Acromegaly
Conditions
Brief summary
Acromegaly is a rare, chronic, and debilitating disease, usually caused by a benign tumor on the pituitary gland, which leads to excessive production of growth hormone (GH). GH excess in turn causes overproduction of another hormone called insulin-like growth factor-1 (IGF-1). IGF-1 levels are currently the most widely accepted measure of disease activity. In Canada, medical therapy with a type of medicine called somatostatin analogues (SSA), such as octreotide and lanreotide, is recommended for treatment of acromegaly. However, studies have shown that a significant number of patients who take SSA medications alone remain with elevated levels of IGF-1 in their blood. Another medication that is used to treat acromegaly is pegvisomant (PEGV), and the investigators plan to study whether strict control of IGF-1, by adding or optimizing the use of PEGV, results in a significant health benefits to patients who still have modestly high levels of IGF-1 in their blood.
Interventions
DRUGPegvisomant
Study medications will be prescribed as per clinical practice with PEGV being added, or optimally dosed, at the Month 0 visit. Subjects who are naïve to PEGV should start their injections from 10 mg twice a week to 10 mg daily if used as combination therapy or 10 to 20 mg daily if used as monotherapy. Maximum dosing should not exceed 40mg/day. Dosing of PEGV can be adjusted as per clinical judgement to meet the normalization of IGF-1 levels (\<1.0 ULN) in increments of 5-10mg/day. In the event of a reduction in IGF-1 below the LLN, the dose of PEGV could be decreased by 5-10 mg/day.
Sponsors
Unity Health Toronto
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adult patients (at least 18 years old) with confirmed acromegaly whose IGF-I levels are persistently but modestly (1.0 ULN \< \[IGF-1 serum level\] \< 1.5 ULN) elevated following medical therapy such as SSA, PEGV, cabergoline alone or in combination.
Exclusion criteria
1. Progressive or recent visual field loss or optic chiasmal compression, or pituitary tumors within 2mm from the chiasm. Patients whose visual field loss, optic chiasmal compression or pituitary tumor has been stable for at least a year will be eligible. 2. Cranial nerve palsies or intracranial hypertension requiring tumour decompression surgery 3. Clinically significant hepatic disease and/or elevated liver enzymes (ALT, AST \> 3 x ULN) 4. Patients who have received pituitary surgery within one year prior to screening visit 5. Patients who have received radiation therapy within one year prior to screening visit 6. History of hypersensitivity to any components of Pegvisomant 7. Inability to fully comprehend the nature of the study or cooperate with study procedures 8. Pregnant / lactating women and subjects refusing to use adequate contraception to prevent pregnancy during the study. 9. Subjects unwilling or unable to self-administer medication on a daily basis 10. known or suspected alcohol / drug abuse 11. Severe acute or chronic medical or psychiatric condition or laboratory abnormality that could increase the risk associated with trial participation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Health Related Quality of Life (AcroQoL) | six months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Serum IGF-1 level | six months | — |
| Acromegaly Disease Activity Tool (ACRODAT) | six months | To assess acromegaly co-morbidities |
| Patient-assessed Acromegaly Symptom Questionnaire | six months | To assess acromegaly signs and symptoms |
| Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment | six months | — |
Countries
Canada
Outcome results
None listed