ALXN2220 for ATTR-CM

(DepleTTR-CM Trial)

Not currently recruiting at 245 trial locations

Overseen By Alexion Pharmaceuticals, Inc. (Sponsor)

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Alexion Pharmaceuticals, Inc.

Must be taking: Loop diuretics

Disqualifiers: Leptomeningeal amyloidosis, AL amyloidosis, AA amyloidosis, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: 4

Duration: 24-48 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ALXN2220 for individuals with ATTR-CM, a heart condition that leads to heart failure due to abnormal protein deposits. The trial aims to determine if ALXN2220 can reduce deaths and serious heart problems compared to a placebo, which contains no active drug. Participants will receive either ALXN2220 or a placebo through regular IV infusions for up to four years. Ideal candidates are those diagnosed with ATTR-CM who exhibit heart failure symptoms and currently use a loop diuretic, a type of water pill. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to potentially groundbreaking treatment advancements.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. However, since participants must have been on a loop diuretic for at least 30 days before screening, it seems that continuing certain medications might be required.

Is there any evidence suggesting that ALXN2220 is likely to be safe for humans?

Research has shown that ALXN2220 is generally safe for people with ATTR-CM, a heart condition caused by protein buildup. Studies have found that it helps reduce these protein deposits, suggesting it could be an effective treatment.

In earlier research, some participants experienced mild side effects, but no serious health issues were directly linked to ALXN2220. As this treatment nears the final stages of testing, it has maintained a good safety record. However, individual reactions may vary, so discussing any concerns with a doctor before joining a trial is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for ATTR-CM, which often focus on stabilizing the transthyretin protein or reducing amyloid deposits, ALXN2220 introduces a novel approach by using a specific monoclonal antibody that targets and removes amyloid deposits from the heart. This mechanism could potentially reverse some of the damage caused by amyloid buildup, rather than just slowing its progression. Researchers are excited about ALXN2220 because it offers a new way to directly address the root cause of the disease, potentially improving heart function and quality of life for patients with ATTR-CM.

What evidence suggests that ALXN2220 might be an effective treatment for ATTR-CM?

Research has shown that ALXN2220, which participants in this trial may receive, is designed to reduce harmful protein deposits in the heart linked to a condition called ATTR-CM. This treatment aims to complement standard therapies to improve heart function and overall health. Studies have found that ALXN2220 can decrease these deposits, potentially leading to better survival rates and fewer heart issues. While ongoing research continues to examine its long-term effects, early results appear promising for patients with this condition. The goal is to determine if ALXN2220 can significantly reduce deaths and heart problems caused by ATTR-CM. ^{1 2 3 5 6}

Are You a Good Fit for This Trial?

This trial is for adults with ATTR-CM, a type of heart disease caused by amyloid buildup. Participants should have symptoms and evidence of the condition but not be on other treatments for it. Those with other types of cardiomyopathy or who can't follow the study protocol are excluded.

Inclusion Criteria

I have been on a water pill for at least 30 days.

NT-proBNP > 2000 pg/mL at Screening

Life expectancy of ≥ 6 months as per the Investigator's judgment

Exclusion Criteria

I have been diagnosed with leptomeningeal amyloidosis.

I haven't had any major heart issues or surgeries in the last 3 months.

My heart rhythm is stable and not causing health issues.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either ALXN2220 or placebo via IV infusion every 4 weeks for at least 24 months up to a maximum of 48 months

24-48 months

IV infusion every 4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

What Are the Treatments Tested in This Trial?

Interventions

• ALXN2220

Trial Overview The study tests ALXN2220's effectiveness against a placebo in treating ATTR-CM. It measures which group has fewer deaths and cardiovascular events over time to see if ALXN2220 makes a difference.

How Is the Trial Designed?

Group I: ALXN2220 Experimental Treatment 1 Intervention

Participants will receive weight-based dose of ALXN2220 via intravenous (IV) infusion every 4 weeks (q4w) for at least 24 months up to a maximum of 48 months.

Group II: Placebo Placebo Group 1 Intervention

Participants will receive placebo via IV infusion q4w for at least 24 months up to a maximum of 48 months.

Who Is Running the Clinical Trial?

Alexion Pharmaceuticals, Inc.

Lead Sponsor

Trials

267

Recruited

141,000+

Dr. Alberto R. Martinez

Alexion Pharmaceuticals, Inc.

Chief Medical Officer since 2010

MD from University of Sao Paulo

Marc Dunoyer

Alexion Pharmaceuticals, Inc.

Chief Executive Officer since 2021

PhD in Molecular Biology

Citations

1. academic.oup.com academic.oup.com/eurheartj/article/45/Supplement_1/ehae666.1095/7835381

Long-term safety and efficacy of antibody ALXN2220 for ... ALXN2220 is designed to deplete cardiac amyloid transthyretin (ATTR) deposits in patients with ATTR-CM on top of standard of care.

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40262023/

Antibody ALXN2220 (Formerly NI006) for the Treatment of ... A multinational, placebo-controlled phase 3 trial (DepleTTR-CM) is ongoing to assess long-term efficacy, functional outcomes, and survival ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06183931

Study of ALXN2220 Versus Placebo in Adults With ATTR-CM The primary objective of this study is to access the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference ...

4. neurimmune.com neurimmune.com/news/long-term-safety-and-efficacy-of-antibody-alxn2220-for-depletion-of-cardiac-attr-to-be-presented-at-esc-congress-2024

Long-term safety and efficacy of antibody ALXN2220 for ... ALXN2220 is an investigational human monoclonal antibody designed to deplete amyloid deposits in ATTR-CM as add-on to standard of care.

5. clinicaltrials.gov clinicaltrials.gov/study/NCT07213583

NCT07213583 | Study of Re-Treatment With ALXN2220 in ... This is a Phase 2, single arm, multicenter study to evaluate the pharmacodynamics and safety of re-treatment with ALXN2220 in the patient ...

6. nejm.org nejm.org/doi/full/10.1056/NEJMoa2303765

Phase 1 Trial of Antibody NI006 for Depletion of Cardiac ... Available data from patients with ATTR cardiomyopathy indicate that extracellular volume on cardiac MRI correlates with the amyloid load on histologic analysis, ...