IRB Study Number 24-577
Status Recruiting
Institute Taussig Cancer Institute
Description
Primary Objective
To determine the efficacy of once-daily treatment with PTX-022 at 168 days for the treatment of microcystic LM using the overall microcystic LM Investigator Global Assessment (mLM-IGA), as assessed live by a clinician.
Secondary Objectives
To determine the efficacy of once-daily treatment with PTX-022 for the treatment of microcystic LM utilizing scores from the independent endpoint assessment committee (IEAC) of the clinician microcystic LM Multicomponent Static Scale (mLM-MCSS).
To determine the efficacy of once-daily treatment with PTX-022 for the treatment of microcystic LM using the mLM-MCSS live assessment, overall Patient Global Impression of Change (PGI-C), the overall Clinician Global Impression of Severity (CGI-S), and the overall Patient Global Impression of Severity (PGI-S).
Inclusion Criteria
1) The participant must be ≥6 years of age at consent.
2) The participant must have a clinically confirmed superficial/ cutaneous (visible on the skin) microcystic lymphatic malformation (microcystic LM), also referred to as the treatment area.
Via in-person skin examination, the investigator must identify that the microcystic LM:
a. is present on the surface of the skin as evidenced by blebs and/or vesicles and/or papules and/or plaques and/or leaking, crusting or bleeding on the surface of the skin, consistent with a microcystic LM.
b. treatment area is ≥ moderate on the overall CGI-S severity scale and at least two individual signs of ≥ moderate on the microcystic LM multicomponent static scale (mLM-MCSS) as rated during live assessment by the clinician.
c. is not interfering with other vascular malformations to the extent that, in the opinion of the investigator, the participant should be excluded
d. does not have macrocystic LM or mixed-type LM within or proximate to the treatment area
3) The participant’s treatment area microcystic LM is ≥ moderate on the overall PGI-S severity scale. For pediatric participants age 6 to less than 12 years old, the caregiver PGI-S will be used for eligibility.
4) The microcystic LM treatment area must have a defined total area of >~9 cm2, and less than < ~200 cm2. The treatment area does not need to be contiguous but must include no more than three total areas to treat.
5) At least 4 weeks have elapsed since the participant has had any major surgery and 12 months since surgery on the treatment area.
6) At least 14 days have elapsed since the participant completed therapy with a Growth Factor (GF) that supports platelet, red or white cell number or function.
7) At least 4 weeks has elapsed since the participant has received investigational drug or biologic (4 weeks or 5 half-lives, whichever is longer), prior to starting treatment with and during treatment with PTX-022.
8) At least 5 months has elapsed since the participant received field radiation (XRT) administered to the microcystic LM treatment area.
9) The participant is willing to abstain from application of other prescription or over the counter topical medications in the treatment area during the treatment period. Moisturizers and emollients are allowed.
10) The participant is willing to forego medical interventional treatment (i.e., topical or systemic pharmacological treatment or interventions such as sclerotherapy) of their microcystic LM with anything other than the study drug during the Treatment Period except when the Investigator believes that delay of treatment potentially might compromise the health of the subject.
11) For participants aged ≥6 years old to <12 years old, a caregiver completing the participant assessments is available to remain consistent throughout the study as much as possible.
Exclusion Criteria
1) The participant has previously participated in a clinical trial evaluating PTX-022.
2) The participant’s treatment area is mainly in any wet mucosa or within the orbital rim. External genital presentation is permitted.
3) Participants who are pregnant, breastfeeding or planning to become pregnant during the study including through the follow-up period.
4) Participants of childbearing potential who are unwilling or unable to comply with contraception measures.
5) The participant has any condition or situation which, in the Investigator's opinion, may put the subject at significant risk, could confound the study results, or could interfere significantly with the subject's participation in the study.
6) Participants deemed by the investigator as unwilling or unable to remain compliant with all tests and procedures, adherence to the study drug administration regimen and other protocol-required activities.
7) The participant has used oral or topical sirolimus, an mTOR inhibitor, any other mTOR inhibitors or a PI3K inhibitor in the past 6 months.
8) The participant is using or has used a topical, oral, or interventional treatment in the previous 3 months that might interfere with the evaluation of the study IP.
9) The participant has had photodynamic therapy (PDT) sclerotherapy or other laser surgeries to the microcystic LM identified for the study within 3 months.
10) The participant is currently taking antibiotics.
11) The participant has had a recent injury to the treatment area that in the opinion of the investigator caused an exacerbation of symptoms.
12) Participant has a known hypersensitivity to sirolimus.
13) Participant reports they are HIV seropositive or has another known immunodeficiency.
14) Participant has complicated vascular anomalies with severe systemic symptoms that require systemic therapy.
15) Participant previously treated for cancer within the past 5 years unless the Investigator concludes history of cancer is not confounding to safety.
16) Interpretable photographs of the treatment area are unable to be obtained.
