
- Prior or planned treatment, including surgery or other medically necessary intervention deemed by the investigator that could adversely affect participation and/or the assessment of the safety and efficacy in the study
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Minnesota Clinical Study Center
New Brighton, Minnesota, 55112
Study Coordinator, 763-502-2929
Steven E Kempers, MD, FAAD
About this study
Official title: A Multicenter Open-Label Treatment Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
PALV-08 is a multicenter, open-label treatment (OLT) study enrolling adults with Pachyonychia Congenita (PC) with genotyped keratin mutations KRT6A, KRT6B, KRT6C or KRT16 who were previously enrolled in the PALV-05 (VAPAUS) trial.
The purpose of this OLT study is to investigate the safety of long term exposure and pharmacokinetics (PK) of QTORIN rapamycin 3.9% anhydrous gel or "PTX-022".
- Conditions
- Pachyonychia Congenita
- Being tested
- PTX-022
- 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
QTORIN rapamycin 3.9% anhydrous gel
| Study group | Role | What they receive |
|---|---|---|
| PTX-022 | Experimental | Not described Receives: Drug: PTX-022 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment emergent adverse events
Measured: 6 months
Secondary outcomes (1)
Pharmacokinetic parameters
AUC area under the sirolimus blood concentration-time profile
Measured: Prior to dose, 12 hours and 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2022
- Study start15 Nov 2022
- Primary completionOct 2023 (estimated)
- Study completionNov 2023 (estimated)
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Palvella Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05643872
- Sponsor's protocol ID
- PALV-08
- Status last verified
- Dec 2022
- First submitted
- 8 Nov 2022
- Last updated
- 9 Dec 2022
- 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 9 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Open-Label Treatment Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
- ClinicalTrials.gov ID
- NCT05643872
- Acronym / sponsor ID
- PALV-08
- Conditions
- Pachyonychia Congenita
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- Palvella Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Nov 2022 (actual)
- Primary completion
- Oct 2023 (estimated)
- Study completion
- Nov 2023 (estimated)
- First posted
- 9 Dec 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.
PALV-08 is a multicenter, open-label treatment (OLT) study enrolling adults with Pachyonychia Congenita (PC) with genotyped keratin mutations KRT6A, KRT6B, KRT6C or KRT16 who were previously enrolled in the PALV-05 (VAPAUS) trial.
The purpose of this OLT study is to investigate the safety of long term exposure and pharmacokinetics (PK) of QTORIN rapamycin 3.9% anhydrous gel or "PTX-022".
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PTX-022 | Experimental | Drug: PTX-022 |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Completed the PALV-05 (VAPAUS) study
- Agree to contraceptive use
Key exclusion criteria (4 listed)
- Females who are pregnant or breastfeeding
- Concomitant use of sirolimus or any sirolimus-containing medications (systemic or topical)
- Any significant concurrent condition that could adversely affect participation and/or the assessment of the safety and efficacy in the study
- Prior or planned treatment, including surgery or other medically necessary intervention deemed by the investigator that could adversely affect participation and/or the assessment of the safety and efficacy in the 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 |
|---|---|---|
| PTX-022QTORIN rapamycin 3.9% anhydrous gel | Drug | PTX-022 |
Primary outcome
Treatment emergent adverse events
Time frame: 6 months
1 secondary outcome
Pharmacokinetic parameters
Time frame: Prior to dose, 12 hours and 24 hours
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Dec 2022.
- Individual participant dataSharing planned: No
Related trials for Pachyonychia Congenita
Not yet recruitingPhase 2
Safety, Tolerability, and Efficacy of Oral KM-023 in Adult Patients With Pachyonychia Congenita (pEDD-PC)
Pachyonychia Congenita
Not yet recruitingPhase 1/2
Epidermal Growth Factor Receptor Inhibition for Keratinopathies
Epidermolytic Ichthyosis, Palmoplantar Keratoderma
RecruitingObservational study
International Pachyonychia Congenita Research Registry
Pachyonychia Congenita
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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing PTX-022 in people with Pachyonychia Congenita, run by Palvella Therapeutics, Inc. at 1 site 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 2 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 9 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Emily Cook (267-738-6366, emily.cook@palvellatx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment emergent adverse events (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 9 Dec 2022.