Clinical trial NCT01538966: Acromegaly Combination Treatment Study
Official title: Comparison of Combination Low-Dose SRL + Daily Pegvisomant Therapy, Low-Dose SRL + Weekly Pegvisomant Therapy, and High-Dose SRL + Weekly Pegvisomant Therapy
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Acromegaly
Keywords: acromegaly
pituitary
pegvisomant
somavert
lanreotide
octreotide
Lead sponsor: Cedars-Sinai Medical Center
Sponsor class: OTHER
First posted: 2012-02-27
Enrollment: 76
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Cost Effectiveness
timeFrame: 24 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
Study officials:
affiliation: Cedars-Sinai Medical Center
name: Shlomo Melmed, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Bonert V, Mirocha J, Carmichael J, Yuen KCJ, Araki T, Melmed S. Cost-Effectiveness and Efficacy of a Novel Combination Regimen in Acromegaly: A Prospective, Randomized Trial. J Clin Endocrinol Metab. 2020 Sep 1;105(9):dgaa444. doi: 10.1210/clinem/dgaa444.