What side effects are associated with this type of intervention?

Common treatments for amyloidosis, such as RNA silencing and gene editing, generally show promise but come with potential side effects. RNA silencing can lead to mild adverse effects, as seen in early studies, while gene editing approaches like CRISPR-Cas9 have shown mild adverse effects in initial trials. Systemic medications used for amyloidosis, such as oral retinoids, cyclosporine, and thalidomide, can have significant side effects, including immunosuppression and increased risk of infections. It is crucial to weigh these risks against the potential benefits when considering treatment options.

What prior FDA approvals exist?

The FDA has approved several treatments for amyloidosis, particularly for ATTR and AL types. For ATTR amyloidosis, RNA-targeted therapies like patisiran and inotersen have been approved, which work by reducing the production of the amyloidogenic protein transthyretin (TTR). In AL amyloidosis, treatments include chemotherapy regimens such as cyclophosphamide, bortezomib, and dexamethasone, as well as monoclonal antibodies like daratumumab. These treatments aim to reduce the production of amyloidogenic light chains and dissolve existing amyloid deposits.

What other types of research exist?

One promising novel alternative to AT-02 for amyloidosis patients is recombinant ADAMTS-13 (BAX 930; SHP655). This treatment has been investigated for its safety, tolerability, and pharmacokinetics in patients with severe congenital ADAMTS-13 deficiency. It was well tolerated, with no serious adverse events or anti-ADAMTS-13 antibodies observed. This suggests it could be a viable option for amyloidosis patients seeking new treatments in 2024.

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Monoclonal Antibodies

AT-02 for Amyloidosis

Phase 2

Recruiting

Research Sponsored by Attralus, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subject must have participated in the study AT01-001 and wishes to receive open-label AT-02.

Be older than 18 years old

Must not have

Has acquired any new, clinically significant underlying illness since enrollment in the parent study.

Is mentally or legally incapacitated, has significant emotional problems at the time of the study, or has a history of psychosis.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 112 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new medicine called AT-02 for people with systemic amyloidosis who were in an earlier study. It aims to see if the medicine is safe and effective over time.

Who is the study for?

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).

What is being tested?

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.

What are the potential side effects?

Specific side effects of AT-02 aren't listed here but generally include reactions related to safety and tolerability that will be monitored throughout the trial since it's an investigational drug.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I was part of the AT01-001 study and want to continue with AT-02.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

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

Select...

I do not have any mental health issues that would prevent me from participating.

Select...

My kidney function is severely reduced.

Select...

I am not taking any medications that are not allowed in this study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 112 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 112 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 112 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Incidence of treatment-emergent Anti-drug antibodies (ADAs)

To assess PK of AT-02 during long-term administration

To evaluate the clinical efficacy of AT-02 during long-term administration through change from baseline in biomarkers

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: A (AT-02)Experimental Treatment1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

AT02

2008

Completed Phase 2

~80

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for amyloidosis include RNA-targeted therapies, gene editing, and proteasome inhibitors. RNA-targeted therapies, such as patisiran and inotersen, work by silencing the genes responsible for producing amyloidogenic proteins, thereby reducing the formation of amyloid deposits. Gene editing techniques, like CRISPR-Cas9, aim to permanently disable the genes that produce these harmful proteins. Proteasome inhibitors, such as bortezomib, help to degrade misfolded proteins that contribute to amyloid formation. These mechanisms are crucial for amyloidosis patients as they target the root cause of the disease—abnormal protein production and deposition—offering the potential for more effective and long-lasting treatments.

Amyloid-β positron emission tomography imaging probes: a critical review.A clinico-epidemiological study of macular amyloidosis from north India.The genetics of the amyloidoses: interactions with immunity and inflammation.