A Phase 3 Study of ALXN2060 in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM).

JRCT ID: jRCT2071200059

Registered date:04/12/2020

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
• Date of first enrollment: 13/11/2020
• Target sample size: 22
• Study type: Interventional
• Intervention(s): ALXN2060 800 mg, a twice daily (bid) is administered orally

Outcome(s)

Primary Outcome

1.Change from Baseline to Month 12 of treatment in distance walked during the 6MWT
2.All-cause mortality and cardiovascular-related hospitalization over a 30-month period

Secondary Outcome

1.Change from Baseline to Months 30 of treatment in distance walked during the 6MWT
2.Change from Baseline to Months 12 of treatment in KCCQ-OS
3.Change from Baseline to Months 30 of treatment in KCCQ-OS
4.Incidence Of Treatment-emergent Serious Adverse Events (SAEs) And Adverse Events (AEs) [ Time Frame: Baseline through Month 12 ]
5. Incidence Of Treatment-emergent SAEs And AEs [ Time Frame: Baseline through Month 30 ]
6. Change from Baseline To Day 28 in Transthyretin(TTR) stabilization
7. Change from Baseline To Month 30 in Transthyretin(TTR) stabilization

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: < 90age old
• Gender: Both

Include criteria

1. Established diagnosis of ATTR-CM with either wild-type TTR or a variant TTR genotype .
2. History of heart failure evidenced by at least one prior hospitalization for heart failure or clinical evidence of heart failure without prior heart failure hospitalization manifested by signs or symptoms of volume overload or elevated pressures or heart failure symptoms that required or requires ongoing treatment with a diuretics.
3. New York Heart Association Class I-III symptoms due to ATTR-CM.
4. On stable doses of cardiovascular medical therapy.
5. Completed >=150 meters on the 6MWT on 2 tests prior to Day 1.
6. Left ventricular (LV) wall (interventricular septum or LV posterior wall) thickness >=12 mm
7. Biomarkers of myocardial wall stress: N-terminal pro-brain-type natriuretic peptide (NT-proBNP)300 pg/mL

Exclude criteria

1. Acute myocardial infarction, acute coronary syndrome or coronary revascularization, or experienced stroke or transient ischemic attack within 90 days prior to screening.
2. Hemodynamic instability at screening.
3. Likely to undergo heart transplantation within a year of screening.
4. Current treatment with marketed drug products and other investigational agents for the treatment of ATTR-CM.
5. Current treatment with calcium channel blockers with conduction system effects (for example, verapamil, diltiazem). The use of dihydropyridine calcium channel blockers is allowed.
6. Confirmed diagnosis of light-chain (AL) amyloidosis.
7. Biomarkers of myocardial wall stress: NT-ProBNP >= 8,500 pg/mL.
8. Measure of kidney function, estimated glomerular filtration rate by Modification of Diet in Renal Disease formula < 30 mL/minute/1.73 meters squared.

Related Information

• Primary Sponsor: Ishioka Tatsunari
• Secondary ID(s): NCT04622046,