Protocol Number: 00-H-0147

Title:

Treatment with Octreotide in Patients with Lymphangioleiomyomatosis

Number:

00-H-0147

Summary:

Lymphangioleiomyomatosis (LAM), a disease primarily of women of child-bearing age, is characterized by cystic lung disease and abdominal tumors (e.g., angiomyolipomas). Within the LAM patient population is a subset of patients who develop chylous ascites, chylous pleural effusions, chyluria, peripheral lymphedema, and/or lymphangioleiomyomas. Lymphangioleiomyomas are believed to result from a proliferation of abnormal smooth muscle cells within the lymphatic system, which appears to obstruct fluid outflow, leading to fluid accumulation and an increase in size. The lymphangioleiomyomas may occur anywhere along the axial lymphatic chain. In patients with LAM, they occur most frequently in the thorax, abdomen and pelvis and may give rise to a myriad of symptoms (e.g., paresthesias, palpitations, peripheral edema). In some patients, treatment of many of these symptoms, i.e., elevation of lower extremities, paracentesis, thoracentesis, diuretics, and/or surgery, has been ineffective. Previous studies with somatostatin and octreotide in other clinical settings (e.g., traumatic damage to the lymphatics) have shown a successful reduction in chylous effusions, chyluria, ascites, and peripheral lymphedema, when other therapies were less effective. This study will assess the effectiveness of octreotide in symptomatic patients with LAM, lymphangioleiomyomas and/or chylous effusions/ascites, peripheral lymphedema and chyluria.

Sponsoring Institute:

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Active Accrual Of New Subjects

Gender: Female

Referral Letter Required: Yes

Population Exclusion(s): Children

Eligibility Criteria:

INCLUSION CRITERIA:

All patients enrolled in the Lymphangioleiomyomatosis natural history protocol who have symptoms associated with one of the following may be included: lymphangioleiomyomas, chylous pleural effusion(s), chylous ascites, peripheral lymph-edema, chlopericardium, chyloptysis, protein-losing enteropathy, and/or chyluria. Patients will be included in this protocol if symptoms are attributed to the above processes and the patient believes the severity of symptoms warrants participation in the study. Patients with malabsorption disorders, diabetes, hypo/hyperthyroidism, or other endocrine-related disorders will be included if justified clinically based on severity of symptoms.

EXCLUSION CRITERIA:

Hypersensitivity to somatostatin, octreotide or its analogues.

Patients with hepatitis B, hepatitis C, or other clinically significant liver diseases, due to effects on hepatic function.

Transplant patients (on cyclosporine).

Pregnant women or women who are beast-feeding.

Patient or another responsible party is unable to give the subcutaneous injection.

Patient unwilling to be followed per the guidelines set forth.

Patients with decreased renal function (creatinine greater than 1.5)

Patients with HIV infection will be excluded because of effects on immune function.

Immunosuppressed patients.

Special Instructions: Currently Not Provided

Keywords:

Chylous Ascites

Chylous Pleural Effusion

Inhibitory Effects

Lymphangioleiomyoma

Somatostatin

Recruitment Keywords:

Lymphangioleiomyomatosis (LAM)

Conditions:

Lymphangioleiomyomatosis

Lymphangiomyomatosis

Investigational Drug(s):

None

Investigational Device(s):

None

Contacts:

Patient Recruitment and Public Liaison Office
Building 61
10 Cloister Court
Bethesda, Maryland 20892-4754
Toll Free: 1-800-411-1222
TTY: 301-594-9774 (local),1-866-411-1010 (toll free)
Fax: 301-480-9793

Electronic Mail:prpl@mail.cc.nih.gov

Citations:

Posttraumatic chyluria due to lymphorenal fistula regressed after somatostatin therapy

Octreotide, a new somatostatin analogue

Reduction of lymphorrhagia from ruptured thoracic duct by somatostatin

• Questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.
• Technical questions regarding the Clinical Center web site, please contact the Department of Networks and Applications, CC.

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