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| Sponsor: | 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 effusions and lymphangioleiomyomas. Treatment of many of these symptoms has been ineffective. Previous studies with somatostatin and octreotide in other clinical settings have shown reduction in chylous effusions. This study assesses the effectiveness of octreotide in symptomatic patients with LAM, lymphangioleiomyomas and/or chylous effusions/ascites, peripheral lymphedema and chyluria.
| Condition | Intervention | Phase |
|---|---|---|
|
Lymphangioleiomyomatosis Lymphangiomyomas Pleural Effusions Ascites |
Drug: Octreotide |
Phase II |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Treatment With Octreotide in Patients With Lymphangioleiomyomatosis |
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.
One or more abnormality of the following liver function tests:
| Enrollment: | 4 |
| Study Start Date: | June 2000 |
| Primary Completion Date: | April 2008 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Octreotide
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.
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Drug: Octreotide
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.
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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. The dose of octreotide starts at 50 micrograms (ug) by the subcutaneous route twice a day. After two weeks the dose will be increased to 200 ug per day and two weeks later to 400 ug/day. Maximal dose is 400 ug twice a day.
Eligibility| Ages Eligible for Study: | 18 Years to 65 Years |
| Genders Eligible for Study: | Female |
| Accepts Healthy Volunteers: | No |
Patients enrolled in the lymphangioleiomyomatosis natural history protocol who have symptoms associated with one of the following:
Patients will be included in this protocol if symptoms are attributed to the above processes. 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:
Contacts and Locations
More Information
| Responsible Party: | Joel Moss, M.D./National Heart, Lung and Blood Institute, National Institutes of Health |
| ClinicalTrials.gov Identifier: | NCT00005906 History of Changes |
| Other Study ID Numbers: | 000147, 00-H-0147 |
| Study First Received: | June 9, 2000 |
| Results First Received: | February 26, 2010 |
| Last Updated: | April 29, 2010 |
| Health Authority: | United States: Federal Government |
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Chylous Ascites Chylous Pleural Effusion Inhibitory Effects |
Lymphangioleiomyoma Somatostatin Lymphangioleiomyomatosis (LAM) |
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Ascites Lymphangiomyoma Pleural Effusion Lymphangioleiomyomatosis Pathologic Processes Lymphatic Vessel Tumors Neoplasms by Histologic Type Neoplasms Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders |
Immune System Diseases Pleural Diseases Respiratory Tract Diseases Perivascular Epithelioid Cell Neoplasms Neoplasms, Connective and Soft Tissue Octreotide Antineoplastic Agents, Hormonal Antineoplastic Agents Therapeutic Uses Pharmacologic Actions Gastrointestinal Agents |