NCT07608354

Brief Summary

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).

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
326

participants targeted

Target at P75+ for phase_2

Timeline
32mo left

Started Jun 2026

Geographic Reach
10 countries

78 active sites

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 12, 2026

Completed
15 days until next milestone

First Posted

Study publicly available on registry

May 27, 2026

Completed
1 month until next milestone

Study Start

First participant enrolled

June 30, 2026

Expected
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 23, 2029

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 23, 2029

Last Updated

June 10, 2026

Status Verified

June 1, 2026

Enrollment Period

2.7 years

First QC Date

May 12, 2026

Last Update Submit

June 9, 2026

Conditions

Keywords

Transthyretin amyloid cardiomyopathyATTR-CMEplontersenALXN2220

Outcome Measures

Primary Outcomes (1)

  • Cardiopulmonary exercise test peak VO2

    Change from baseline in Cardiopulmonary exercise test peak VO2 at week 52

    52 week

Secondary Outcomes (3)

  • Extracellular volume

    52 week

  • NT-proBNP

    52 week

  • Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score

    52 week

Study Arms (2)

Eplontersen and ALXN2220

EXPERIMENTAL

Subcutaneous eplontersen and intravenous ALXN2220 every 4 weeks

Drug: EplontersenBiological: ALXN2220

Eplontersen and placebo

PLACEBO COMPARATOR

Subcutaneous eplontersen and intravenous placebo every 4 weeks

Drug: EplontersenOther: Placebo

Interventions

Eplontersen delivered subcutaneously, once every 4 weeks

Eplontersen and ALXN2220Eplontersen and placebo
ALXN2220BIOLOGICAL

ALXN2220 delivered intravenously, once every 4 weeks

Eplontersen and ALXN2220
PlaceboOTHER

Placebo delivered intravenously, once every 4 weeks

Eplontersen and placebo

Eligibility Criteria

Age18 Years - 85 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Participant must be ≥ 18 years to ≤ 85 years at the time of signing the informed consent.
  • 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 cardiac uptake on 99mTc scintigraphy in the absence of monoclonal gammopathy OR
  • Grade 2 or 3 cardiac uptake on 99mTc scintigraphy AND confirmatory TTR amyloid typing in the presence of monoclonal gammopathy.
  • NYHA Class I to III at Screening and life expectancy of ≥ 1 year as per the Investigator's judgement.
  • End-diastolic IVST ≥ 12 mm on echocardiography.
  • NT-proBNP ≥ 600pg/mL for participants without ongoing atrial fibrillation/flutter at Screening or NT-proBNP ≥ 1200pg/mL for participants with ongoing atrial fibrillation/flutter at Screening.
  • Able to complete symptom-limited maximal CPET at Screening based on the following test criteria:
  • Able to exercise to near exhaustion during CPET as exhibited by RER ≥ 1.0 during symptom-limited CPET conducted during screening.
  • If participant does not achieve RER ≥1.0, the CPET may be repeated once, at least 48 hours but less than 2 weeks (but before randomization) after the initial test.
  • Treated according to locally recognised guidelines on standard-of-care treatment for patients with HF. Therapy should have been individually optimised and stable for ≥ 4 weeks (except diuretics) and include, unless contraindicated or not tolerated, treatment of high BP (targeting SBP \< 130 mmHg as suggested in 2022 American College of Cardiology/American Heart Association/Heart Failure Society of America HF guidelines), and ischaemic heart disease.
  • Willingness to adhere to daily self-administered vitamin A supplementation (3000 IU).

You may not qualify if:

  • 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 heart disease, or ischaemic heart disease per Investigator's assessment.
  • Acute coronary syndrome, unstable angina, stroke, transient ischaemic attack, coronary revascularisation, cardiac device implantation, cardiac valve repair, or major surgery within 12 weeks of Screening.
  • Uncontrolled hypertension (average resting SBP \> 160 mmHg or DBP \> 100 mmHg at Screening).
  • Average resting SBP \< 90 mmHg or symptomatic orthostatic hypotension, despite appropriate treatment, at Screening per Investigator's assessment.
  • Uncontrolled ventricular clinically significant cardiac arrhythmia, per Investigator's assessment.
  • Left ventricular ejection fraction \< 30% on echocardiography measured locally at Screening.
  • Severe pulmonary impairment (SpO₂ \< 92%) defined as resting SpO₂ below 92% on room air, measured by pulse oximetry, indicative of severe lung disease. Participants requiring supplemental oxygen to maintain SpO₂ ≥ 92%.
  • Participants with renal failure requiring dialysis.
  • Suspected or known intolerance/allergy to proteins or any components of the study intervention.
  • Any of the following results conducted at screening:
  • i) Haemoglobin \<8g/dL for women or \<9g/dL for men. ii) Platelet count \<125 X10\*9/L or other disorder associated with clinically significant thrombocytopenia.
  • iii) ALT \>2.0 X ULN iv) TBL \>2.5 X ULN (participants with known Gilbert's syndrome can be included with TBL \>2.5 X ULN as long as direct bilirubin is ≤ 1.5 X ULN) v) Serum retinol level \< LLN vi) By CKD-EPI formula, eGFR \<20 mL/min/1.73 m2 measured by the central laboratory at Screening.
  • Current unstable liver or biliary disease per Investigator's assessment.
  • +6 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (78)

Research Site

La Jolla, California, 92037, United States

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San Francisco, California, 94115, United States

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Stanford, California, 94305, United States

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Aurora, Colorado, 80045, United States

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Washington D.C., District of Columbia, 20010, United States

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Jacksonville, Florida, 32224, United States

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Miami, Florida, 33176, United States

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Weston, Florida, 33331, United States

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Chicago, Illinois, 60637, United States

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Boston, Massachusetts, 02115, United States

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Kansas City, Missouri, 64111, United States

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St Louis, Missouri, 63110, United States

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New York, New York, 10032, United States

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Chapel Hill, North Carolina, 27599, United States

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Durham, North Carolina, 27710, United States

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Cleveland, Ohio, 44195, United States

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Portland, Oregon, 97239, United States

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Danville, Pennsylvania, 17822, United States

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Philadelphia, Pennsylvania, 19104, United States

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Germantown, Tennessee, 38138, United States

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Dallas, Texas, 75390, United States

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Houston, Texas, 77030, United States

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Salt Lake City, Utah, 84132, United States

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Falls Church, Virginia, 22042, United States

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Calgary, Alberta, T2N 2T9, Canada

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Vancouver, British Columbia, V6Z 1Y6, Canada

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Halifax, Nova Scotia, B3H 3A7, Canada

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London, Ontario, N6C 2R5, Canada

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Toronto, Ontario, M5G 2C4, Canada

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Montreal, Quebec, H1T 1C8, Canada

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Beijing, 100034, China

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Beijing, 100730, China

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Changsha, 410012, China

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Chongqing, 400042, China

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Guangzhou, 510080, China

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Hangzhou, 310009, China

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Créteil, 94010, France

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Marseille, 13005, France

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Rennes, 35033, France

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Toulouse, 31059, France

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Bad Krozingen, 79189, Germany

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Berlin, 10117, Germany

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Cologne, 50937, Germany

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Essen, 45147, Germany

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Hamburg, 22767, Germany

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Heidelberg, 69120, Germany

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Homburg, 66421, Germany

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München, 81377, Germany

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Münster, 48149, Germany

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Bologna, 40138, Italy

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Florence, 50134, Italy

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Milan, 20138, Italy

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Padova, 35128, Italy

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Pavia, 27100, Italy

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Pisa, 56124, Italy

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Torino, 10154, Italy

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Torrette - Ancona, 60126, Italy

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Trieste, IT-34149, Italy

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Bunkyō City, 113-8431, Japan

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Kurume-shi, 830-0011, Japan

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Sapporo, 060-8543, Japan

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Shinjuku-ku, 160-8582, Japan

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Suita, 565-8565, Japan

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Barcelona, 08035, Spain

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Jaén, 23007, Spain

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L'Hospitalet de Llobregat, 08907, Spain

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Majadahonda, 28222, Spain

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Málaga, 29010, Spain

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Pamplona, 31008, Spain

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Salamanca, 37007, Spain

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Valencia, 46010, Spain

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Gothenburg, 413 45, Sweden

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Lund, 22242, Sweden

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Stockholm, 171 64, Sweden

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Umeå, 90737, Sweden

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Uppsala, 751 85, Sweden

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London, NW10 2PB, United Kingdom

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London, NW3 2QG, United Kingdom

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MeSH Terms

Interventions

eplontersen

Central Study Contacts

AstraZeneca Clinical Study Information Center

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 12, 2026

First Posted

May 27, 2026

Study Start (Estimated)

June 30, 2026

Primary Completion (Estimated)

February 23, 2029

Study Completion (Estimated)

February 23, 2029

Last Updated

June 10, 2026

Record last verified: 2026-06

Data Sharing

IPD Sharing
Will share

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

Shared Documents
STUDY PROTOCOL, SAP
Time Frame
AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA Pharma Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
Access Criteria
When a request has been approved AstraZeneca will provide access to the de-identified individual patient-level data in an approved sponsored tool . Signed Data Sharing Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information. Additionally, all users will need to accept the terms and conditions of the SAS MSE to gain access. For additional details, please review the Disclosure Statements at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
More information

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