Acromegaly
Conditions
Keywords
Acromegaly
Brief summary
Acromegaly is a disease of the pituitary gland that involves overproduction of growth hormone. Pegvisomant works by blocking binding of GH to receptors found in tissues throughout the body. Human studies have evaluated pegvisomant action by measuring reduction of IGF-I levels in the blood. However, no studies have evaluated the effects of blocking GH receptors in tissues. In this study, we will study tissue biomarkers for pegvisomant action in GH and IGF-I dependent signaling pathways in colon tissue of patients with acromegaly treated with pegvisomant.
Interventions
DRUGPegvisomant
Pegvisomant used as indicated
Sponsors
Cedars-Sinai Medical Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosis of acromegaly established on the basis of symptoms and signs at presentation, evidence of a pituitary adenoma on MRI, elevated serum concentrations of IGF1 (\>1.3 X ULN), and inadequate GH suppression (\>0.4 ng/mL) following OGTT * Candidates to receive pegvisomant therapy following pituitary adenoma surgery, or intolerant of other medical treatments or had not undergone previous therapy * Normal LFTs before treatment * Dynamic testing of the pituitary axis and, if applicable, appropriate hormone replacement
Exclusion criteria
* Treatment with a long-acting SRL within 12 weeks before enrollment * Presence of a macroadenoma with visual field defects as a result of chiasmatic compression * Clinically significant hepatic abnormalities and/or AST or ALT \>3 X ULN on screening * Known hypersensitivity to any of the test materials or related compounds * History of, or known current, problems with alcohol or drug abuse * 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 |
|---|---|---|
| Effect of Pegvisomant on Colon Tissue p53 Expression | 8 weeks | Induction of colon tissue expression of p53, a tumor suppressor, using Western blot analysis, after GH receptor blockade with pegvisomant |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Pegvisomant Pegvisomant: Pegvisomant used as indicated | 7 |
| Total | 7 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Screen failure | 1 |
Baseline characteristics
| Characteristic | Pegvisomant |
|---|---|
| Age, Continuous | 50 years STANDARD_DEVIATION 21 |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 8 |
| other Total, other adverse events | 0 / 8 |
| serious Total, serious adverse events | 1 / 8 |
Outcome results
Primary
Effect of Pegvisomant on Colon Tissue p53 Expression
Induction of colon tissue expression of p53, a tumor suppressor, using Western blot analysis, after GH receptor blockade with pegvisomant
Time frame: 8 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Pegvisomant | Effect of Pegvisomant on Colon Tissue p53 Expression | 330.57 ng/mL | Standard Deviation 107.33 |