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.

Pulmonary Lymphangioleiomyomatosis

Saracatinib is escalated as follows: Three dose levels will be administered for 1 month each: 50 mg, 125 mg and 175 mg. Three subjects will be treated at the lowest daily dose of 50 mg. If no subject experiences DLT (dose limiting toxicity), the dose level is escalated to 125 mg/day for the next cohort of 3 different subjects and so on to next dose.

Saracatinib

University of Cincinnati

Baylor College of Medicine

University of Texas Health Science Center-Houston

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

One of three escalating daily oral doses of saracatinib; 50, 125 and 175 mg. Saracatinib will be given orally once a day for four weeks. Adverse events will be monitored. The subject will receive only one of the doses as determined by the escalation of the study.

This objective is to generate a safety profile utilizing the data collected along with the adverse events. Subjects will be followed closely during the 4 weeks while taking drug and again for four weeks after stopping the study drug.

explore the efficacy measurements (e.g., pulmonary function test, six minute walk test, and VEGF-D)

The subject is aware of his/her data at the time of the study. They are told if they are/are not using the correct technique for MDI. There is no more data to share with the subject.

Tony Eissa

Professor of Medicine - Pulmonary

University of Texas

Patients with lymphangioleiomyomatosis and lymphatic tumors, ascites or pleural effusions who are symptomatic will receive subcutaneous injections of octreotide starting at a dose of 100 micrograms per day. Doses will be gradually increased to a maximum of 800 micrograms per day, two months after enrollment, if there is no response to lower doses.

Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain

Octreotide

Sex: Female, Male

Region of Enrollment

Octreotide treatment will be considered successful if the patient receiving treatment for six months shows a reduction in total tumor mass/ fluid collection or reaccumulation of at least 20%.

Number of Participants With a Reduction in Total Tumor Volume of at Least 20%.

Octreotide treatment will be considered successful if the reported pain/symptom score is reduced by at least 2 levels at termination of treatment.
A simple visual numeric distress scale ranging from zero to 10 will be employed to rate the severity of individual symptoms. The best score is zero, which means absence of symptoms and the maximal is 10, meaning that the symptoms are very severe.

Number of Participants With a Reduction of Pain/Symptoms as Measured by a Simple Numeric Symptom Distress Scale (NDS) to Rate the Severity of Individual Symptoms.

One or more abnormality of the following liver function tests:
Alkaline phosphatase above 116 i.u.
SGPT above 41 i.u.
SGOT from 34 i.u.
Total bilirubin above 1.0 mg/dl

Number of Participants With Liver Function Abnormalities

Overall Study

Joel Moss

Lymphangioleiomyomatosis

Lymphangiomyomas

Pleural Effusions

Ascites

Treatment with octreotide starts at a dose of 50 micrograms(ug) twice a day which is increased to 100 ug twice a day after two weeks and to 200 ug twice a day two weeks later. After two months, if there is no response the dose shall be increased to 400 ug twice a day.

lymphangioleiomyomatosis/ödəm

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

Treatment With Octreotide in Patients With Lymphangioleiomyomatosis

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