Efficacy and safety of NNC6019-0001 at two dose levels in participants with transthyretin amyloid cardiomyopathy (ATTR CM)(NN6019-4940)

JRCT ID: jRCT2061230027

Registered date:21/06/2023

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: transthyretin amyloid cardiomyopathy (hATTR CM or wtATTR CM)
• Date of first enrollment: 13/07/2023
• Target sample size: 9
• Countries of recruitment: United states,Japan,Canada,Japan,Czech Republic,Japan,France,Japan,Germany,Japan,Italy,Japan,Netherlands,Japan,Portugal,Japan,Spain,Japan
• Study type: Interventional
• Intervention(s): To administer i.v. 10 mg/kg NNC6019-0001, 60 mg/kg NNC6019-0001 or placebo Q4W added to standard of care

Outcome(s)

Primary Outcome

-To compare the effect of two dose levels of NNC6019-0001 (10 mg/kg and 60 mg/kg) versus placebo on:
change in 6-minute walk test and
-change in NT-proBNP from baseline to week 52 in participants with hATTR or wtATTR cardiomyopathy.

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: < 85age old
• Gender: Both

Include criteria

-Male or female.
-Age >=18 to < 85 years at the time of signing informed consent.
-Have an established diagnosis of ATTR CM with either wild-type TTR or hereditary TTR genotype as per local standards.
-Expected to be on stable doses of cardiovascular medical therapy 6 weeks prior to the randomisation visit.
-Known end-diastolic interventricular septal wall thickness >= 12 mm.
-Presently classified as New York Heart Association (NYHA) Class II-III.
-NT-proBNP concentration >=650 pg/mL in sinus cardiac rhythm and >1000 pg/mL in atrial fibrillation at screening.
-Completed >=150 meters to <=450 meters on the 6MWT at screening.
-Estimated glomerular filtration rate (eGFR) >=25 mL/min/1.73 m^2 at screening.

Exclude criteria

-Cardiomyopathy not primarily caused by ATTR CM, for example, cardiomyopathy due to hypertension, valvular heart disease, or ischemic heart disease
-A prior solid organ transplant.
-Planned solid organ transplant during the study.
-Presence or history of malignant neoplasm (other than basal or squamous cell skin cancer, in-situ carcinomas of the cervix, or in-situ/high grade prostatic intraepithelial neoplasia (PIN) or low-grade prostate cancer) within 5 years before screening.
-Current treatment with calcium channel blockers with conduction system effects (e.g., verapamil, diltiazem). The use of dihydropyridine calcium channel blockers is allowed. The use of digoxin will only be allowed if required for management of atrial fibrillation with rapid ventricular response.
-Acute coronary syndrome, unstable angina, stroke, transient ischemic attack (TIA), coronary revascularization, cardiac valve repair, or major surgery within 3 months of screening.
-Body weight >120 kg (264.6 lb) at screening.
-History of contrast allergy or adverse reactions to gadolinium-containing agents.

Related Information

• Primary Sponsor: Ito Junji
• Secondary ID(s): NCT05442047