AT-02 for Amyloidosis

Recruiting at 10 trial locations

Overseen By Deepika Aggarwal

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Attralus, Inc.

Must not be taking: Prohibited medications

Disqualifiers: Pregnancy, Psychosis, Low eGFR, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 104 weeks

120 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called AT-02 for individuals with systemic amyloidosis, a condition where abnormal proteins accumulate in organs. The trial aims to determine if AT-02 is safe and effective over an extended period. Participants will receive the treatment via IV every two to four weeks for approximately two years. The trial seeks individuals diagnosed with AL amyloidosis who have shown a stable response to previous treatments. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it mentions that you cannot use any prohibited medications. It's best to discuss your current medications with the study team to see if they are allowed.

Is there any evidence suggesting that AT-02 is likely to be safe for humans?

Research has shown that AT-02 has been tested in earlier studies to assess its safety and tolerability. In one study with healthy volunteers, researchers administered single doses of AT-02 to observe how the body processes it. The study found that participants generally tolerated AT-02 well.

While exact numbers on side effects are unavailable, the study suggests that AT-02 did not cause serious problems in these volunteers. AT-02 is also undergoing testing in individuals with systemic amyloidosis, but safety results from those trials are still being collected.

This trial is in Phase 2, indicating that AT-02 has already passed initial safety tests. This phase focuses on evaluating its safety in a larger group. If AT-02 were unsafe, it would not have reached this stage. However, like any new treatment, risks may still exist, so discussing participation with a healthcare provider is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

AT-02 is unique because it is specifically designed to target and reduce amyloid deposits in patients with amyloidosis through intravenous infusion. Unlike current treatments, which often focus on managing symptoms, AT-02 directly addresses the underlying cause of amyloidosis by breaking down the amyloid proteins. Researchers are excited about its potential to provide a more targeted and effective approach, potentially improving patient outcomes over the traditional therapies that mainly offer symptomatic relief.

What evidence suggests that AT-02 might be an effective treatment for amyloidosis?

Research has shown that AT-02, the investigational treatment in this trial, can help the body eliminate amyloid, a protein that accumulates in organs in people with amyloidosis. AT-02 enhances the body's natural process of clearing these amyloid deposits. In patients with a type of amyloidosis who were in remission, those who took AT-02 for 40 weeks showed improvement. Lab studies indicate that AT-02 can attach to different types of amyloid in major organs, suggesting it might be useful for various forms of the disease. These findings offer hope that AT-02 could be a promising treatment for people dealing with systemic amyloidosis. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for people who have systemic amyloidosis and were part of the AT01-001 study. They should be able to follow the study plan, not be in other trials recently, and not planning pregnancy. They must have finished previous parts of this research without serious issues and their kidneys must work well (eGFR >30 mL/min/1.73 m2).

Inclusion Criteria

Subject understands the study procedures and can give signed informed consent.

I am willing and able to follow the study rules and can participate for its full duration.

You must have been diagnosed with SA according to the rules in the main study's plan.

Exclusion Criteria

Is currently participating in an interventional clinical study or has participated in another clinical study (other than AT02-001) within the last four (4) weeks or within five (5) half-lives of the prior study treatment, whichever is longer.

Is pregnant, breastfeeding, or is planning to become pregnant or breastfeed during this study and follow-up period.

I have developed a new serious illness since joining the initial study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

8 weeks

Treatment

Participants receive AT-02 via intravenous infusion once every two or four weeks for 104 weeks

104 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

Open-label extension

Participants continue to receive AT-02 to evaluate long-term safety and tolerability

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• AT-02

Trial Overview The trial is testing a new treatment called AT-02 for systemic amyloidosis over a long period to see how safe it is, if there are any side effects, and how effective it might be at treating the condition.

How Is the Trial Designed?

Group I: A (AT-02) Experimental Treatment 1 Intervention

Subjects will receive AT-02 via intravenous infusion once every two or 4 weeks for 104 weeks (52 total AT-02 administrations).

AT-02 is already approved in European Union, United States for the following indications:

Approved in European Union as AT-02 for:

• Transthyretin-associated amyloidosis (ATTR)
• Immunoglobulin light-chain-associated amyloidosis (AL)

Approved in United States as AT-02 for:

• Transthyretin-associated amyloidosis (ATTR)

Who Is Running the Clinical Trial?

Attralus, Inc.

Lead Sponsor

Trials

8

Recruited

670+

Novotech (Australia) Pty Limited

Industry Sponsor

Trials

76

Recruited

7,800+

Dr. John Moller

Novotech (Australia) Pty Limited

Chief Executive Officer

MD and MBA from the University of Oxford

Dr. Judith Ng-Cashin

Novotech (Australia) Pty Limited

Chief Medical Officer since 2023

MD

Citations

1. nature.com nature.com/articles/s44386-025-00015-4

The antibody-peptide fusion protein, AT-02, is an effective ... We demonstrate that AT-02 is an effective opsonin for amyloid by enhancing phagocytosis of amyloid extracts and inducing complement fixation.

2. attralus.com attralus.com/press-releases/attralus-presents-new-data-on-its-pan-amyloid-therapeutic-and-diagnostic-candidates-at-the-xix-international-symposium-on-amyloidosis-isa

Attralus Presents New Data on Its Pan-Amyloid ... Preclinical data have shown the ability of AT-02 to bind to multiple amyloid types in major organs, induce macrophage mediated phagocytosis, and ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05521022

NCT05521022 | Study of AT-02 in Healthy Volunteers and ... Systemic amyloidosis is an incurable disease, and about 20% of patients with cardiac or advanced kidney involvement experience early deaths (<1 year). Despite ...

4. globenewswire.com globenewswire.com/news-release/2025/11/04/3180123/0/en/Attralus-To-Present-Phase-1-2-Data-on-Its-Pan-Amyloid-Depleter-at-the-67th-American-Society-of-Hematology-ASH-Annual-Meeting-and-Exposition.html

Attralus To Present Phase 1/2 Data on Its Pan Amyloid The presentation includes data on AL patients in hematological remission who received AT-02 (2.5 gm q2w) for 40 weeks and had improvement in ...

5. attralus.com attralus.com/press-releases/attralus-therapeutic-at-02-receives-orphan-drug-designation-from-the-u-s-fda-for-the-treatment-of-attr-amyloidosis

Attralus Therapeutic AT-02 Receives Orphan Drug ... Preclinical data have shown the ability of AT-02 to bind to multiple amyloid types in major organs and induce macrophage mediated amyloid ...

6. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/489635

A Study of AT-02 in Subjects With Systemic Amyloidosis. This is a Phase 2 open-label extension study to evaluate the long-term safety, tolerability, and clinical activity of AT-02.