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Clinical Development

Rare Disease Spotlight: Acromegaly

  • July 30, 2020

What is Acromegaly?

Acromegaly is a rare disease characterized by overproduction of growth hormone (GH), and affects approximately 40 – 60 patients per million population. In adults, a benign pituitary tumor is the most common cause of excess GH production. The signs and symptoms of the disease include enlarged bones in the face, feet, and hands, enlarged tongue, weight gain and arthritis. Acromegaly is associated with hypertension, diabetes and increased cardiovascular risk.


First generation Somatostatin Analogues (SSAs) have been used for many years to treat acromegaly, and are effective in a majority of patients in reducing GH and insulin-like growth factor-1 (IGF-1) levels. Newer ‘second generation’ SSAs have been approved more recently, and offer a potentially better efficacy profile. Pharmaceutical companies are currently evaluating newer SSAs in clinical studies – which may offer more convenient dosing eg, oral formulations. A significant unmet clinical need in acromegaly is new treatment options which can improve cardiovascular risk, or other acromegaly-related comorbidities.

Ideal Partner for Acromegaly Studies

Acromegaly is a disease which affects all regions in the world including North America, Europe, Asia Pacific and South America. Medpace is a global full-service CRO with deep experience in Endocrinology and Metabolic diseases, including acromegaly.

The ability to recruit acromegaly study participants requires a comprehensive site feasibility assessment, a well-designed study, and established relationships with key opinion leaders and principal investigators. Acromegaly is considered a rare or orphan disease, and the successful conduct of clinical development studies in this field requires strong collaboration with KOLs, established relationships with site investigators, and expert study design and execution.

Medpace Labs

Medpace’s central laboratories have global reach and capabilities to conduct acromegaly studies with locations in the US, Europe, China, and Singapore.   Medpace has supported acromegaly studies with safety testing as well as biomarker testing that includes the following:  GH, IGF-1, Thyroid testing, HbA1c, glucose, insulin, c-peptide, proinsulin, hs-CRP, TNFalpha, Vitamin B12, ACTH, cortisol, and prolactin.   Medpace’s central laboratories are led by a team of PhD scientists that are readily accessible to our clients to discuss other biomarkers that can be useful in acromegaly studies.   Medpace is continually validating new biomarkers to meet our client’s needs.

Medpace Imaging Core Lab provides holistic central imaging services including site assessment, qualification and training, recording equipment, provisioning, image processing (blinding and quality control) and expert evaluation. In particular, Medpace has expertise with Magnetic Resonance Imaging (MRI) to support brain imaging if necessary.

Why choose Medpace for an Acromegaly Study?

  • Scientifically-driven approach takes advantage of our deep clinical trial experience and scientific expertise in the endocrinology and metabolic disease, including acromegaly studies
  • Full-service capabilities inclusive of Central Lab and Core Imaging Lab services
  • Global phase II & III study experience including two recent global Phase III studies, involving >200 acromegaly patients
  • Central lab provider for two additional Phase III acromegaly studies
  • Existing investigative site relationships with local KOLs who specialize in acromegaly patient recruitment
  • Strong global operational experience in running acromegaly studies

Contact us for more information about acromegaly or other rare disease trials.