If you have been diagnosed with Alpha-1 Antitrypsin Deficiency Associated Liver Disease, there is hope on the horizon. With the newest advancements in medicine, progress is underway to help find a potential treatment for this condition. No matter where you are in your journey with Alpha-1 liver disease, you may be able to take part. In the rare disease community, it is often clinical trials that offer hope to find better treatments for others in the future.
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 if 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 (treatment or therapy) 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.
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. Ultimately, the goal of clinical trials is to determine if a new test or treatment works and is safe.
Why are clinical trials important?
If you have been diagnosed with Alpha-1 Liver Disease or if it runs in your family, then you may know that currently there is no approved treatment option for this condition. In the rare disease community, it is often clinical trials that offer the most hope to find better treatments for others in the future.
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 latest treatment and to have additional care and attention from the clinical trial staff.
How do I know if I am eligible to enroll in a study?
Eligibility criteria, which includes exclusion and inclusion 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 consultation 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.
For more information on clinical trials, please click here.
The Redwood Clinical Study for Liver-Affected Alphas
The Takeda-sponsored Redwood Study for Alpha-1 Liver Disease is enrolling! The investigational study drug, TAK-999 (fazirsiran), aims to reduce the production of the abnormal Z-AAT protein and its buildup in the liver. The reduction in levels of the protein may result in a decrease in liver scarring. The investigational study drug being evaluated alongside the placebo, which appears similar to TAK-999 but contains no active medication, may help researchers understand whether TAK-999 can reduce liver scarring caused by Alpha-1 Liver Disease.
Who can participate in the Redwood Study?
- Individuals between 18 and 75 years of age
- Individuals diagnosed with AATD and have the PiZZ genotype or are willing to undergo confirmatory testing at screening
- Individuals or their family members who have a confirmed or suspected diagnosis of Alpha-1 Liver Disease
- Individuals who do not have a history of the following: liver cancer or any organ transplant
- Individuals who have not smoked any substance for the past 12 months
Study Details:
- Participation in The Redwood Study will last approximately 4.5 years.
- Eligible participants will be chosen at random to receive either TAK-999 (fazirsiran) or a placebo.
- The procedures performed during the study (at no cost to participants) include liver biopsies, questionnaires, physical examinations, vital sign measurements, pulmonary function tests, and blood sample collections.
How do I enroll?
The first step is to complete the following pre-screening form to see if you may qualify. Please note this is not a full list of criteria and additional screening may be needed. Please click the link below to begin.
To access the Alpha-1 Foundation Pre-Screening, click here.
For any questions or concerns, please contact the Alpha-1 Foundation Research Registry Coordinators at 1-877-228-7321 ext. 245 or 252, or alpha1registry@alpha1.org.
