Acromegaly
Conditions
Keywords
acromegaly, pituitary, pegvisomant, somavert, lanreotide, octreotide
Brief summary
In this study the investigators will evaluate whether combination low dose somatostatin receptor ligand (SRL) and weekly or daily pegvisomant will attain equivalent control of serum insulin-like growth factor (IGF)-1 levels at a lower cost, compared to combination high dose SRL and weekly pegvisomant. Lower doses of therapy will greatly reduce cost of acromegaly therapy.
Detailed description
Subjects will be stratified based on prior response to SRL therapy and then randomized to one of three treatment arms. The study will begin when the combination of octreotide long-acting release (LAR) or lanreotide and pegvisomant is first administered. Study visits will occur every 4 weeks from the start of the study. However, for subjects who cannot make every study visit due to distance, the study team will facilitate monthly blood draws at their local Quest laboratory facility. After the 24 weeks of combination therapy, subjects with a controlled IGF-1 will then start pegvisomant monotherapy at the same dose and schedule that they attained normalization in combination therapy. IGF-1 levels, liver function test, and blood glucose will be performed monthly. Magnetic resonance imaging (MRI) will be performed at the conclusion of the study.
Interventions
Sponsors
Cedars-Sinai Medical Center
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
* Newly diagnosed patients with acromegaly who have not had surgery or medical therapy * Acromegaly patients who are at least 3 months post surgery, who are/are not receiving adjuvant medical therapy * Acromegaly patients on SRL monotherapy with normal or elevated IGF-1 levels * Acromegaly patients on Pegvisomant monotherapy with normal or elevated IGF-1 levels. * Acromegaly patients who have received SRL and dopamine agonist therapy, after a 6 week washout period of the dopamine agonist. * Acromegaly patients on combination therapy with maximum doses of SRL and Pegvisomant (daily or weekly * Normal liver function tests before randomization to treatment * The patient has had appropriate dynamic testing of the pituitary axis and, if applicable, is receiving appropriate hormone replacement therapy.
Exclusion criteria
* The patient harbors a macroadenoma with visual field defects due to chiasmatic compression * The patient has clinically significant hepatic abnormalities and/or aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) above 3 times the upper limit of normal at the baseline visit. * The patient had pituitary surgery within 3 months prior to study entry * The patient had radiotherapy within 12 months prior to study entry * The patient has abnormal CBC and chemistry panel at the baseline visit, any other medical condition(s) or laboratory findings that, in the opinion of the investigator, might jeopardize the patient's safety. * The patient has a known hypersensitivity to any of the test materials or related compounds. * The patient has a history of, or known current problems with alcohol or drug abuse. * The patient has any mental condition rendering the patient unable to understand the nature, scope, and possible consequences of the study, and/or evidence of an uncooperative attitude.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Cost Effectiveness | 24 weeks | We evaluated cost-effectiveness of combination low-dose SRL and weekly or daily pegvisomant compared to combination high-dose SRL and weekly pegvisomant by assessing cost of therapy per month in each treatment arm among patients who achieved normal IGF-1 levels. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| High Dose SRL + Weekly Pegvisomant High dose of SRL monthly
* Octreotide LAR 30mg
* Lanreotide 120mg
Weekly Pegvisomant (40-120mg/week) | 15 |
| Low Dose SRL + Daily Pegvisomant Low dose of SRL monthly
* Octreotide LAR 10mg
* Lanreotide 60mg
Daily Pegvisomant (15-60mg/day) | 14 |
| Low Dose SRL + Weekly Pegvisomant Low dose of SRL monthly
* Octreotide LAR 10mg
* Lanreotide 60mg
Weekly Pegvisomant (40-120mg/week) | 23 |
| Total | 52 |
Baseline characteristics
| Characteristic | High Dose SRL + Weekly Pegvisomant | Low Dose SRL + Daily Pegvisomant | Low Dose SRL + Weekly Pegvisomant | Total |
|---|---|---|---|---|
| Age, Continuous | 48.6 Years STANDARD_DEVIATION 17.6 | 52.9 Years STANDARD_DEVIATION 12.1 | 48.0 Years STANDARD_DEVIATION 13.3 | 49.5 Years STANDARD_DEVIATION 14.3 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 1 Participants | 4 Participants | 5 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 14 Participants | 13 Participants | 18 Participants | 45 Participants |
| Sex: Female, Male Female | 10 Participants | 8 Participants | 14 Participants | 32 Participants |
| Sex: Female, Male Male | 5 Participants | 6 Participants | 9 Participants | 20 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 15 | 0 / 14 | 0 / 23 |
| other Total, other adverse events | 4 / 15 | 2 / 14 | 9 / 23 |
| serious Total, serious adverse events | 0 / 15 | 0 / 14 | 1 / 23 |
Outcome results
Primary
Cost Effectiveness
We evaluated cost-effectiveness of combination low-dose SRL and weekly or daily pegvisomant compared to combination high-dose SRL and weekly pegvisomant by assessing cost of therapy per month in each treatment arm among patients who achieved normal IGF-1 levels.
Time frame: 24 weeks
| Arm | Measure | Value (MEAN) |
|---|---|---|
| High Dose SRL + Weekly Pegvisomant | Cost Effectiveness | 14,261.33 US dollars/month |
| Low Dose SRL + Daily Pegvisomant | Cost Effectiveness | 22,542.86 US dollars/month |
| Low Dose SRL + Weekly Pegvisomant | Cost Effectiveness | 9,836.52 US dollars/month |