What side effects are associated with this type of intervention?

Common treatments for Transthyretin Amyloid Cardiomyopathy (ATTR-CM) include tafamidis, patisiran, and inotersen. Tafamidis is generally well-tolerated but can cause side effects such as urinary tract infections and vaginal infections. Patisiran, an RNAi therapeutic, may lead to infusion-related reactions, peripheral edema, and gastrointestinal symptoms like nausea and diarrhea. Inotersen, another RNA-targeted therapy, has been associated with thrombocytopenia and glomerulonephritis, requiring regular monitoring of platelet counts and renal function.

What prior FDA approvals exist?

The FDA has approved several treatments for Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Tafamidis (Vyndaqel and Vyndamax) is a notable drug that stabilizes the transthyretin protein, preventing it from forming amyloid deposits. Additionally, RNA-targeted therapies like patisiran (Onpattro) and inotersen (Tegsedi) have been approved; these drugs reduce the production of transthyretin by interfering with its synthesis in the liver. These treatments aim to reduce symptoms and slow the progression of heart disease due to TTR amyloidosis, similar to the investigational drug NNC6019-0001.

What other types of research exist?

Promising novel alternatives to NNC6019-0001 for treating transthyretin amyloid cardiomyopathy (ATTR-CM) include: 1. Tafamidis: A transthyretin stabilizer that has been shown to reduce mortality and cardiovascular-related hospitalizations in patients with ATTR-CM. 2. Patisiran: An RNA interference therapy that reduces the production of transthyretin, thereby addressing the underlying cause of amyloidosis. 3. Inotersen: An antisense oligonucleotide that also reduces transthyretin production and has shown benefits in patients with hereditary ATTR amyloidosis. These therapies represent significant advancements in the management of ATTR-CM and offer alternative options for patients considering treatment in 2024.

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NNC6019-0001 for Transthyretin Amyloid Cardiomyopathy

Phase 2

Waitlist Available

Research Sponsored by Novo Nordisk A/S

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Estimated glomerular filtration rate (eGFR) >= 25 milliliter per minute per 1.73 meter square (mL/min/1.73 m^2) at screening

Presently classified as New York Heart Association (NYHA) Class II-III

Must not have

Presence or history of malignant neoplasm within 5 years before screening

Acute coronary syndrome, unstable angina, stroke, transient ischemic attack (TIA), coronary revascularization, cardiac valve repair, or major surgery within 3 months of screening

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline (week 0) to visit 16 (week 64)

Summary

This trial is testing a new medicine, NNC6019-0001, for people with heart disease due to TTR amyloidosis. Participants will receive the medicine by chance, with a higher likelihood of getting the medicine. The goal is to see if it can reduce heart disease symptoms over a period of time.

Who is the study for?

This trial is for adults aged 18-85 with heart disease due to TTR amyloidosis, classified as NYHA Class II-III. They must have a specific diagnosis of ATTR CM, be on stable heart medication for 6 weeks prior, and meet certain lab test criteria. Exclusions include recent major cardiovascular events or surgeries, planned organ transplants, other types of cardiomyopathy, cancer history within 5 years, contrast allergies, and weight over 120 kg.

What is being tested?

The study tests NNC6019-0001's effectiveness in reducing symptoms of heart disease caused by TTR amyloidosis compared to a placebo. Participants are randomly assigned treatment: the chance of receiving NNC6019-0001 is twice that of getting the placebo. The drug is administered via infusion every four weeks for approximately 64 weeks.

What are the potential side effects?

Since NNC6019-0001 is new and not yet approved for use outside trials, potential side effects aren't fully known but may include reactions at the infusion site or general symptoms like fatigue or nausea which are common with infusions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My kidney function, measured by eGFR, is adequate.

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My heart condition limits my physical activity but I can still perform light tasks.

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I walked between 150 and 450 meters in a 6-minute test.

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I have been diagnosed with Transthyretin amyloid cardiomyopathy.

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I am between 18 and 85 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had cancer or was diagnosed with cancer in the last 5 years.

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I haven't had a heart attack, stroke, or major surgery in the last 3 months.

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My heart condition is not mainly due to transthyretin amyloid cardiomyopathy.

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I am scheduled for an organ transplant during the study.

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My weight is over 120 kg.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline (week 0) to visit 16 (week 64)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline (week 0) to visit 16 (week 64)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline (week 0) to visit 16 (week 64) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in 6-minute walk test (6-MWT)

Change in N-terminal-pro brain natriuretic peptide (NT-proBNP)

Secondary study objectives

Change in Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score (CSS)

Change in global longitudinal strain (GLS) on echocardiography

Change in myocardial extracellular volume (ECV)

+5 more

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: NNC6019-0001, Dose 2Experimental Treatment1 Intervention

Participants will receive dose 2 i.v. infusion of NNC6019-0001 Q4W added to standard of care until week 52.

Group II: NNC6019-0001, Dose 1Experimental Treatment1 Intervention

Participants will receive dose 1 intravenous (i.v.) infusion of NNC6019-0001 every 4 weeks (Q4W) added to standard of care until week 52.

Group III: PlaceboPlacebo Group1 Intervention

Participants will receive i.v. infusion of placebo (NNC6019-0001) Q4W added to standard of care until week 52.

0 / 10Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Transthyretin Amyloid Cardiomyopathy (ATTR-CM) include stabilizers of the transthyretin (TTR) protein, which prevent it from misfolding and forming amyloid deposits, and agents that reduce TTR production, thereby decreasing the amount of TTR available to form amyloid. Investigational agents like NNC6019-0001 are being studied for their potential to reduce symptoms of heart disease caused by TTR amyloidosis. These treatments are significant for ATTR-CM patients as they target the root cause of the disease, potentially improving heart function and quality of life.

Cardiac amyloidosis: an update on diagnosis, current therapy, and future directions.[The Changing Appearance of Cardiac Amyloidosis].Advances in the diagnosis and treatment of transthyretin amyloidosis with cardiac involvement.