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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental: PTX-022 | Experimental | PTX-022 QTORIN |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PTX-022 | Drug | Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events | 4 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | United States | ||
| Stanford University |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35852876 | Derived | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Experimental: PTX-022 | PTX-022 QTORIN PTX-022: Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jun 27, 2022 |
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This is an open-label, baseline-controlled study
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| Palo Alto |
| California |
| 94305 |
| United States |
| Minnesota Clinical Study Center | Fridley | Minnesota | 55432 | United States |
| Vascular Birthmark Institute | New York | New York | 10065 | United States |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229 | United States |
| Oregon Health and Science University | Portland | Oregon | 97239 | United States |
| Children's Hospital of Pennsylvania (CHOP) | Philadelphia | Pennsylvania | 19104 | United States |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Experimental: PTX-022 | PTX-022 QTORIN PTX-022: Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Incidence of Treatment-Emergent Adverse Events | Posted | Number | participants | 4 months |
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Adverse events were collected from screening until Month 4
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Experimental: PTX-022 | PTX-022 QTORIN PTX-022: Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations | 0 | 12 | 0 | 12 | 9 | 12 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Application Site Discharge | General disorders | MedDRA 25.1 | Systematic Assessment |
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| Application Site Erythema | General disorders | MedDRA 25.1 | Systematic Assessment |
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| Application Site Pain | General disorders | MedDRA 25.1 | Systematic Assessment |
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| Application Site Paraesthesia | General disorders | MedDRA 25.1 | Systematic Assessment |
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| Application Site Pruritus | General disorders | MedDRA 25.1 | Systematic Assessment |
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| Nodule | General disorders | MedDRA 25.1 | Systematic Assessment |
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| Folliculitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Reticulocyte count increased | Investigations | MedDRA 25.1 | Systematic Assessment |
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| Joint Swelling | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
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| Micturition urgency | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
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| Blister | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Dermatitis Contact | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Eczema | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Skin Exfoliation | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Christine Kampf, Vice President of Regulatory Affairs | Palvella Therapeutics, Inc. | 484-844-0865 | christine.kampf@palvellatx.com |
| Dec 23, 2024 |
| Prot_SAP_000.pdf |
| ID | Term |
|---|---|
| D008202 | Lymphangioma |
| ID | Term |
|---|---|
| D018190 | Neoplasm, Lymphatic Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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| Unknown or Not Reported |
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| Black or African American |
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| Native Hawaiian or Other Pacific Islander |
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| White |
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| Other |
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| Title | Measurements |
|---|---|
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| Infections and Infestations |
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| Investigations |
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| Musculoskeletal and Connective tissue Disorders |
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| Nervous System Disorders |
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| Renal and Urinary Disorders |
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| Skin and Subcutaneous Tissue Disorders |
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