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Treatment With Octreotide in Patients With Lymphangioleiomyomatosis

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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)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Lymphangioleiomyomatosis
Lymphangiomyomas
Pleural Effusions
Ascites
Intervention Drug: Octreotide
Enrollment 4
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Octreotide
Hide Arm/Group Description Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
Period Title: Overall Study
Started 4
Completed 1
Not Completed 3
Reason Not Completed
Lack of Efficacy             3
Arm/Group Title Octreotide
Hide Arm/Group Description Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
Overall Number of Baseline Participants 4
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
<=18 years
0
   0.0%
Between 18 and 65 years
4
 100.0%
>=65 years
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants
43  (5.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
Female
4
 100.0%
Male
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 4 participants
4
1.Primary Outcome
Title Number of Participants With a Reduction in Total Tumor Volume of at Least 20%.
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Octreotide
Hide Arm/Group Description:
Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
Overall Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: Participants
0
2.Primary Outcome
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.
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
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
Arm/Group Title Octreotide
Hide Arm/Group Description:
Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
Overall Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: Participants
1
3.Secondary Outcome
Title Number of Participants With Liver Function Abnormalities
Hide Description

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
Time Frame Six months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Octreotide
Hide Arm/Group Description:
Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
Overall Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: Participants
1
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Octreotide
Hide Arm/Group Description Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
All-Cause Mortality
Octreotide
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Octreotide
Affected / at Risk (%) # Events
Total   1/4 (25.00%)    
Hepatobiliary disorders   
Gastrointestinal disorders [1]  1/4 (25.00%)  1
[1]
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.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Octreotide
Affected / at Risk (%) # Events
Total   1/4 (25.00%)    
Gastrointestinal disorders   
Liver function abnormalities  [1]  1/4 (25.00%)  1
Indicates events were collected by systematic assessment
[1]
Mild elevation of liver function tests.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Joel Moss
Organization: TMB/NHLBI
Phone: 301-496-1597
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 ( Other Identifier: NHLBI )
First Submitted: June 9, 2000
First Posted: June 12, 2000
Results First Submitted: February 26, 2010
Results First Posted: April 30, 2010
Last Update Posted: April 30, 2010