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A study to determine if ALXN2220 is effective, safe, and tolerable in adults with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
EU CTIS ID: 2023-506669-70-00
What this study is testing
To assess the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo groups as assessed by the total occurrences of ACM and CV clinical events.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male or female ≥ 18 years to ≤ 90 years of age at time of randomization.
- If treated with locally approved TTR gene silencing agent for ATTR amyloidosis (except as listed in Section 6.9.2 of the protocol), must be on a stable dose for at least 90 calendar days prior to Screening
- Male and/or female (according to their reproductive organs and functions assigned by chromosomal complement). Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Female participant is eligible to participate if she is not pregnant or breastfeeding, and is a woman of nonchildbearing potential or is a WOCBP and using a highly effective contraceptive method, and agrees not to donate eggs. Male participants are eligible to participate if they agree to refrain from donating fresh unwashed semen plus abstinent from heterosexual intercourse or must agree to use contraception/barrier,
- Diagnosis of ATTR-CM with either wild-type or a variant TTR genotype based on one of the following: a. Endomyocardial biopsy with confirmatory TTR amyloid typing by either immunohistochemistry or mass spectrometry; OR b. Grade 2 or 3 cardiac uptake on 99mTc scintigraphy (99mTc DPD, 99mTc PYP, or 99mTc HMDP) in the absence of monoclonal gammopathy; OR c. Grade 2 or 3 cardiac uptake on 99mTc scintigraphy (99mTc DPD, 99mTc PYP, or 99mTc HMDP) AND confirmatory TTR amyloid typing by either immunohistochemistry or mass spectrometry in tissue biopsy in the presence of monoclonal gammopathy
- End-diastolic interventricular septal wall thickness ≥ 12 mm on echocardiography measured at Screening
- NT-proBNP > 2000 pg/mL measured by a central laboratory at Screening
You likely can't join if
- Known leptomeningeal amyloidosis
- Participants with renal failure requiring dialysis or who have an eGFR by CKD-Epi formula < 20 mL/min/1.73 m2 measured by a central laboratory at Screening
- Polyneuropathy secondary to ATTR amyloidosis requiring a wheelchair (ie, PND score IV)
- Known light chain (AL) or secondary amyloidosis (AA), or any other form of systemic amyloidosis
- Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
- Uncontrolled clinically significant cardiac arrhythmia, per Investigator's assessment.
See the full eligibility criteria
Who can join
- Male or female ≥ 18 years to ≤ 90 years of age at time of randomization.
- If treated with locally approved TTR gene silencing agent for ATTR amyloidosis (except as listed in Section 6.9.2 of the protocol), must be on a stable dose for at least 90 calendar days prior to Screening
- Male and/or female (according to their reproductive organs and functions assigned by chromosomal complement). Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Female participant is eligible to participate if she is not pregnant or breastfeeding, and is a woman of nonchildbearing potential or is a WOCBP and using a highly effective contraceptive method, and agrees not to donate eggs. Male participants are eligible to participate if they agree to refrain from donating fresh unwashed semen plus abstinent from heterosexual intercourse or must agree to use contraception/barrier,
- Diagnosis of ATTR-CM with either wild-type or a variant TTR genotype based on one of the following: a. Endomyocardial biopsy with confirmatory TTR amyloid typing by either immunohistochemistry or mass spectrometry; OR b. Grade 2 or 3 cardiac uptake on 99mTc scintigraphy (99mTc DPD, 99mTc PYP, or 99mTc HMDP) in the absence of monoclonal gammopathy; OR c. Grade 2 or 3 cardiac uptake on 99mTc scintigraphy (99mTc DPD, 99mTc PYP, or 99mTc HMDP) AND confirmatory TTR amyloid typing by either immunohistochemistry or mass spectrometry in tissue biopsy in the presence of monoclonal gammopathy
- End-diastolic interventricular septal wall thickness ≥ 12 mm on echocardiography measured at Screening
- NT-proBNP > 2000 pg/mL measured by a central laboratory at Screening
- Treatment with a loop diuretic for at least 30 days prior to Screening
- History of heart failure as documented by one of the following events within 1 year prior to Screening: a. heart failure hospitalization b. urgent heart failure visit c. episode of volume overload documented by NT-proBNP > 2000 pg/mL (or equivalent BNP)
- NYHA Class II-IV at Screening
- Life expectancy of ≥ 6 months as per the Investigator’s judgment
What rules you out
- Known leptomeningeal amyloidosis
- Participants with renal failure requiring dialysis or who have an eGFR by CKD-Epi formula < 20 mL/min/1.73 m2 measured by a central laboratory at Screening
- Polyneuropathy secondary to ATTR amyloidosis requiring a wheelchair (ie, PND score IV)
- Known light chain (AL) or secondary amyloidosis (AA), or any other form of systemic amyloidosis
- Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
- Uncontrolled clinically significant cardiac arrhythmia, per Investigator's assessment.
- LVEF < 30% on echocardiography
The study team makes the final eligibility decision.
Where it's taking place
- Israel
- Korea, Republic of
- Taiwan
- Brazil
- Australia
- United States
- Turkey
- Argentina
- United Kingdom
- China
- Japan
- Switzerland
- Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Israel; Korea, Republic of; Taiwan; Brazil; Australia; United States and 7 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.