The Tolerability of Saracatinib in Subjects With Lymphangioleiomyomatosis (LAM) (SLAM-1)
Pulmonary Lymphangioleiomyomatosis
About this trial
This is an interventional treatment trial for Pulmonary Lymphangioleiomyomatosis focused on measuring LAM, saracatinib, Lymphangioleiomyomatosis
Eligibility Criteria
Inclusion Criteria:
- Male or female patients. It should be noted, however, that LAM occurs predominantly in women.
- 18 to 65 years of age.
- All patients must have a diagnosis of LAM as defined by one of the following:
Open lung, transbronchial or thoracic needle biopsy consistent with LAM Open or needle abdominal biopsy findings consistent with LAM Computed tomography (CT) of chest or abdomen consistent with LAM in the setting of TSC, renal angiomyolipoma (AML), cystic abdominal lymphangiomas, or history of chylous effusion in the chest or abdomen CT of chest consistent with LAM plus serum vascular endothelial growth factor (VEGF-D) > 800 pg/ml In cases where the diagnosis of LAM is based on biopsy, review of the pathology specimens by pathologists who are experienced with LAM, such as those at the NIH or the Mayo Clinic, will be obtained (if not done so previously).
Exclusion Criteria:
- Current infection.
- Major surgery within the past 2 months
- Advanced hematologic, renal, hepatic, or metabolic diseases
- The use of another investigational drug within 30 days
- The use of mammalian target of rapamycin (mTOR) inhibitors within 30 days
- Previous lung transplantation or active on transplant list.
- Inability to attend scheduled clinic visits
- Inability to give informed consent
- Inability to perform pulmonary function testing
- History of malignancy in the past two years, other than squamous or basal cell skin cancer or mild cervical cancer.
- Nursing mothers
- Current or planned pregnancy.
- Not using adequate contraception (in woman of childbearing potential).
- Significant clinical change in health in the past 30 days
Sites / Locations
- University of Cincinnati
- Baylor College of Medicine
- University of Texas Health Science Center-Houston
Arms of the Study
Arm 1
Experimental
Saracatinib
Only one arm: Intervention is Saracatinib. We plan to study three escalating doses of oral saracatinib; 50, 125 and 175 mg. Saracatinib is given orally once a day. More regarding dose escalation is included in intervention below.