The Tolerability of Saracatinib in Subjects With Lymphangioleiomyomatosis (LAM) (SLAM-1)

• Status: Completed
• Trial end date: 2015-07-01
• Target enrollment: 0
• Participant gender: All

Summary

Lymphangioleiomyomatosis (LAM) is a rare lung disease that mostly affects women of childbearing age. In LAM, abnormal, muscle-like cells begin to grow out of control in the lungs. As a result, air can't move freely in and out of the lungs. In some cases, this means the lungs can't supply the body's other organs with enough oxygen.

This study is being conducted to find out what dose of a drug called saracatinib is best tolerated by people with LAM. This drug has been tested in patients with certain types of cancer but is not currently approved by the United States Food and Drug Administration (FDA). Saracatinib may work in cancer by preventing the growth, movement and invasiveness of cancer cells. The use of saracatinib to treat LAM is considered experimental. Preliminary testing already completed suggests that the study drug, saracatinib, may suppress certain substances in the lungs of patients with LAM thus may be effective in slowing down the disease process

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Tony Eissa
• Collaborators: University of Cincinnati; University of Texas
• Treatments: Saracatinib

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