Trial record 1 of 1 for:    "Chylous ascites"
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Treatment With Octreotide in Patients With Lymphangioleiomyomatosis

This study has been completed.
Sponsor:
Information provided by:
National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:
NCT00005906
First received: June 9, 2000
Last updated: April 29, 2010
Last verified: April 2010
  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 2

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

Resource links provided by NLM:


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

Primary Outcome Measures:
  • Number of Participants With a Reduction in Total Tumor Volume of at Least 20%. [ Time Frame: Six months ] [ Designated as safety issue: No ]
    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%.

  • 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. [ Time Frame: Six months ] [ Designated as safety issue: No ]

    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.



Secondary Outcome Measures:
  • Number of Participants With Liver Function Abnormalities [ Time Frame: Six months ] [ Designated as safety issue: Yes ]

    One or more abnormality of the following liver function tests:

    1. Alkaline phosphatase above 116 i.u.
    2. SGPT above 41 i.u.
    3. SGOT from 34 i.u.
    4. Total bilirubin above 1.0 mg/dl


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.
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.

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

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Female
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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:
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

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

Additional relevant MeSH terms:
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
Gastrointestinal Agents
Therapeutic Uses
Pharmacologic Actions
Antineoplastic Agents, Hormonal
Antineoplastic Agents

ClinicalTrials.gov processed this record on August 28, 2014