Trial · Interventional

CompletedPhase 2Results postedRegistry data

Treatment With Octreotide in Patients With Lymphangioleiomyomatosis

NCTNCT00005906Other id000147Other id00-H-0147

Open at ClinicalTrials.govAsk about this trial

Overview

Status: Completed
Phase: Phase 2
Enrollment: 4 participants (actual)
Sponsor: National Heart, Lung, and Blood Institute (NHLBI) · Nih
Design: Allocation not applicable · Open label (no masking)
Countries: 1 country
United States
Start: Jun 2000
Primary completion: Apr 2008
Completion: Not stated
First posted: 12 Jun 2000 (estimated)
Last update posted: 30 Apr 2010 (estimated)
Conditions: Lymphangioleiomyomatosis · Lymphangiomyomas · Pleural Effusions · Ascites

Timeline

1 trial · 1 completed · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

20002002200420062008

Ph 2
Treatment With Octreotide in Patients With Lymphangioleiomyomatosis
NCT00005906
Jun 2000 to Apr 2008 (primary)

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

OctreotideDrug

Arms1

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.

Primary outcomes2

Outcomes the sponsor listed as primary.

Number of Participants With a Reduction in Total Tumor Volume of at Least 20%.
Time frame · Six months
Registry 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%.
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
Registry 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.

Secondary outcomes(1)

Number of Participants With Liver Function Abnormalities
Time frame · Six months
Registry 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

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeNumber· Six months

Number of Participants With a Reduction in Total Tumor Volume of at Least 20%.

Group	Value (Participants)	Spread
Octreotide	0	5

Primary outcomeNumber· 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.

Group	Value (Participants)	Spread
Octreotide	1	5

Secondary and other outcome results(1)

Secondary outcomeNumber· Six months

Number of Participants With Liver Function Abnormalities

Group	Value (Participants)
Octreotide	1

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
Group	Deaths	Serious events	Other events
OctreotidePatients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain	–	1 / 4	1 / 4

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex: Female
Age: from 18 Years up to 65 Years
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

* 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

PepBank is a research reference. Talk to a doctor before you use any peptide.