Treatment With Octreotide in Patients With Lymphangioleiomyomatosis
|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|
- 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.
- 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:
- Alkaline phosphatase above 116 i.u.
- SGPT above 41 i.u.
- SGOT from 34 i.u.
- Total bilirubin above 1.0 mg/dl
|Study Start Date:||June 2000|
|Primary Completion Date:||April 2008 (Final data collection date for primary outcome measure)|
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.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00005906
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|