Clinical trials are useful not only for initially establishing the efficacy of treatments for rare diseases, but for demonstrating that these results are durable as well. The MPOWER trial, which investigates oral octreotide capsules for adult acromegaly patients, is the perfect example. This study has been evaluating the long-term effects of this drug, and has now compiled up to three years’ worth of data.

The highlight of the data is the maintenance of efficacy – over 90% of participants continued to show a biochemical response to oral octreotide after three years of treatment. A response can be defined as an insulin-like growth factor I that is under 1.3x the upper limit of normal. This high percentage joins other positive response rates. At one year, 94% of patients demonstrated a response, 90% of patients responded at year two, and by year three, 93% of participants were responding. Other results include:

• Of the 19 patients originally treated with injectable somatostatin receptor ligands in the phase 3 trial, there was a 67% increase in the number of patients who described their symptom control as very good or excellent
  • Similar improvements in treatment convenience and satisfaction were also observed
• Consistent safety profile
• No new adverse events (AEs)
• Consistent and durable effect of oral octreotide

Want to find out more? Healio released an article about the results, which you can find here.

What is Acromegaly?

Now that we’ve learned more about a possible treatment for this condition, let’s learn more about the disease itself. Acromegaly is a rare disease that is characterized by an excess of growth hormone. This causes abnormal growth, meaning symptoms like:

• Growth in the hands and feet, making them disproportionate to the rest of the body
• Protrusion of the lower jaw and brow
• Enlarged nasal bone
• Enlarged facial features
  • This includes the tongue, nose, lips, and vocal cords
• Thick, oily, and coarse skin
  • Patients are also prone to sweating
• Enlarged organs within the body

Heightened levels of GH cause these symptoms, and this hormone is released by benign tumors – called adenomas – that form on the pituitary gland. Because of this, surgery to remove the tumors is often the first option for patients. If this is not an option or is unsuccessful, doctors may utilize GH receptor antagonists (GHRAs), somatostatin analogs (SSAs), and dopamine agonists.