A Phase 2 interventional study of PTX-022 in Microcystic Lymphatic Malformation, sponsored by Palvella Therapeutics, Inc.. Completed at 7 sites in United States. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2025-01-17.
Sponsored by Palvella Therapeutics, Inc. · Phase 2, Interventional, and Treatment
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.
13 studies on the registry are indexed under Lymphangioma; 5 are open to participants now.
Browse Lymphangioma studies →Palvella Therapeutics, Inc. is the lead sponsor of 9 studies on the registry; 2 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 1 (20%) have results posted.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
PTX-022 QTORIN
Drug: PTX-022
Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations
Incidence of Treatment-Emergent Adverse Events
Time frame: 4 months
| Milestone | Experimental: PTX-022 |
|---|---|
| Started | 12 |
| Completed | 12 |
| Not completed | 0 |
| participants | Experimental: PTX-022 |
|---|---|
| Subjects with TEAEs | 9 |
| Gastrointestinal Disorders | 2 |
| General Disorders and Administration Site Conditions | 6 |
| Infections and Infestations | 2 |
| Investigations | 1 |
| Musculoskeletal and Connective tissue Disorders | 1 |
| Nervous System Disorders | 2 |
| Renal and Urinary Disorders | 1 |
| Skin and Subcutaneous Tissue Disorders | 4 |
Collected over Adverse events were collected from screening until Month 4. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Experimental: PTX-022 | 0/12 (0%) | 0/12 (0%) | 9/12 (75%) |
| Event | Experimental: PTX-022 |
|---|---|
| Application Site PainGeneral disorders | 3/12 |
| Application Site PruritusGeneral disorders | 3/12 |
| NauseaGastrointestinal disorders | 2/12 |
| HeadacheNervous system disorders | 2/12 |
| Abdominal painGastrointestinal disorders | 1/12 |
| DiarrhoeaGastrointestinal disorders | 1/12 |
| Application Site DischargeGeneral disorders | 1/12 |
| Application Site ErythemaGeneral disorders | 1/12 |
| Application Site ParaesthesiaGeneral disorders | 1/12 |
| NoduleGeneral disorders | 1/12 |
| Age, Categorical(Participants) | Experimental: PTX-022 |
|---|---|
| <=18 years | 5 |
| Between 18 and 65 years | 7 |
| >=65 years | 0 |
| Age, Continuous(years) | Experimental: PTX-022 |
|---|---|
| Mean | 27.3 ± 13.56 |
| Sex: Female, Male(Participants) | Experimental: PTX-022 |
|---|---|
| Female | 7 |
| Male | 5 |
| Ethnicity (NIH/OMB)(Participants) | Experimental: PTX-022 |
|---|---|
| Hispanic or Latino | 2 |
| Not Hispanic or Latino | 10 |
| Unknown or Not Reported | 0 |
| Race/Ethnicity, Customized(Participants) | Experimental: PTX-022 |
|---|---|
| Race — American Indian or Alaska Native | 0 |
| Race — Asian | 1 |
| Race — Black or African American | 2 |
| Race — Native Hawaiian or Other Pacific Islander | 0 |
| Race — White | 8 |
| Race — Other | 1 |
| Region of Enrollment(participants) | Experimental: PTX-022 |
|---|---|
| United States | 12 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Jan 2022 to primary completion in Nov 2022, 10 months.
Results were first posted in Jan 2025, 2 years 2 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Dec 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Sirolimus Topical Gel PTX-022
Palvella Therapeutics, Inc.