Eplontersen for Cardiomyopathy

Recruiting at 163 trial locations

Overseen By Ionis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Ionis Pharmaceuticals, Inc.

Must not be taking: Tegsedi, Onpattro, Diflunisal, others

Disqualifiers: Acute coronary syndrome, Cardiomyopathy, MGUS, 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: 31

Duration: 121 weeks

1438 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

Yes, if you are currently taking diflunisal, doxycycline, or certain calcium-channel blockers like verapamil or diltiazem, you will need to stop taking them at least 14 days before joining the trial.

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.

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.

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.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive subcutaneous injections of either eplontersen or placebo once every 4 weeks, along with daily supplemental doses of vitamin A

121 weeks

31 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• AKCEA-TTR-LRx
• Eplontersen
• Placebo

Trial Overview The 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

Group I: Eplontersen Experimental Treatment 1 Intervention

Eplontersen by subcutaneous injection once every 4 weeks

Group II: Placebo Placebo Group 1 Intervention

Eplontersen-matching placebo by subcutaneous injection once every 4 weeks

Who Is Running the Clinical Trial?

Ionis Pharmaceuticals, Inc.

Lead Sponsor

Trials

151

Recruited

27,800+

Dr. Brett P. Monia

Ionis Pharmaceuticals, Inc.

Chief Executive Officer since 2020

PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College

Dr. Eric Bastings

Ionis Pharmaceuticals, Inc.

Chief Medical Officer

MD