Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies

Completed · Phase 2

Conditions studied: Kaposiform Hemangioendotheliomas, Tufted Angioma, Capillary Venous Lymphatic Malformation, Venous Lymphatic Malformation, Microcystic Lymphatic Malformation, Mucocutaneous Lymphangiomatosis and Thrombocytopenia, Capillary Lymphatic Arterial Venous Malformations, PTEN Overgrowth Syndrome With Vascular Anomaly, Lymphangiectasia Syndromes

In brief

The purpose of this study is to determine if the use of sirolimus in the treatment of children and young adults with complicated vascular anomalies will prove to be safe and provide objective response resulting in improved clinical status and quality of life. Funding Source - FDA OOPD (Food and Drug Administration - Office of Orphan Products Development)

Key facts

Study ID

NCT00975819

Run by

Denise Martin Adams

People needed

61

Starts

2009-10-01

Expected to finish

2020-10-01

Last updated by the study team

2026-01-06

Who can join

Age: any, up to 31. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Inclusion will be strictly limited to children and young adults with vascular anomalies with complications that require systemic therapy for control.
• Diagnosis: All patients must have one of the following vascular anomalies as determined by clinical, radiographic and histologic criteria (when possible):
• Kaposiform Hemangioendotheliomas with Kasabach-Merritt Phenomenon
• Kaposiform Hemangioendotheliomas without Kasabach-Merritt Phenomenon
• Tufted Angioma with Kasabach-Merritt Phenomenon
• Tufted Angioma without Kasabach-Merritt Phenomenon
• Capillary Lymphatico-Venous Malformation (CLVM)
• Venous Lymphatic Malformation (VLM)
• Microcystic Lymphatic Malformation (MLM)
• Multifocal Lymphangiomatosis and Thrombocytopenia (MLT)/Cutaneovisceral Angiomatosis and Thrombocytopenia (CAT)
• Capillary Lymphatic Arterial Venous Malformations (CLAVM)
• PTEN Overgrowth syndrome with vascular anomaly
• Lymphangiectasia Syndromes
• If archived tissue is available, histological diagnosis will be confirmed by the pathology lab at the enrolling site.
• Complications: Patients must have vascular anomalies that have potential to cause significant morbidity. In addition to the above diagnosis, one or more of the following criteria needs to be met:
• Coagulopathy
• Chronic pain
• Recurrent cellulitis (>3 episodes/year)
• Ulceration
• Visceral and/or bone involvement
• Cardiac dysfunction
• Age: Patients must be 0 - 31 years of age at the time of study entry. Enrollment includes patients of both genders and all ethnic groups.
• Organ function requirements:
• Adequate liver function defined as:
• Total bilirubin (sum of conjugated and unconjugated) ≤1.5 x ULN for age, and

You may not qualify if…

• Dental braces or prosthesis only if it interferes with radiologic analysis of vascular anomaly.
• Concurrent severe and/or uncontrolled medical disease which could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe infection, severe malnutrition, chronic liver or renal disease, active upper GI tract ulceration).
• Chronic treatment with systemic steroids or another immunosuppressive agent. Patients with endocrine deficiencies are allowed to receive physiologic or stress doses of steroids if necessary. Patients with the diagnosis of a vascular tumor (KHE, TA) can be on a weaning dose of steroids.
• Patients who require medications that inhibit/induce CYP3A4 enzyme activity to control concurrent medical conditions.
• Known history of HIV seropositivity or known immunodeficiency. Testing is not required unless a condition is suspected.
• Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of sirolimus (e.g. ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome or small bowel resection). A gastric tube or nasogastric tube is allowed.
• Women who are pregnant or breast feeding.
• Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method during the period they are receiving the study drug and for 3 months thereafter. Abstinence is an acceptable method of birth control. Women of childbearing potential will be given a pregnancy test within 7 days prior to administration of sirolimus and must have a negative urine or serum pregnancy test.
• Patients who have received prior treatment with an mTOR inhibitor.
• Patients unwilling or unable to comply with the protocol, or who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study.
• Patients who currently have an uncontrolled infection, defined as receiving intravenous antibiotics.

Where it is running

• Children's Hospital Boston — Boston, Massachusetts, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States

Full record on ClinicalTrials.gov

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