At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Eplontersen (drug), ALXN2220 (biological), Placebo (other)
- How long the study runs
- Study runs about 32 months (dates as stated)
- About the drug or intervention
- Eplontersen — drug: Eplontersen delivered subcutaneously, once every 4 weeks · ALXN2220 — biological: ALXN2220 delivered intravenously, once every 4 weeks · Placebo — other: Placebo delivered intravenously, once every 4 weeks
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 18 Years to 85 Years
- Who
- All
- Number of participants
- 326
- Started
- 2026-06-11
- Last checked
- 2026-07
Plain English Summary
What is this study?
- • Testing a new treatment for transthyretin amyloid cardiomyopathy (attr-cm)
- • Phase2 - 326 participants
- • The purpose of this randomised, double-blind, placebo-controlled, multicenter study is 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)
Who can take part?
- • Ages 18 Years to 85 Years
- • Diagnosed with transthyretin amyloid cardiomyopathy (attr-cm)
Where?
- • London - Research Site
- • London - Research Site
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The purpose of this randomised, double-blind, placebo-controlled, multicenter study is 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).
More detail
This is a Phase IIb, multicenter, double-blind study in 326 participants, who will be randomized to receive either eplontersen and ALXN2220 or eplontersen and placebo once every four weeks. Participants will also receive daily supplemental doses of the recommended daily allowance of vitamin A.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years - 85 Years
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓Participant must be ≥ 18 years to ≤ 85 years at the time of signing the agreement to take part.
- ✓Participants who have a diagnosis of ATTR-CM with either wild-type or variant TTR genotype based on 1 of the following:
- ✓Endomyocardial biopsy with confirmatory TTR amyloid typing OR
- ✓Grade 2 or 3 heart uptake on 99mTc scintigraphy in the absence of monoclonal gammopathy OR
- ✓NYHA Class I to III at Screening and life expectancy of ≥ 1 year as per the Investigator's judgement.
Who cannot take part
- ✗Known leptomeningeal amyloidosis.
- ✗Known light chain (AL) or secondary (amyloid A) amyloidosis, or any other form of systemic amyloidosis.
- ✗Cardiomyopathy not primarily caused by ATTR-CM, for example, cardiomyopathy primarily due to hypertension, valvular...
- ✗Uncontrolled hypertension (average resting SBP \> 160 mmHg or DBP \> 100 mmHg at Screening).
- ✗Average resting SBP \< 90 mmHg or causing symptoms orthostatic hypotension, despite appropriate treatment, at Screening...
See the full criteria
Where Is This Study? (2 UK sites)
Research Site
London NW10 2PB, United Kingdom
Research Site
London NW3 2QG, United Kingdom
