Trial Outcomes & Findings for Treatment With Octreotide in Patients With Lymphangioleiomyomatosis (NCT NCT00005906)

Results Overview

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%.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

Six months

Results posted on

2010-04-30

Participant Flow

Participant milestones

Participant milestones
Measure	Octreotide Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
Overall Study STARTED	4
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Octreotide Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
Overall Study Lack of Efficacy	3

Baseline Characteristics

Treatment With Octreotide in Patients With Lymphangioleiomyomatosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Octreotide n=4 Participants Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age Continuous	43 years STANDARD_DEVIATION 5.5 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants

PRIMARY outcome

Timeframe: 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%.

Outcome measures

Outcome measures
Measure	Octreotide n=4 Participants Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
Number of Participants With a Reduction in Total Tumor Volume of at Least 20%.	0 Participants 5

PRIMARY outcome

Timeframe: Six months

Population: 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

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.

Outcome measures

Outcome measures
Measure	Octreotide n=4 Participants Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
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.	1 Participants 5

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	Octreotide n=4 Participants Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
Number of Participants With Liver Function Abnormalities	1 Participants

Adverse Events

Octreotide

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Octreotide n=4 participants at risk Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
Hepatobiliary disorders Gastrointestinal disorders	25.0% 1/4 • Number of events 1

Other adverse events

Other adverse events
Measure	Octreotide n=4 participants at risk Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain
Gastrointestinal disorders Liver function abnormalities	25.0% 1/4 • Number of events 1

Additional Information

Joel Moss

TMB/NHLBI

Phone: 301-496-1597

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place