Safety and Efficacy of Saracatinib In Subjects With Lymphangioleiomyomatosis

• Status: Terminated
• Trial end date: 2019-07-30
• Target enrollment: 0
• Participant gender: Female

Summary

This study is being done to determine if there is a potential benefit of saracatinib in LAM subjects. Based on the information of this trial, additional clinical development trials will be needed. The study will also test the tolerability of 125 mg of saracatinib given once daily over a 9 month period.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Baylor College of Medicine
• Collaborators: Brigham and Women's Hospital; Loyola University; National Institutes of Health (NIH); Stanford University; University of Cincinnati; University of South Florida
• Treatments: Saracatinib

Criteria

Inclusion Criteria:

• Female patients. It should be noted that LAM occurs almost exclusively in women.

• 18 to 65 years of age.

• All patients must have a diagnosis of LAM as defined by compatible cystic changes on chest computed tomography (CT) and one of the following:

• Open lung, transbronchial or thoracic needle biopsy consistent with LAM

• Open or needle abdominal biopsy findings consistent with LAM

• Clinical findings of tuberous scleroma complex (TSC), renal angiomyolipoma, cystic abdominal lymphangiomas, or history of chylous effusion in the chest or abdomen

• Serum vascular endothelial growth factor D (VEGF-D) > 800 pg/ml

• Subjects must have had a recent reduction in forced expiratory volume at 1-second (FEV1) of > 50ml/year, as shown by at least two pulmonary function testing (PFT) measured at least 6 months apart in the last 24 months prior to enrolling study.

Exclusion Criteria:

• Current infection.

• Major surgery within the past 2 months

• Advanced hematologic, renal, hepatic, non-LAM lung disease or metabolic diseases; or BMI of >35

• The use of another investigational drug within 30 days

• The use of mTOR (mammalian target of rapamycin) inhibitors within 30 days

• Previous lung transplantation.

• 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 status post successful excision or treatment.

• 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