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 Antitrypsin Deficiency and improve health care for Alphas in the future.
The Alpha-1 Research Registry is a confidential database made up of individuals diagnosed with Alpha-1 Antitrypsin Deficiency (Alpha-1) and individuals identified as Alpha-1 carriers. The Registry was established to facilitate research initiatives and promote the development of improved treatments and a cure for Alpha-1. Located at the Alpha-1 Foundation, Miami, FL, the Registry procedures ensure stringent confidentiality of participants. 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.
A clinical trial will always start with the informed consent so that you can ask the study team any questions they may have regarding the clinical trial. The informed consent is a document that is presented to you explaining the potential risks/benefits associated with the clinical trial. Once the participant agrees to take part in the clinical trial, the informed consent will be signed by both the participant 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 always withdraw your consent at any point.
Eligibility criteria are the reasons that 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, sex and established diagnosis of Alpha-1 Antitrypsin. Some exclusion criteria examples are clinically significant health concerns other than alpha-1 or lung or liver transplant.
The Alpha-1 Foundation has worked hard to prevent placebo controlled trials for new or next generation plasma based products. But we may not be able to prevent a placebo controlled arm in new transformative trials.