Microcystic Lymphatic MalformationPhase 2NCT05050149
Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations
A Phase 2 study testing PTX-022 in people with Microcystic Lymphatic Malformation, run by Palvella Therapeutics, Inc. at 7 sites in United States.
Trial summary
This study evaluates the safety and efficacy of PTX-022 (sirolimus) Topical Gel 3.9% w/w in the treatment of Microcystic Lymphatic Malformations. The participant will receive 3 months of PTX-022 treatment by the end of the study. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Microcystic Lymphatic Malformation
- What is being tested?
- PTX-022
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 7 sitesUnited States
- Study dates
- 13 Jan 2022to 15 Dec 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participants must be 6 years or older
- Diagnosed with Microcystic Lymphatic Malformations
- 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 that could adversely affect participation.
- Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications or to PTX-022
- 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
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Minnesota Clinical Study Center
Fridley, Minnesota, 55432
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Vascular Birthmark Institute
New York, New York, 10065
Stanford University
Palo Alto, California, 94305
Children's Hospital of Pennsylvania (CHOP)
Philadelphia, Pennsylvania, 19104
Oregon Health and Science University
Portland, Oregon, 97239
About this study
Official title: A Multicenter, Phase 2, Open-Label Study Evaluating The Safety And Efficacy of Sirolimus 3.9% Topical Gel (PTX-022) In The Treatment of Microcystic Lymphatic Malformations
This study evaluates the safety and efficacy of PTX-022 (sirolimus) Topical Gel 3.9% w/w in the treatment of Microcystic Lymphatic Malformations. The participant will receive 3 months of PTX-022 treatment by the end of the study.
- Conditions
- Microcystic Lymphatic Malformation
- Being tested
- PTX-022
- Medical subject headings
- Lymphangioma
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 Microcystic Lymphatic Malformations
| Study group | Role | What they receive |
|---|---|---|
| Experimental: PTX-022 | 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: 4 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2021
- Study start13 Jan 2022
- Primary completion15 Nov 2022
- Study completion15 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Microcystic Lymphatic MalformationStudies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is an open-label, baseline-controlled study
- Masking
- None (open label)
- Enrolment
- 12 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
- NCT05050149
- Sponsor's protocol ID
- PALV-06
- Status last verified
- Dec 2024
- First submitted
- 8 Sep 2021
- Last updated
- 17 Jan 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Teng JMC, Hammill A, Martini J, Treat J. Sirolimus in the Treatment of Microcystic Lymphatic Malformations: A Systematic Review. Lymphat Res Biol. 2023 Apr;21(2):101-110. doi: 10.1089/lrb.2021.0103. Epub 2022 Jul 18.
PubMed 35852876
Study at a glance
From the registry record, last updated by the sponsor on 17 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Phase 2, Open-Label Study Evaluating The Safety And Efficacy of Sirolimus 3.9% Topical Gel (PTX-022) In The Treatment of Microcystic Lymphatic Malformations
- ClinicalTrials.gov ID
- NCT05050149
- Acronym / sponsor ID
- PALV-06
- Conditions
- Microcystic Lymphatic Malformation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Palvella Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Jan 2022 (actual)
- Primary completion
- 15 Nov 2022 (actual)
- Study completion
- 15 Dec 2022 (actual)
- First posted
- 20 Sep 2021 (actual)
- Results
- Posted 17 Jan 2025
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 (sirolimus) Topical Gel 3.9% w/w in the treatment of Microcystic Lymphatic Malformations. The participant will receive 3 months of PTX-022 treatment by the end of the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is an open-label, baseline-controlled study
| Arm | Type | Receives |
|---|---|---|
| Experimental: PTX-022 | Experimental | Drug: PTX-022PTX-022 QTORIN |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Participants must be 6 years or older
- Diagnosed with Microcystic Lymphatic Malformations
- 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 that could adversely affect participation.
- Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications or to PTX-022
- 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-022Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations | Drug | Experimental: PTX-022 |
Primary outcome
Incidence of Treatment-Emergent Adverse Events
Time frame: 4 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jan 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 35852876Teng JMC, Hammill A, Martini J, Treat J. Sirolimus in the Treatment of Microcystic Lymphatic Malformations: A Systematic Review. Lymphat Res Biol. 2023 Apr;21(2):101-110. doi: 10.1089/lrb.2021.0103. Epub 2022 Jul 18.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PTX-022 in people with Microcystic Lymphatic Malformation, run by Palvella Therapeutics, Inc. at 7 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 4 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 17 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Jan 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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: 4 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05050149, which also lists 1 linked publication.
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 17 Jan 2025.