Study of ALXN2220 Versus Placebo in Adults With ATTR-CM (DepleTTR-CM)

Alexion Pharmaceuticals

Status and phase

Active, not recruiting

Phase 3

Conditions

Transthyretin Amyloid Cardiomyopathy

Treatments

Drug: ALXN2220

Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT06183931

D6810C00001

ALXN2220-ATTRCM-301 (Other Identifier)

Details and patient eligibility

About

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 between the ALXN2220 and placebo groups as assessed by the total occurrences of all-cause mortality (ACM) and cardiovascular (CV) clinical events.

Enrollment

1,181 patients

Sex

All

Ages

18 to 90 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Diagnosis of ATTR-CM with either wild-type or variant TTR genotype
End-diastolic interventricular septal wall thickness ≥ 12 mm on echocardiography measured at Screening
NT-proBNP > 2000 pg/mL at Screening
Treatment with a loop diuretic for at least 30 days prior to Screening
History of heart failure NYHA Class II-IV at Screening
Life expectancy of ≥ 6 months as per the Investigator's judgment
Males and females of childbearing ability must use contraception

Exclusion criteria

Known leptomeningeal amyloidosis
Known light chain (AL) or secondary amyloidosis (AA), or any other form of systemic amyloidosis
Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
Uncontrolled clinically significant cardiac arrhythmia, per Investigator's assessment
LVEF < 30% on echocardiography
Renal failure requiring dialysis or an eGFR < 20 mL/min/1.73 m2 at Screening
Polyneuropathy with PND score IV

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Quadruple Blind

1,181 participants in 2 patient groups, including a placebo group

ALXN2220

Experimental group

Description:

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.

Treatment:

Drug: ALXN2220

Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo