A Study of Propranolol to Treat Kaposi Sarcoma
This phase II trial tests how well propranolol hydrochloride (propranolol) works in treating patients with Kaposi sarcoma. Kaposi sarcoma is a type of cancer in which lesions (abnormal areas) grow in the skin, lymph nodes, lining of the mouth, nose, and throat, and other tissues of the body. The lesions are usually purple and are made of cancer cells, new blood vessels, and blood cells. They may begin in more than one place in the body at the same time. Propranolol is a drug that is classified as a beta-blocker. Beta-blockers affect the heart and circulation (blood flow through arteries and veins). Propranolol has been found to be safe and effective for treating other types of skin problems, and may be effective in treating patients with Kaposi sarcoma.
Inclusion Criteria
- Participant (or parent/legal guardian) has the ability to understand and the willingness to sign a written informed consent document
- Pediatric (< 18 years) or adult (≥ 18 years) participants with biopsy-proven and measurable KS as defined in the KS Manual of Procedures (MOP)
- For pediatric (< 18 years) participants, KS stage must be one of the following: * 1A (Mild): disease limited to skin, flat oral mucosal lesions, and/or flesh-colored subcutaneous nodules, total < 10 lesions or * 1B (Moderate): having any of the following features, alone or in combination: a total of 10-19 hyperpigmented skin/oral lesions, nodular oral involvement, conjunctival eye involvement, or exophytic mass.
- For adult (≥ 18 years) participants, KS stage must be one of the following: * T0: confined to skin and/or lymph nodes and/or minimal oral lesions or * T1 limited to tumor-associated edema of cutaneous lesions without functional impairment or flat oral lesions
- For pediatric (< 18 years) participants: Lansky performance status > 70%
- For adult (≥ 18 years) participants: Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
- Bilirubin (direct or total) within normal age-specific range, or total bilirubin < 3.0 mg/dl for participants with Gilbert syndrome (at screening)
- Calculated creatinine clearance >= 30 mL/min for participants >= 12 years; serum creatinine < 1.5 upper limit of normal (ULN) for participants < 12 years (at screening)
- Hemoglobin > 9 g/dL (at screening)
- Platelets > 100 × 10^9/L (at screening)
- Absolute neutrophil count (ANC) > 1000 cells/mm^3 (at screening)
- If HIV positive, participant is receiving antiretroviral therapy (ART) that conforms to local standards of care. Participant has been on a stable ART regimen for at least 12 weeks prior to study dosing. Participants will not be excluded based on CD4 count or HIV viral load
- If HIV positive, participant has to not show recent improvement on ART that may confound response evaluation prior to study dosing: * If on ART 12 to 24 weeks, prior to study dosing, participant has shown evidence of KS progression requiring further systemic treatment * If on ART for > 24 weeks, prior to study dosing, participant has no evidence of regression in the last eight weeks prior to study dosing
- HIV-negative participants must not show evidence of improvement in the three months prior to study dosing
- Participant has no history of diabetes mellitus (as it is a risk factor for hypoglycemia)
- Participant has no history of a diagnosed obstructive airway disease such as asthma, chronic obstructive pulmonary disease (COPD), or bronchiolitis
- Participant has no clinically significant cardiovascular disease other than hypertension, which is permitted
- Participant has not used beta-adrenergic antagonists for other indications within the past 6 months
- Female participants of childbearing potential (defined as a sexually mature woman who has not undergone a hysterectomy or bilateral oophorectomy or has not been naturally postmenopausal for at least 24 consecutive months) agree to use effective contraception. Female participants of childbearing potential must agree to use adequate contraception (oral contraceptive pills, intrauterine device, Nexplanon, Depo-Provera, or permanent sterilization, etc., or another acceptable method as determined by the investigator) prior to study entry, for the duration of study participation
- Female participants of childbearing potential are not pregnant or planning to become pregnant as proven by serum beta-human chorionic gonadotropin (BhCG) at screening and urine test on visit 1 (day 1) prior to study drug administration. Propranolol is United States (US) Food and Drug administration (FDA) pregnancy category C. At this time, the study team has determined that the unknown risk to a developing fetus is greater than the potential benefit of treatment
- Female participants are not breast feeding or planning on breast feeding for the duration of study participation
Exclusion Criteria
- Pediatric (< 18 years) participants have lymph node or symptomatic or asymptomatic visceral disease, woody edema, or ≥ 20 cutaneous lesions
- Pediatric (< 18 years) participants have heart rate or systolic blood pressure < 10th percentile for age
- Adult (≥ 18 years) participants have symptomatic or asymptomatic visceral disease or tumor-associated edema causing functional impairment
- Participant has clinically significant shortness of breath, hemoptysis, or moderate/severe cough
- Participant has clinically significant bleeding from the mouth or rectum
- Participant is receiving treatment for an active and serious infection
- Participant has evidence of an active infection based on local screening practices and epidemiology
- Children (< 12 years) with severe acute malnutrition based on World Health Organization (WHO) criteria (mid- upper arm circumference < 11.5 cm, weight-for height Z-score < -3, or presence of symmetrical pitting edema)
- Participant has a known allergy to propranolol or allergy to other beta (β)-blockers
- Patients with a history of uncompensated heart failure; severe sinus bradycardia; sick sinus syndrome; or heart block greater than first-degree
- Participant is receiving concurrent treatment with an anticancer therapy. Participant must not have received any anticancer therapies such as cytotoxic chemotherapy within 30 days prior to receiving the first dose of study drug
- Participant has high clinical suspicion of concurrent Castleman disease or interleukin (IL)-6 related inflammatory disease* * Signs and symptoms consistent with IL-6 related inflammatory disease or Symptomatic Kaposi Sarcoma Herpesvirus (KSHV) Inflammatory Cytokine Syndrome (KICS) were reviewed in Clinical Features and Outcomes of Patients with Symptomatic Kaposi Sarcoma Herpesvirus (KSHV)-associated Inflammation: Prospective Characterization of KSHV KICS
- Participant has been treated with systemic corticosteroids within two months of screening (excluding treatment for adrenal insufficiency)
- Participant has an active tuberculosis (TB) infection, with or without current treatment
- Participant is receiving concurrent investigational drugs or concurrently participating in another clinical trial
- Any active comorbidity which, in the opinion of the investigator, would interfere with protocol objectives
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT05797662.
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PRIMARY OBJECTIVE:
I. To determine the objective response rate (ORR) when participants with Kaposi sarcoma (KS) are treated with propranolol hydrochloride (propranolol).
SECONDARY OBJECTIVES:
I. To evaluate the safety and tolerability of propranolol in participants with KS.
II. To evaluate the safety, tolerability, and overall response rates, complete + partial response (CR + PR) separately in children and adults.
III. To evaluate the duration of response.
OUTLINE:
Patients receive propranolol orally (PO) twice daily (BID) for 2 or 4 days. Patients who tolerate propranolol then continue to receive propranolol PO BID on days 1-28 of each cycle. Cycles repeat every 28 days for 3 cycles in the absence of disease progression or unacceptable toxicity. Patients with CR or PR receive propranolol PO BID for additional 84 days in the absence of disease progression or unacceptable toxicity. Patients also undergo chest x-ray and possible computed tomography (CT) at screening and undergo collection of blood samples on study.
After completion of study treatment, all patients are followed up at 60 days.
Trial PhasePhase II
Trial Typetreatment
Lead OrganizationAIDS Malignancy Consortium
Principal InvestigatorShane McAllister
- Primary IDAMC-116
- Secondary IDsNCI-2023-03576
- ClinicalTrials.gov IDNCT05797662