Can I take part ?

Making
AL amyloidosis
a priority

The PRONTO Amyloidosis Study

Find out about The PRONTO 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 kind 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, and it’s this overproduction that causes them to become misfolded. 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.

AL Amyloidosis body
Nerves
AL Amyloidosis body

Heart (50-70%)

  • Fatigue (inability to be active)
  • Shortness of breath (dyspnea)
  • Irregular heart beat
  • Fainting (syncope)
  • Leads to congestive heart failure

Kidneys (60-80%)

  • Large amounts of protein
    in urine (proteinuria)
  • Swelling of feet and legs
  • End stage kidney disease
  • Transplant can be required

Peripheral nerves (20-45%)

  • Neuropathy
  • Impotence
  • No temperature sensation
    in hands and feet

Other organs

  • Liver (15-30%)
  • GI Tract (5-16%)
  • Eyes (10-25%)
  • Tongue (~10%)
  • Soft Tissue (20-35%)

ABOUT THE 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 (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; 57% achieved a cardiac response and 43% achieved disease stabilization. For patients that were eligible for renal evaluation; 60% achieved a renal response and 40% achieved disease stabilization. These results compare favourably to historical studies.

ABOUT THIS STUDY

  • IN BRIEF
  • STUDY LENGTH
  • STUDY DRUG
  • VISITS

How quickly can we find a potential new treatment for AL Amyloidosis?

The PRONTO Amyloidosis Study, is making the search into finding a potential new treatment a priority. 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 PRONTO study is expected to last up to 14 months, including the screening and treatment periods.

Screening Period

1 month

Dosing Period

up to 12 months


Follow up Period

up to 12 months

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
NEOD001 (the investigational study drug) with standard of care chemotherapy*
Group 2
A placebo (a ‘dummy’ drug) with standard of care chemotherapy*

How and when is this given?

Patients will be given a transfusion given by the study staff during the study visits

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) Patients will be given a transfusion by the study staff during the study visit
Group 2 A placebo (a ‘dummy’ drug)

You will need to visit a study site at regular intervals

  • Your first visit (screening visit)
  • Then once a 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 Blood tests
  • Urine tests Urine tests
  • Electrocardiograms Electrocardiograms
  • Physical<br />assessments Physical
    assessments
  • Vital signs<br />examinations Vital signs
    examinations
  • 6-minute<br />walk tests 6-minute
    walk tests
  • Quality of life<br />questionnaires Quality of life
    questionnaires

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 clinical research 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 PRONTO 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:

  1. Are you over 18 years of age?
  2. Have you been diagnosed with AL amyloidosis with heart involvement?
  3. Have you been treated for AL amyloidosis with heart involvement?

Submit

Lastly, please leave your details

So that we can get in touch with you about your potential involvement in The PRONTO Amyloidosis Study, please complete this form with your details.

Only the clinical study team will have access to your contact details.
Prothena will not use your details for promotional purposes.
*Mandatory

Thank you

Many thanks for your interest in The PRONTO 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 PRONTO Amyloidosis Study. Please contact your doctor to discuss your options or join our mailing list for upcoming news.

Can I take part?

Thank you for your interest.

Unfortunately, you are not eligible for The PRONTO Amyloidosis Study, however you may be eligible for the VITAL study. Here is a link to the website.

USEFUL RESOURCES

Patient groups are a good place to find out more about amyloidosis

Amyloidosis Foundation

Amyloidosis Support Group

Michael York video