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Treatment With Octreotide in Patients With Lymphangioleiomyomatosis

Primary Purpose

Lymphangioleiomyomatosis, Lymphangiomyomas, Pleural Effusions

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Octreotide
Sponsored by
National Heart, Lung, and Blood Institute (NHLBI)
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Lymphangioleiomyomatosis focused on measuring Chylous Ascites, Chylous Pleural Effusion, Inhibitory Effects, Lymphangioleiomyoma, Somatostatin, Lymphangioleiomyomatosis (LAM)

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)FemaleDoes not accept healthy volunteers

INCLUSION CRITERIA: Patients enrolled in the lymphangioleiomyomatosis natural history protocol who have symptoms associated with one of the following: lymphangioleiomyomas chylous pleural effusions peripheral lymph-edema chyloptysis protein-losing enteropathy 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: Hypersensitivity to somatostatin, octreotide or its analogues Patients with hepatitis B, hepatitis C, or other clinically significant liver diseases Transplant patients 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 Immunosuppressed patients

Sites / Locations

  • National Institutes of Health Clinical Center, 9000 Rockville Pike

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Octreotide

Arm Description

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.

Outcomes

Primary Outcome Measures

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%.
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.

Secondary Outcome Measures

Number of Participants With Liver Function Abnormalities
One or more abnormality of the following liver function tests: Alkaline phosphatase above 116 i.u. SGPT above 41 i.u. SGOT from 34 i.u. Total bilirubin above 1.0 mg/dl

Full Information

First Posted
June 9, 2000
Last Updated
April 29, 2010
Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
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1. Study Identification

Unique Protocol Identification Number
NCT00005906
Brief Title
Treatment With Octreotide in Patients With Lymphangioleiomyomatosis
Official Title
Treatment With Octreotide in Patients With Lymphangioleiomyomatosis
Study Type
Interventional

2. Study Status

Record Verification Date
April 2010
Overall Recruitment Status
Completed
Study Start Date
June 2000 (undefined)
Primary Completion Date
April 2008 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
National Heart, Lung, and Blood Institute (NHLBI)

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Lymphangioleiomyomatosis, Lymphangiomyomas, Pleural Effusions, Ascites
Keywords
Chylous Ascites, Chylous Pleural Effusion, Inhibitory Effects, Lymphangioleiomyoma, Somatostatin, Lymphangioleiomyomatosis (LAM)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
4 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Octreotide
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Octreotide
Intervention Description
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.
Primary Outcome Measure Information:
Title
Number of Participants With a Reduction in Total Tumor Volume of at Least 20%.
Description
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%.
Time Frame
Six months
Title
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.
Description
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.
Time Frame
Six months
Secondary Outcome Measure Information:
Title
Number of Participants With Liver Function Abnormalities
Description
One or more abnormality of the following liver function tests: Alkaline phosphatase above 116 i.u. SGPT above 41 i.u. SGOT from 34 i.u. Total bilirubin above 1.0 mg/dl
Time Frame
Six months

10. Eligibility

Sex
Female
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
INCLUSION CRITERIA: Patients enrolled in the lymphangioleiomyomatosis natural history protocol who have symptoms associated with one of the following: lymphangioleiomyomas chylous pleural effusions peripheral lymph-edema chyloptysis protein-losing enteropathy 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: Hypersensitivity to somatostatin, octreotide or its analogues Patients with hepatitis B, hepatitis C, or other clinically significant liver diseases Transplant patients 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 Immunosuppressed patients
Facility Information:
Facility Name
National Institutes of Health Clinical Center, 9000 Rockville Pike
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
1973814
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.
Results Reference
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Treatment With Octreotide in Patients With Lymphangioleiomyomatosis

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