Can we unfold
The VITAL Amyloidosis Study
Find out about The VITAL Amyloidosis Study and discover if you can take part.
If you have any questions, please do not hesitate to contact us.
About AL amyloidosis
A rare and difficult to diagnose disease
Systemic amyloidosis is a family of rare progressive diseases in which abnormally folded proteins cluster together (aggregate). These are known as amyloid aggregates, and they can build up and cause damage in organs and tissues.
There are different kinds of systemic amyloidosis, with AL (amyloid light chain) amyloidosis being the most common form.
Misfolded protein in the heart
In AL amyloidosis, plasma cells, a part of the immune system, overproduce an antibody fragment called light chain protein in an abnormal form that misfolds. The resulting amyloid accumulates in one or more organs, where it can disrupt normal functioning. The heart and kidneys are often sites of amyloid deposition in this disease. There are currently no approved therapies for AL amyloidosis.
- Fatigue (inability to be active)
- Shortness of breath (dyspnea)
- Irregular heart beat
- Fainting (syncope)
- Leads to congestive heart failure
- Large amounts of protein
in urine (proteinuria)
- Swelling of feet and legs
- End stage kidney disease
- Transplant can be required
Peripheral nerves (20–45%)
- No temperature sensation
in hands and feet
- Liver (15–30%)
- GI Tract (5–16%)
- Eyes (10–25%)
- Tongue (~10%)
- Soft Tissue (20–35%)
About study drug
The investigational study drug, NEOD001, is the first monoclonal antibody that specifically targets misfolded light chain proteins, which are the disease causing proteins of AL amyloidosis. The proposed mechanism of action of NEOD001 is to (i) neutralize soluble aggregates and (ii) clear insoluble aggregates from organs.
In an ongoing phase 1/2 study in 27 patients with AL amyloidosis and persistent organ dysfunction, NEOD001 was safe and generally well tolerated. The pharmacokinetic profile was consistent with IV dosing once every 28 days.
In the phase 1/2 study, for patients that were eligible for cardiac evaluation, 50% patients achieved a cardiac response and 50% patients achieved disease stabilization. For patients that were eligible for renal evaluation, 43% patients achieved a renal response and 57% patients achieved disease stabilization. These results compare favorably to historical studies.
About this study
- In brief
- Study length
- Study drug
Investigating a different approach to AL Amyloidosis
The VITAL Amyloidsosis Study, is investigating a potential new treatment option for patients. The investigational study drug is the first monoclonal antibody that specifically targets misfolded light chain proteins, which are the disease-causing proteins of AL amyloidosis. The study is expected to support approval of the first disease-modifying therapy for patients with AL amyloidosis.
How long will the study last?
The VITAL study is expected to last approximately 42 months, including the enrollment and treatment periods.
Not everyone receives the investigational study drug NEOD001
If you join our study you will be placed randomly (by chance) into one of two groups. You won’t know or be allowed to choose which group you’re placed in.
The investigational study drug and study related procedures would be provided at no cost.
|Group 1 regimen|
|NEOD001 (the investigational study drug) with standard of care chemotherapy*|
|Group 2 regimen|
|A placebo (a ‘dummy’ drug) with standard of care chemotherapy*|
How and when is this given?
Patients will be given a transfusion by the study staff during the study visit.
What is a placebo and why is it needed?
- A placebo is a ‘dummy’ drug that contains no active ingredient
- It looks just like the investigational study drug and is given in the same way
- This allows us to be sure that any effects seen are due to the investigational study drug and nothing else
|Which regimen will I receive?||How and when is this given?|
|Group 1||NEOD001 (the investigational study drug) with standard of care chemotherapy*||Patients will be given a transfusion by the study staff during the study visit|
|Group 2||A placebo (a ‘dummy’ drug) with standard of care chemotherapy*|
* The current standard of care is aimed at reducing or eliminating bone marrow disorder, the plasma cell dyscrasia.
You will need to visit a study site at regular intervals
- Every week or two weeks during the first two months (some visits may be able to take place at your home)
- Then every month for the remainder of the study
- There may be additional weekly visits during the second and third months for chemotherapy
During these visits we’ll administer your investigational study drug infusion and at some visits we’ll administer additional chemotherapy. Additionally we’ll monitor your health using procedures and tests such as:
- Blood tests
- Urine tests
Quality of life
About clinical research
What is a clinical research study?
Clinical research studies are carefully designed scientific investigations of potential new medications. They help us answer important medical questions, such as:
- How an investigational medication acts in the body
- How it affects certain diseases or conditions
- Whether or not it is safe for wider use
Every year, thousands of people around the world take part in such studies. Independent institutional review boards and ethics committees look after patients’ best interests by protecting their rights and ensuring they aren’t exposed to unnecessary risks.
Can I take part?
Thank you for your interest in The VITAL Amyloidosis Study.
To find out if you are eligible for this study, please take a few minutes to complete the following pre-screening questions. All information you provide will be kept confidential.
If you meet the pre-screening criteria, someone from your nearest study site will contact you.
Now please answer these questions about yourself:
- Are you over 18 years of age?
- Are you recently diagnosed with AL amyloidosis with heart involvement?
- Have you previously been treated (e.g. chemotherapy) for your AL amyloidosis?
Many thanks for your interest in The VITAL Amyloidosis Study. One of our study team will be in touch shortly.
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Can I take part?
Thank you for your interest.
Unfortunately, you are not eligible for The VITAL Amyloidosis Study. Please contact your doctor to discuss your options or join our mailing list for upcoming news.