Do you or a loved one have advanced AL amyloidosis?
Find out more about the AFFIRM-AL clinical research study.
Contact usThe AFFIRM-AL study is now enrolling adults 18 years of age or older with AL amyloidosis. Participants must be in Mayo Stage IV and have not yet received any medical treatment for AL amyloidosis.
AL amyloidosis is a rare disease that occurs when an abnormal protein, called amyloid, builds up and interferes with normal organ function. One classification system used to stage the prognosis of AL amyloidosis is the Mayo Staging system, a classification system for newly diagnosed patients. Specific laboratory tests determine the stage. Mayo Stage IV is one of the stages within this system.
The AFFIRM-AL study is a phase 3 clinical research study evaluating an investigational drug called birtamimab to find out if it works and if it is safe when given with chemotherapy to people with AL amyloidosis who are in Mayo Stage IV.
A Phase 3 clinical research study is the final phase of research evaluating an investigational drug’s effectiveness and safety before it can be approved for use in the general patient population.
Birtamimab is being tested in adults who are newly diagnosed with AL amyloidosis and are in Mayo Stage IV.
Study participants will receive either the investigational drug and standard of care chemotherapy or placebo and standard of care chemotherapy. Placebo is a substance that looks like the investigational drug but has no active drug in it. In this study, the placebo is saline.
You may potentially be eligible to participate in AFFIRM-AL if:
- You are 18 years of age (and legal age of consent) or older
- You are newly diagnosed with Mayo Stage IV AL amyloidosis
- You have not yet received any medical treatment for AL amyloidosis
This is not a complete list of study requirements. Your doctor will review all requirements with you.
-
Study purpose:
To measure the efficacy and safety of birtamimab plus chemotherapy in adults with AL amyloidosis in Mayo Stage IV -
Study drugs:
All participants will receive standard of care chemotherapy as prescribed by the study doctor. In addition, all participants will receive one of the following monthly intravenously (by IV, or through a vein):- Birtamimab (investigational drug)
- Placebo (saline)
-
Study group assignment:
All participants will be randomly assigned to a study group.- You have a 2-in-3 chance to receive the investigational drug and standard of care chemotherapy (about a 66% chance)
- You have a 1-in-3 chance to receive placebo and standard of care chemotherapy (about a 33% chance)
- The study will be double-blinded. This means neither you, as a study participant, nor the study team will know whether you are receiving birtamimab or placebo
-
Length of study:
2.5 to 3.5 years (until the study is completed) -
Number of visits:
Varies- You will receive lab tests and health assessments in the weeks following each infusion
- The number of total visits will be determined by when you enroll
- Your study doctor will decide if you receive chemotherapy at the study clinic or through your local physician. This may increase the number of clinic visits
-
Number of participants:
About 220 people -
Completing the study:
Once this study is complete, you may qualify to continue taking birtamimab as part of another study called an Open-Label Extension Study. The study doctor and staff will give you details on this study if you are interested and eligible to enroll.
The interactive map below shows the countries and cities of the study centers that are actively participating in the AFFIRM-AL study. The list shows the study centers within the United States first. To view other countries, please scroll down.
To view a country on the interactive map:
- You can either click on the + sign (bottom left) to zoom in on an area
- Click the pin to view the name and location of the study center, or you can move your mouse over the map
- Click down on your mouse, to move the map to the left, the right, or up and down. If you’re using a Smartphone or tablet, use your finger to move the map around.
Please note, this list may not include all participating study centers. Please refer to ClinicalTrials.gov/NCT04973137 to view the most up-to-date list of participating study centers.
Before a new therapy can be made available to the general patient population, multiple studies involving patient volunteers must be conducted to evaluate the safety and effectiveness of the therapy. These studies, referred to as clinical trials, are regulated by the Food and Drug Administration (FDA) in the United States and by similar regulatory agencies worldwide.
Participation in the AFFIRM-AL study will help us understand whether the investigational drug, birtamimab, is effective and safe for adults with newly diagnosed AL Amyloidosis in Mayo Stage IV.
Taking part in a clinical study is voluntary. If you are eligible to enroll, you may choose to join the study but leave at a later date for any reason at any time. Regardless of whether you choose to enroll or leave the study early, your future healthcare won’t be affected.
Frequently asked questions (FAQs)
AL amyloidosis is a rare disease that occurs when an abnormal protein, called amyloid, builds up and interferes with normal organ function. One classification system used to stage the prognosis of AL amyloidosis is the Mayo Staging system, a classification system for newly diagnosed patients. Specific laboratory tests determine the stage. Mayo Stage IV is one of the stages within this system.
In a clinical research study, participants may receive an investigational drug to help researchers answer questions about the study drug, find out if it works, and if it has side effects.
An investigational drug is a substance that is being tested in clinical studies. It has been reviewed by ethics committees for testing in people and has not been approved for use in the general patient population by the FDA in the United States or by similar regulatory agencies worldwide.
AL amyloidosis is a rare disease and there are very few treatment options for advanced-stage patients. Clinical studies (also called clinical trials) are important for medical advances. Current treatments for diseases are only available because of research study volunteers. This study will help researchers better understand whether the investigational drug, birtamimab, is effective and safe for adults with newly diagnosed AL Amyloidosis in Mayo Stage IV.
This study is enrolling adults who have been newly diagnosed with AL amyloidosis and are in Mayo Stage IV and have not yet received any treatment for AL amyloidosis.
You do not have to pay for the study drug, study supplies, or tests that are part of the research study. There may be services for which patients may have a co-payment or deductible.
Study participation will be up to 2.5 to 3.5 years.
Once you have agreed to participate and it is determined that you meet all the study requirements to participate in the AFFIRM-AL study, you will be closely monitored for any side effects throughout the study. The investigational drug may cause side effects, although not everyone may experience them. The possibility of serious side effects that may require treatment cannot be ruled out. Please see the informed consent form (ICF) for the details regarding potential side effects or speak to the study doctor for any safety concerns.
During the clinical study, you must immediately inform the study doctor if you notice any changes in your health or suspect that you are experiencing any side effects. The study staff may treat side effects if they occur. If you do experience a side effect, you may be asked to visit the study site more frequently for follow-up and examination.
Taking part in a clinical study is voluntary. If you are eligible to enroll, you may choose to join the study but leave at a later date for any reason at any time. Regardless of whether you choose to enroll or leave the study early, your future healthcare won’t be affected.
Prothena Biosciences Limited is the sponsor for conducting this clinical study.
FAQ2
A list of participating study centers appears in the Find a study center section above.
For more information on study enrollment, email AFFIRM-ALClinicalTrial@prothena.com.
A placebo is a substance that looks like the investigational drug but has no active drug in it. In this study, the placebo is saline.
Once this study is complete, you may qualify to continue taking birtamimab as part of another study called an Open-Label Extension Study. The study doctor and staff will give you details on this study if you are interested and eligible to enroll.
In addition to receiving either birtamimab (investigational drug) or placebo (saline) in this study, all participants will receive standard of care chemotherapy as prescribed by the study doctor. Your study doctor may, at the time of randomization, give you another drug call daratumumab. Your study doctor will decide if you receive chemotherapy at the study clinic or through your local physician. This may increase the number of clinic visits.
No. You do not need health insurance to participate or to receive the study drug, study supplies, or tests that are part of the research study. However, there may be services for which patients may have a co-payment or deductible. The study staff can explain which services may require payment.
The study staff will review all medications you are currently taking or have recently taken and will let you know if you need to stop taking any of these medications.
Birtamimab is a type of protein known as a monoclonal antibody. In this study, participants will receive either birtamimab (investigational drug) or placebo (saline) by intravenous (IV) infusion once monthly.
Ask your doctor about clinical research studies, or you can visit the National Institute on Aging or the US National Library of Medicine for more information.
