ALXN2220 for ATTR-CM

(DepleTTR-CM Trial)

Recruiting at 205 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

1158 Participants Needed

Trial Summary

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.

What is the purpose of this trial?

This trial is testing a new drug called ALXN2220 to help adults with a heart condition known as ATTR-CM. The goal is to see if the drug can lower the risk of death and serious heart issues. Researchers are comparing the outcomes between those who receive the drug and those who do not.

Eligibility Criteria

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

NT-proBNP > 2000 pg/mL at Screening

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

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

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

Treatment Details

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.

Participant Groups

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