NCT07608354
A Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo for Adults Participants With ATTR-CM
Active, Not Recruiting
PHASE2Ages 18–85InterventionalTreatmentAstraZeneca
~326 participants
Updated 2026-07-14 on ClinicalTrials.gov
What's tested:EplontersenALXN2220Placebo
At a glance
Recruiting sites
0 of 76 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Cardiopulmonary exercise test peak VO2
Measured over 52 week
Conditions
NCT07608354
Where you'd take part
This study runs at 76 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Research Site
La Jolla, Californiano site contact published
Research Site
San Francisco, Californiano site contact published
Research Site
Stanford, Californiano site contact published
Research Site
Aurora, Coloradono site contact published
Research Site
Washington D.C., District of Columbiano site contact published
Research Site
Jacksonville, Floridano site contact published
Research Site
Miami, Floridano site contact published
Research Site
Weston, Floridano site contact published
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
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Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
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:
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:
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).
Exclusion
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.
History of solid organ transplantation or ventricular assist device or listing for heart transplantation at Screening. Note: prior history of planned corneal transplant is not an exclusion criterion.
Suspected or known intolerance/allergy to proteins or any components of the study intervention.
Any of the following results conducted at screening:
Current unstable liver or biliary disease per Investigator's assessment.
Multiple myeloma, lymphoma, leukemia, or any malignancy or clonal stem cell disorder within the past 5 years (except basal cell or squamous epithelial carcinomas of the skin, melanoma in situ or cervical carcinoma in situ that have been curatively resected, Stage I cancer in remission, or adequately treated prostate cancer stage I, IIA, or IIB with Gleason score ≤ 3+4 and prostate-specific antigen \< 20 ng/mL).
Any prior treatment with an ATTR amyloid depleter or a TTR gene silencing agent approved or in clinical development.
Participated in a structured exercise training programme within the 1 month prior to Screening or planned to start during the trial.
Participation in another investigational clinical study or intake of another investigational drug within 30 calendar days or 5 half-lives of the IMP, whichever is longer before signing the ICF.
Judgement by the Investigator that the participant should not participate in the study if the participant has a known medical or psychological condition or other risk factor that might interfere with the participant's full participation in the study, pose any additional risk for the participant, or confound the assessment of the participant or outcome of the study.
Previous enrolment or randomisation in the present study.
What this trial measures
- Cardiopulmonary exercise test peak VO252 week
Change from baseline in Cardiopulmonary exercise test peak VO2 at week 52