Eplontersen for Cardiomyopathy

Recruiting in Palo Alto (17 mi)

+163 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Ionis Pharmaceuticals, Inc.

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

To evaluate the efficacy of eplontersen compared to placebo in participants with ATTR-CM receiving available standard of care (SoC). For more information, please visit https://www.cardio-ttransform.com.

Research Team

Eligibility Criteria

This trial is for adults with ATTR-CM, a type of heart disease caused by amyloid deposits. Participants must have certain heart measurements and confirmed amyloid deposits but can't be on specific heart medications, have had recent major cardiovascular events or procedures, or expect to need a heart transplant or assist device within a year.

Inclusion Criteria

I am not pregnant or breastfeeding, and I use effective birth control or am not able to have children.

My tests show amyloid deposits in my heart or other tissues.

My heart condition allows me to perform daily activities with minimal to moderate difficulty.

See 2 more

Exclusion Criteria

I have had or am expected to have a liver or heart transplant, or need an LVAD within a year.

My heart condition is not mainly due to ATTR-CM.

I am not currently taking diflunisal, doxycycline, ursodeoxycholic acid, verapamil, or diltiazem, or I stopped them at least 14 days ago.

See 3 more

Treatment Details

Interventions

AKCEA-TTR-LRx (Other)
Eplontersen (Antisense Oligonucleotide)
Placebo (Other)

Trial OverviewThe study tests Eplontersen's effectiveness against a placebo in patients with ATTR-CM who are also receiving standard care. The goal is to see if Eplontersen improves the condition more than an inactive substance.

Participant Groups

2Treatment groups

Experimental Treatment

Placebo Group

Group I: EplontersenExperimental Treatment1 Intervention

Eplontersen by subcutaneous injection once every 4 weeks

Group II: PlaceboPlacebo Group1 Intervention

Eplontersen-matching placebo by subcutaneous injection once every 4 weeks