Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Treatment With Octreotide in Patients With Lymphangioleiomyomatosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00005906
Recruitment Status : Completed
First Posted : June 12, 2000
Results First Posted : April 30, 2010
Last Update Posted : April 30, 2010
Sponsor:
Information provided by:
National Institutes of Health Clinical Center (CC)

Tracking Information
First Submitted Date  ICMJE June 9, 2000
First Posted Date  ICMJE June 12, 2000
Results First Submitted Date  ICMJE February 26, 2010
Results First Posted Date  ICMJE April 30, 2010
Last Update Posted Date April 30, 2010
Study Start Date  ICMJE June 2000
Actual Primary Completion Date April 2008   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 29, 2010)
  • Number of Participants With a Reduction in Total Tumor Volume of at Least 20%. [ Time Frame: Six months ]
    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 ]
    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.
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00005906 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 29, 2010)
Number of Participants With Liver Function Abnormalities [ Time Frame: Six months ]
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
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Treatment With Octreotide in Patients With Lymphangioleiomyomatosis
Official Title  ICMJE Treatment With Octreotide in Patients With Lymphangioleiomyomatosis
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Lymphangioleiomyomatosis
  • Lymphangiomyomas
  • Pleural Effusions
  • Ascites
Intervention  ICMJE 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.
Study Arms  ICMJE 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.
Intervention: Drug: Octreotide
Publications * Ulíbarri JI, Sanz Y, Fuentes C, Mancha A, Aramendia M, Sánchez S. Reduction of lymphorrhagia from ruptured thoracic duct by somatostatin. Lancet. 1990 Jul 28;336(8709):258.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 29, 2010)
4
Original Enrollment  ICMJE
 (submitted: June 23, 2005)
14
Study Completion Date  ICMJE Not Provided
Actual Primary Completion Date April 2008   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: Female
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00005906
Other Study ID Numbers  ICMJE 000147
00-H-0147 ( Other Identifier: NHLBI )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Joel Moss, M.D./National Heart, Lung and Blood Institute, National Institutes of Health
Study Sponsor  ICMJE National Heart, Lung, and Blood Institute (NHLBI)
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account National Institutes of Health Clinical Center (CC)
Verification Date April 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP