Symptomatic Transthyretin Amyloid Cardiomyopathy Clinical Trial
Official title:
A Phase 3, Prospective, Multicenter, Open Label, 2-Part Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN2060 in Japanese Participants With Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
| Verified date | April 2024 |
| Source | Alexion Pharmaceuticals, Inc. |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
This prospective study is designed to evaluate the efficacy, safety, and tolerability of ALXN2060 (also known as AG10), as well as to establish its pharmacokinetic and pharmacodynamic profile in Japanese participants with symptomatic ATTR-CM administered on a background of stable heart failure therapy.
| Status | Active, not recruiting |
| Enrollment | 25 |
| Est. completion date | September 23, 2025 |
| Est. primary completion date | November 8, 2023 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | N/A and older |
| Eligibility | Inclusion 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 1 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 diuretic. 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 millimeters. 7. Biomarkers of myocardial wall stress: N-terminal pro-brain-type natriuretic pep (NT-proBNP) level = 300 picograms/milliliter (pg/mL). Exclusion 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. |
| Country | Name | City | State |
|---|---|---|---|
| Japan | Research Site | Bunkyo-ku | |
| Japan | Research Site | Fukuoka-shi | |
| Japan | Research Site | Kumamoto-shi | |
| Japan | Research Site | Kurume-shi | |
| Japan | Research Site | Matsumoto-shi | |
| Japan | Research Site | Nagoya-shi | |
| Japan | Research Site | Nankoku-shi | |
| Japan | Research Site | Sagamihara-shi | |
| Japan | Research Site | Sapporo-shi | |
| Japan | Research Site | Shinjuku-ku | |
| Japan | Research Site | Suita-shi |
| Lead Sponsor | Collaborator |
|---|---|
| Alexion Pharmaceuticals, Inc. | Eidos Therapeutics, a BridgeBio company |
Japan,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Change From Baseline To Month 12 Of Treatment In Distance Walked During The Six-minute Walk Test (6MWT) | Baseline, Month 12 | ||
| Primary | All-cause Mortality And Cardiovascular-related Hospitalization Over A 30-month Period | Baseline through Month 30 | ||
| Secondary | Change From Baseline To Month 30 Of Treatment In Distance Walked During The 6MWT | Baseline, Month 30 | ||
| Secondary | Change From Baseline To Month 12 Of Treatment In The Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS) | Baseline, Month 12 | ||
| Secondary | Change From Baseline To Month 30 Of Treatment In The KCCQ-OS | Baseline, Month 30 | ||
| Secondary | Incidence Of Treatment-emergent Serious Adverse Events (SAEs) And Adverse Events (AEs) | Baseline through Month 12 | ||
| Secondary | Incidence Of Treatment-emergent SAEs And AEs | Baseline through Month 30 | ||
| Secondary | Change From Baseline To Day 28 In Transthyretin (TTR) Stabilization | Baseline, Day 28 | ||
| Secondary | Change From Baseline To Month 30 In TTR Stabilization | Baseline, Month 30 |