Microcystic Lymphatic Malformation
Conditions
Brief 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.
Interventions
DRUGPTX-022
Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations
Sponsors
Palvella Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
This is an open-label, baseline-controlled study
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of Treatment-Emergent Adverse Events | 4 months |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Experimental: PTX-022 PTX-022 QTORIN
PTX-022: Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations | 12 |
| Total | 12 |
Baseline characteristics
| Characteristic | Experimental: PTX-022 |
|---|---|
| Age, Categorical <=18 years | 5 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 7 Participants |
| Age, Continuous | 27.3 years STANDARD_DEVIATION 13.56 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 10 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race/Ethnicity, Customized Race American Indian or Alaska Native | 0 Participants |
| Race/Ethnicity, Customized Race Asian | 1 Participants |
| Race/Ethnicity, Customized Race Black or African American | 2 Participants |
| Race/Ethnicity, Customized Race Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race/Ethnicity, Customized Race Other | 1 Participants |
| Race/Ethnicity, Customized Race White | 8 Participants |
| Region of Enrollment United States | 12 participants |
| Sex: Female, Male Female | 7 Participants |
| Sex: Female, Male Male | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 12 |
| other Total, other adverse events | 9 / 12 |
| serious Total, serious adverse events | 0 / 12 |
Outcome results
Primary
Incidence of Treatment-Emergent Adverse Events
Time frame: 4 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Experimental: PTX-022 | Incidence of Treatment-Emergent Adverse Events | Subjects with TEAEs | 9 participants |
| Experimental: PTX-022 | Incidence of Treatment-Emergent Adverse Events | Gastrointestinal Disorders | 2 participants |
| Experimental: PTX-022 | Incidence of Treatment-Emergent Adverse Events | General Disorders and Administration Site Conditions | 6 participants |
| Experimental: PTX-022 | Incidence of Treatment-Emergent Adverse Events | Infections and Infestations | 2 participants |
| Experimental: PTX-022 | Incidence of Treatment-Emergent Adverse Events | Investigations | 1 participants |
| Experimental: PTX-022 | Incidence of Treatment-Emergent Adverse Events | Musculoskeletal and Connective tissue Disorders | 1 participants |
| Experimental: PTX-022 | Incidence of Treatment-Emergent Adverse Events | Nervous System Disorders | 2 participants |
| Experimental: PTX-022 | Incidence of Treatment-Emergent Adverse Events | Renal and Urinary Disorders | 1 participants |
| Experimental: PTX-022 | Incidence of Treatment-Emergent Adverse Events | Skin and Subcutaneous Tissue Disorders | 4 participants |