Clinical trials
A ‘clinical trial’ is a research study in which a potential new treatment is investigated to prevent, cure or improve a disease or medical condition. A clinical trial also looks at how participants react to the potential new treatment and if any unwanted effects occur. This helps to determine if the new investigational treatment works, is safe, and is better than those that are already available. Many clinical trials also compare existing treatments or test new ways to use or combine with existing treatments.
All new drugs must be tested in clinical trials before they can be prescribed to patients. Without people taking part in these research studies, we would have no new drugs to help others with their condition.
The Oxtend-01 clinical trial of Debio 4126 for patients with acromegaly or functioning Gastroenteropancreatic Neuroendocrine Tumors (GEP-NETs)
The OxtendTM-01 (Debio4126-102) clinical trial was a Phase 1b, open-label multicenter trial conducted to test a new drug, Debio4126, in patients with acromegaly or gastroenteropancreatic neuroendocrine tumors (GEP-NET Definition Gastroenteropancreatic Neuroendocrine Tumors (GEP-NETs) are a group of rare cancers that develop from specialized cells in the digestive system and pancreas. These tumors can sometimes produce excess hormones, leading to a variety of symptoms like diarrhea, flushing, and abdominal pain. The exact cause is often unknown, though some are linked to genetic syndromes. Treatment options vary based on the tumor’s location, size, and whether it has spread, and can include surgery to remove the tumor, chemotherapy, targeted therapies, and sometimes medications to control hormone-related symptoms. While these tumors are considered slow growing, the outlook depends heavily on the stage at which they are diagnosed. Mortality rates have been decreasing due to improved treatments and early detection. s).
The trial aimed to understand how Debio4126 behaves in the body, how it affects the body, how well it works, and how safe and tolerable it is for patients.
Debio4126 is an innovative injectable extended-release octreotide formulation. Compared to the existing treatments typically administered every 4 weeks, Debio 4126 is injected less often (every 3 months). This is because Debio 4126 is released in the body over a longer period of time. Therefore, patients with acromegaly or GEP-NET Definition Gastroenteropancreatic Neuroendocrine Tumors (GEP-NETs) are a group of rare cancers that develop from specialized cells in the digestive system and pancreas. These tumors can sometimes produce excess hormones, leading to a variety of symptoms like diarrhea, flushing, and abdominal pain. The exact cause is often unknown, though some are linked to genetic syndromes. Treatment options vary based on the tumor’s location, size, and whether it has spread, and can include surgery to remove the tumor, chemotherapy, targeted therapies, and sometimes medications to control hormone-related symptoms. While these tumors are considered slow growing, the outlook depends heavily on the stage at which they are diagnosed. Mortality rates have been decreasing due to improved treatments and early detection. s need fewer injections over their lifetime, while Debio 4126 is expected to work just as well and be just as safe as other somatostatin analogs.
Three different dose levels of Debio4126 were tested in the trial.
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Phases
When a potential new medication is being developed, it is first tested in a laboratory setting. If the results are positive, the drug may enter a clinical trial program. This means that it will be tested in humans in several ‘phases’ of study.
Phase I = Safety evaluation. The very first administration in humans, typically carried out in a small group of healthy volunteers to assess if the drug is safe.
Phase II = Efficacy evaluation. The first trials in patients with the intended disease to check if the drug works efficiently and if there are any unwanted side effects.
Phase III = Confirming findings. Trials in large numbers of patients that generally compare the drug to the best treatments available.
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