ALXN2220 for ATTR-CM
(DepleTTR-CM Trial)

Recruiting in Palo Alto (17 mi)

+195 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Alexion Pharmaceuticals, Inc.

Must be taking: Loop diuretics

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

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

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.

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.

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

+4 more

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.

+4 more

Trial 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

Participant Groups

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.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: ALXN2220Experimental Treatment1 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: PlaceboPlacebo Group1 Intervention

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