Clinical trial NCT00005906: Treatment With Octreotide in Patients With Lymphangioleiomyomatosis
Official title: Treatment With Octreotide in Patients With Lymphangioleiomyomatosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Lymphangioleiomyomatosis
Lymphangiomyomas
Pleural Effusions
Ascites
Keywords: Chylous Ascites
Chylous Pleural Effusion
Inhibitory Effects
Lymphangioleiomyoma
Somatostatin
Lymphangioleiomyomatosis (LAM)
Lead sponsor: National Institutes of Health Clinical Center (CC)
Sponsor class: NIH
First posted: 2000-06-12
Enrollment: 4
Sex: FEMALE
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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 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.

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. 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 criteria: * INCLUSION CRITERIA:

Patients enrolled in the lymphangioleiomyomatosis natural history protocol who have symptoms associated with one of the following:

1. lymphangioleiomyomas
2. chylous pleural effusions
3. peripheral lymph-edema
4. chyloptysis
5. protein-losing enteropathy
6. chyluria

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:

1. Hypersensitivity to somatostatin, octreotide or its analogues
2. Patients with hepatitis B, hepatitis C, or other clinically significant liver diseases
3. Transplant patients
4. Pregnant women or women who are beast-feeding
5. Patient or another responsible party is unable to give the subcutaneous injection
6. Patient unwilling to be followed per the guidelines set forth
7. Patients with decreased renal function (creatinine greater than 1.5)
8. Patients with HIV infection
9. Immunosuppressed patients

Primary outcomes:
measure: Number of Participants With a Reduction in Total Tumor Volume of at Least 20%.
timeFrame: Six months
measure: 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.
timeFrame: Six months
Secondary outcomes:
measure: Number of Participants With Liver Function Abnormalities
timeFrame: Six months
Other outcomes:
Locations:
city: Bethesda
country: United States
facility: National Institutes of Health Clinical Center, 9000 Rockville Pike
state: Maryland
Study officials:
References:
citation: 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.