D8456C00001- Phase2b - Eplontersen & ALXN2220 vs Eplontersen & Placebo in Adult Pts.with ATTRCM

• Research type

  Research Study

• Full title

  A Phase IIb, Randomised, Double-blind, Placebo-controlled, Multicentre Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo in Adult Participants with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

• IRAS ID

  1013827

• Contact name

  Philip Marmon

• Contact email

  AZ_RDU_StudyStartUp@astrazeneca.com

• Sponsor organisation

  AstraZeneca AB

• Clinicaltrials.gov Identifier

  NCT07608354

• Research summary

  Researchers are looking for a better way to treat a heart condition called transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). ATTR-CM is a serious heart condition that happens when a protein called transthyretin changes shape and builds up in the
  heart as clumps called amyloids. Over time, these clumps can make the heart stiff and weak, so it has a harder time pumping
  blood. People with ATTR-CM may have a shorter lifespan than those without the condition. Common symptoms include an
  abnormal heartbeat, shortness of breath, fatigue, and swelling in the legs.
  Current medicines for ATTR-CM can help some people live longer and have fewer symptoms. However, they may not work for
  everyone, and many people continue to get worse over time even while taking medication.
  Eplontersen is a medicine that is approved in some countries to treat nerve problems caused by an abnormal build-up of
  transthyretin in the body. It is designed to lower the amount of both normal and misshaped transthyretin the body makes, which
  may help reduce build-up in the heart. The trial drug ALXN2220 is designed to find and clear out the abnormal transthyretin that has already built up in the body’s tissues, such as the heart. Researchers want to learn how safe eplontersen with ALXN2220 is and how well it works to remove abnormal transthyretin from the heart compared to how well eplontersen without ALXN2220 works. A placebo is used to help show whether the results are due to the trial treatment or if they could be due to chance. In this trial, the placebo looks like ALXN2220 but does not contain any active medicine. Participants can remain on standard therapy, as determined by their treating physician and the investigator. Standard therapy can include conventional heart failure therapies and approved disease-modifying therapies for ATTR-CM. Use of TTR silencing or depleter therapies, other than eplontersen and ALNX2220, is prohibited prior to and during the study.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  26/LO/0243

• Date of REC Opinion

  13 May 2026

• REC opinion

  Further Information Favourable Opinion