Clinical trials are essential to the development and advancement of medicine and healthcare. They are research studies conducted in humans to test if new medical treatments work, how well they work in comparison with existing treatments and at what dosages, and what are their potential side effects. One key goal of clinical trials is to document whether benefits outweigh the risks. In other words, researchers want to make sure the new medicine or treatment is more effective than it may cause harm.

During a clinical trial, patients who participate are carefully selected to take part. Some of them receive the new medicine or treatment, while others might receive one of the current standards of care or a placebo (a substance that has no therapeutic effect, used as a control in testing new drugs). Researchers compare the results from both groups to see if the experimental treatment being tested is better than the current standard of care or if it has any unwanted effects.

Clinical trials are conducted in four phases, each phase focusing on a different aspect of the treatment being studied:

• Phase I clinical trials are typically small-scale studies to test the safety of the treatment in a small group of patients. If the treatment is found to be safe, it is then investigated in phase II trials.
• Phase II clinical trials involve a medium size group of patients to determine the effectiveness of the new treatment and to further study its safety. Pending positive results, the new drug or treatment is studied in phase III trials.
• Phase III clinical trials recruit larger group of patients to confirm a new treatment’s effectiveness and monitor side effects.
• Phase IV clinical trials refer to research studies conducted after the marketing authorisation has been granted. Their objective is to track the effectiveness, optimal use, safety, and quality of life in the patient population of interest.

Clinical trials follow strict rules and guidelines to ensure the safety and well-being of the participants. The studies are carefully designed and monitored to collect accurate and reliable information. Ethics committees and regulatory authorities closely oversee clinical trials to make sure they are conducted ethically and with the participants’ best interests in mind.

For patients, clinical trials offer the opportunity to access cutting-edge medical treatments, medical care and monitoring that may not yet be provided by their regular doctor. Patient participation in clinical trials also plays a critical role in advancing medical science by helping researchers to determine whether a new treatment is safe and effective. Overall, clinical trials are an essential component of modern healthcare and offer important benefits for both patients and the medical community.

If you are trying to find a clinical trial to enter, start by asking your hematologist/doctor whether they know about a clinical trial that could be a good option for you. He or she may also be able to provide information and help you decide about joining a clinical trial.

If your hematologist/doctor is not be aware of clinical trials that could be appropriate for you, you may want to consult another hematologist and get a second opinion about your treatment options, including taking part in a clinical trial.

If you decide to look for trials on your own, there are many international and national trial registries, platforms, and search engines. You will find below the three most used registries worldwide:

• International Clinical Trials Registry Platform (ICTRP): https://trialsearch.who.int/
• US National Library of Medicine ClinicalTrials.gov: https://www.clinicaltrials.gov/
• EU Clinical Trial Register: https://www.clinicaltrialsregister.eu/

We also recommend you use Ancora.ai, which is a user-friendly search engine to helps you find your personalized trial options: https://www.ancora.ai/ .

Please do not hesitate to contact us should you need additional support in identifying a suitable trial to treat acute leukemia.