Treatment With Octreotide in Patients With Lymphangioleiomyomatosis

NCT00005906 | Completed Phase 2 Results DMC

• 2 other identifiers: 00014700-H-0147
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

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.

Conditions

Lymphangioleiomyomatosis; Lymphangiomyomas; Pleural Effusions; Ascites

Keywords

Chylous Ascites; Chylous Pleural Effusion; Inhibitory Effects; Lymphangioleiomyoma; Somatostatin; Lymphangioleiomyomatosis (LAM)

Outcome Measures

Primary Outcomes (2)

• 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%.

  Six months

• 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.

  Six months

Secondary Outcomes (1)

• Number of Participants With Liver Function Abnormalities

  Six months

Study Arms (1)

Octreotide

EXPERIMENTAL

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

Interventions

Octreotide DRUG

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.

Octreotide

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI): lead

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (1)

• 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.

  PMID: 1973814 BACKGROUND

MeSH Terms

Conditions

Lymphangioleiomyomatosis; Lymphangiomyoma; Pleural Effusion; Ascites; Chylous Ascites; Chylothorax

Interventions

Octreotide

Condition Hierarchy (Ancestors)

Neoplasm, Lymphatic Tissue; Neoplasms by Histologic Type; Neoplasms; Perivascular Epithelioid Cell Neoplasms; Neoplasms, Connective and Soft Tissue; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Pleural Diseases; Respiratory Tract Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Peritoneal Diseases; Digestive System Diseases

Intervention Hierarchy (Ancestors)

Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Joel Moss
• Organization: TMB/NHLBI

mossj@nhlbi.nih.gov

301-496-1597

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH

Study Record Dates

• First Submitted: June 9, 2000
• First Posted: June 12, 2000
• Study Start: June 1, 2000
• Primary Completion: April 1, 2008
• Last Updated: April 30, 2010
• Results First Posted: April 30, 2010

Record last verified: 2010-04

Locations

US (1)