Clinical trials are an important part of clinical research and at the heart of all medical advances. Without them, we would not have any of the approved treatments or medicines that help enhance, extend, and in some cases, save lives.
At their core, clinical trials look at new ways to prevent, detect, or treat disease. Those treatments might be new drugs or new combinations of drugs, new surgical procedures or devices, or new ways to use existing treatments. Clinical trials can also look at other aspects of care, such as improving the quality of life for people with chronic illnesses, like Alpha-1 Antitrypsin Deficiency (Alpha-1). Ultimately, the goal of clinical trials is to determine if a new test or treatment works and is safe.
Patients participate in clinical trials for a variety of reasons. Healthy volunteers say they participate to help others and to contribute to moving science forward. Participants with an illness or disease participate to help others, but also to possibly receive the newest treatment and to have the additional care and attention from the clinical trial staff.
In the rare disease community, it is often clinical trials that offer the most hope to find better treatments for others in the future. In the Alpha-1 world, there is hope on the horizon. Over the next few months and years, there will be an influx of clinical trials for both liver and lung affected patients to learn about and consider enrolling in. Here’s what you need to know to help you navigate the clinical trial world and determine if you want to enroll and participate, and what to expect once you do.
What is a clinical trial?
A clinical trial is a research study in which one or more human subjects are prospectively assigned to one or more interventions to evaluate the effects on health-related biomedical or behavioral outcomes. When you volunteer to take part in clinical research, you help physicians and researchers learn more about Alpha-1 and improve healthcare for Alphas in the future.
A clinical trial will always start with an informed consent so that you can ask the study team any questions you may have regarding participating in the clinical trial.
What is an informed consent?
The informed consent is a document that is presented to you explaining the potential risks and benefits associated with the clinical trial. Once you agree to take part in the clinical trial, the informed consent will be signed by both you and a member of the study team (physician, nurse practitioner, research nurse, or clinical research coordinator). A copy of the informed consent will be provided for your records. If you ever want to, you can withdraw your consent at any point.
How do I know if I am eligible to enroll in a trial?
Eligibility criteria are the main reasons why a person can or cannot participate in a clinical study. These requirements are created by the clinical trial sponsor (often in consult with medical experts and regulatory agencies) for safety purposes to protect participants from harm. Some examples of inclusion criteria include age and established diagnosis of Alpha-1.
Some exclusion criteria examples are clinically significant health concerns other than Alpha-1, and lung or liver transplant. You can search for current clinical trials by visiting the Alpha-1 Clinical Trials Search Engine: https://www.alpha1.org/alphas-friends-family/resources/ct/
What role does the Alpha-1 Research Registry play in the clinical trial recruitment process?
The Alpha-1 Research Registry is a confidential database comprised of individuals diagnosed with Alpha-1 and individuals identified as Alpha-1 carriers. The Registry facilitates research initiatives and promotes the development of improved treatments and a cure for Alpha-1. Individuals enrolled in the Registry have the ongoing opportunity to participate directly in clinical trials of new therapeutic approaches in addition to other research opportunities. To determine if you are currently enrolled in the Alpha-1 Research Registry, simply use our new look up tool: www.alpha1.org/rrlt/
Being a part of the Alpha-1 Research Registry allows you to be notified first of new clinical trials that you might qualify to participate in. Being a part of the Registry helps move research forward quicker by identifying and notifying Alphas that can participate in a study as soon as they become available.
Over the next several months, the Alpha-1 Foundation will be providing updates on clinical trials that are being launched, opportunities to take part in those trials and all pertinent information you will need to learn about and participate in trials and be part of helping advance the field toward a cure for Alpha-1.