Pachyonychia CongenitaPhase 3VALO-2NCT04520750
VALO-2: Study Evaluating the Safety and Efficacy of PTX022 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 4 sites in United States.
Trial summary
VALO-2 is a multicenter, open-label extension (OLE) study enrolling patients with genotyped keratin mutations KRT6A, KRT6B and KRT16 who were previously treated with investigational PTX-022 during the VALO study. 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?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 4 sitesUnited States
- Study dates
- 1 Sep 2020to 15 Dec 2022
- 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,
- Previously completed the VALO protocol, or VALO-2 K6C/17 sub-study, and received meaningful benefit from investigational PTX-022 as determined by the clinician.
Who cannot join
- Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications or to PTX-022
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Minnesota Clinical Study Center
Fridley, Minnesota, 55432
University of Utah
Murray, Utah, 84107
Stanford University
Palo Alto, California, 94304
Arizona Research Center
Phoenix, Arizona, 85053
About this study
Official title: VALO-2: A Multicenter, Phase 3b, Open-Label Treatment Extension Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita
VALO-2 is a multicenter, open-label extension (OLE) study enrolling patients with genotyped keratin mutations KRT6A, KRT6B and KRT16 who were previously treated with investigational PTX-022 during the VALO study. The purpose of the OLE study is to investigate long term exposure to investigational PTX-022 and evaluate safety and efficacy data. A sub-study is included to evaluate safety and efficacy of patients with genotyped keratin mutations of KRT6C and KRT17 not previously treated with investigational 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
Safety and Efficacy of PTX-022 in the Treatment of Adults with Pachyonychia Congenita
| Study group | Role | What they receive |
|---|---|---|
| Open label treatment arm | Experimental | PTX-022 QTORIN Receives: Drug: PTX-022 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events
Measured: 18 Months
Changes in vital sign measurements from baseline
Measured: 18 months
Changes in weight measurements from baseline
Measured: 18 months
Changes in clinical laboratory measurements from baseline
Measured: 18 months
Secondary outcomes (6)
Patient Global Assessment of Activities Scale
Measured: 18 Months
Pain at its worst as assessed by NRS
Measured: 18 months
PROMIS Pain Interference Short 6A as assessed by likert scale
Measured: 18 months
PROMIS Physical Function as assessed by likert scale
Measured: 18 months
Patient Global Impression of Severity as assessed by likert scale
Measured: 18 months
Clinician Global Impression of Severity as assessed by likert scale
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2020
- Study start1 Sep 2020
- Primary completion30 Jun 2021
- Study completion15 Dec 2022
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
- Model description
- Open label
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Palvella Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04520750
- Sponsor's protocol ID
- PALV-03
- Acronym
- VALO-2
- Status last verified
- Aug 2024
- First submitted
- 6 Mar 2020
- Last updated
- 29 Aug 2024
- Results posted
- No
- Data monitoring committee
- 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
- VALO-2: A Multicenter, Phase 3b, Open-Label Treatment Extension Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita
- ClinicalTrials.gov ID
- NCT04520750
- Acronym / sponsor ID
- VALO-2 / PALV-03
- Conditions
- Pachyonychia Congenita
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Palvella Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Sep 2020 (actual)
- Primary completion
- 30 Jun 2021 (actual)
- Study completion
- 15 Dec 2022 (actual)
- First posted
- 20 Aug 2020 (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.
VALO-2 is a multicenter, open-label extension (OLE) study enrolling patients with genotyped keratin mutations KRT6A, KRT6B and KRT16 who were previously treated with investigational PTX-022 during the VALO study. The purpose of the OLE study is to investigate long term exposure to investigational PTX-022 and evaluate safety and efficacy data. A sub-study is included to evaluate safety and efficacy of patients with genotyped keratin mutations of KRT6C and KRT17 not previously treated with investigational PTX-022.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open label
| Arm | Type | Receives |
|---|---|---|
| Open label treatment arm | Experimental | Drug: PTX-022PTX-022 QTORIN |
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,
- Previously completed the VALO protocol, or VALO-2 K6C/17 sub-study, and received meaningful benefit from investigational PTX-022 as determined by the clinician.
Key exclusion criteria (1 listed)
- Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications or to PTX-022
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-022Safety and Efficacy of PTX-022 in the Treatment of Adults with Pachyonychia Congenita | Drug | Open label treatment arm |
Primary outcome
Incidence of Treatment-Emergent Adverse Events
Time frame: 18 Months
Changes in vital sign measurements from baseline
Time frame: 18 months
Changes in weight measurements from baseline
Time frame: 18 months
Changes in clinical laboratory measurements from baseline
Time frame: 18 months
6 secondary outcomes
Patient Global Assessment of Activities Scale
Time frame: 18 Months
Pain at its worst as assessed by NRS
Time frame: 18 months
PROMIS Pain Interference Short 6A as assessed by likert scale
Time frame: 18 months
PROMIS Physical Function as assessed by likert scale
Time frame: 18 months
Patient Global Impression of Severity as assessed by likert scale
Time frame: 18 months
Clinician Global Impression of Severity as assessed by likert scale
Time frame: 18 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.
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
RecruitingPhase 3
A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
Pachyonychia Congenita
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.
The record lists no contact. Use the official record to reach the sponsor.
4 sites 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 4 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 2 inclusion and 1 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 4 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. 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?
Incidence of Treatment-Emergent Adverse Events (time frame: 18 Months). The study lists 4 primary outcomes in all. 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.