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Treatment With Octreotide in Patients With Lymphangioleiomyomatosis
This study has been completed.
First Received: June 9, 2000   Last Updated: April 22, 2008   History of Changes
Sponsored by: National Heart, Lung, and Blood Institute (NHLBI)
Information provided by: National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT00005906
  Purpose

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.


Condition Intervention Phase
Lymphangioleiomyomatosis
Lymphangiomyomatosis
Drug: Octreotide
Phase II

MedlinePlus related topics: Lymphedema
Drug Information available for: Octreotide acetate Octreotide
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Open Label, Single Group Assignment, Efficacy Study
Official Title: Treatment With Octreotide in Patients With Lymphangioleiomyomatosis

Further study details as provided by National Institutes of Health Clinical Center (CC):

Estimated Enrollment: 14
Study Start Date: June 2000
Estimated Study Completion Date: April 2008
Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Female
Accepts Healthy Volunteers:   No
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.

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00005906

Locations
United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
  More Information

Publications:
Study ID Numbers: 000147, 00-H-0147
Study First Received: June 9, 2000
Last Updated: April 22, 2008
ClinicalTrials.gov Identifier: NCT00005906     History of Changes
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Chylous Ascites
Chylous Pleural Effusion
Inhibitory Effects
Lymphangioleiomyoma
Somatostatin
Lymphangioleiomyomatosis (LAM)

Study placed in the following topic categories:
Lymphatic Diseases
Pleural Effusion
Lymphangioleiomyomatosis
Immunoproliferative Disorders
Antineoplastic Agents, Hormonal
Ascites
Octreotide
Lymphangiomyoma
Lymphoproliferative Disorders
Somatostatin

Additional relevant MeSH terms:
Neoplasms by Histologic Type
Immunoproliferative Disorders
Immune System Diseases
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Gastrointestinal Agents
Lymphangiomyoma
Octreotide
Pharmacologic Actions
Lymphatic Vessel Tumors
Lymphangioleiomyomatosis
Lymphatic Diseases
Neoplasms
Therapeutic Uses
Lymphoproliferative Disorders

ClinicalTrials.gov processed this record on May 07, 2009