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| ID | Type | Description | Link |
|---|---|---|---|
| 00-H-0147 | Other Identifier | NHLBI |
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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 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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Octreotide | Experimental | 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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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Octreotide | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With a Reduction in Total Tumor Volume of at Least 20%. | 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%. | Six months |
| 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. | 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. | Six months |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Liver Function Abnormalities | One or more abnormality of the following liver function tests:
| Six months |
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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:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| National Institutes of Health Clinical Center, 9000 Rockville Pike | Bethesda | Maryland | 20892 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 1973814 | Background | Ulibarri JI, Sanz Y, Fuentes C, Mancha A, Aramendia M, Sanchez S. Reduction of lymphorrhagia from ruptured thoracic duct by somatostatin. Lancet. 1990 Jul 28;336(8709):258. doi: 10.1016/0140-6736(90)91793-a. No abstract available. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Octreotide | Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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| ID | Title | Description |
|---|---|---|
| BG000 | Octreotide | Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With a Reduction in Total Tumor Volume of at Least 20%. | 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%. | Posted | Apr 2004 | Number | Participants | Six months |
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Octreotide | Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gastrointestinal disorders | Hepatobiliary disorders | On September 2000 the patient referred right upper quadrant abdominal pain. An abdominal ultrasound showed gallbladder sludge. Octreotide was discontinued. In December, an ultrasound showed gallstones. In February 2001 she had a cholecystectomy. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Liver function abnormalities | Gastrointestinal disorders | Systematic Assessment | Mild elevation of liver function tests. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Joel Moss | TMB/NHLBI | 301-496-1597 | mossj@nhlbi.nih.gov |
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| ID | Term |
|---|---|
| D018192 | Lymphangioleiomyomatosis |
| D008203 | Lymphangiomyoma |
| D010996 | Pleural Effusion |
| D001201 | Ascites |
| D002915 | Chylous Ascites |
| D002916 | Chylothorax |
| ID | Term |
|---|---|
| D018190 | Neoplasm, Lymphatic Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D054973 | Perivascular Epithelioid Cell Neoplasms |
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| ID | Term |
|---|---|
| D015282 | Octreotide |
| ID | Term |
|---|---|
| D010456 | Peptides, Cyclic |
| D047028 | Macrocyclic Compounds |
| D011083 | Polycyclic Compounds |
| D010455 | Peptides |
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|
| Participants |
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| Age Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
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| Primary | 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. | 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. | Four patients with lymphangioleiomyomatosis and lymphangioleiomyomas and chylous effusions treated with octreotide injections by the subcutaneous route to determine whether the size of the tumors and effusions decrease with the therapy | Posted | Apr 2004 | Number | Participants | Six months |
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| Secondary | Number of Participants With Liver Function Abnormalities | One or more abnormality of the following liver function tests:
| Posted | Apr 2004 | Number | Participants | Six months |
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| 1 |
| 4 |
| 1 |
| 4 |
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| D018204 | Neoplasms, Connective and Soft Tissue |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D010995 | Pleural Diseases |
| D012140 | Respiratory Tract Diseases |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D010532 | Peritoneal Diseases |
| D004066 | Digestive System Diseases |
| D000602 |
| Amino Acids, Peptides, and Proteins |