A Research Study to Look at How a New Medicine Called NNC6019-0001 Works and How Safe it is for People Who Have Heart Disease Due to Transthyretin (TTR) Amyloidosis

NCT ID: NCT05442047

Last Updated: 2025-11-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

105 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-08-02

Study Completion Date

2025-05-13

Brief Summary

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This study is testing a potential new medicine, NNC6019-0001, for people who have a heart disease due to TTR amyloidosis.The study will look at if this medicine can reduce the symptoms of a heart disease due to TTR amyloidosis, such as heart failure. Participants will either get NNC6019-0001 (apotential new medicine) or placebo (a medicine which has no effect on the body). Which treatment participants get is decided by chance. The chance of getting NNC6019-0001 is two times higher than getting placebo. NNC6019-0001 is not yet approved in any country or region in the world. It is a new medicine that doctors cannot prescribe yet. Participants will get an infusion of the study medicine 13 times, once every 4 weeks. The study will last for about 64 weeks after the first dose of medicine. Participants cannot participate in this study if they have a heart disease other than a heart disease due to TTR amyloidosis.

Detailed Description

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Conditions

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Transthyretin Amyloid Cardiomyopathy (ATTR CM)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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NNC6019-0001, Dose 1

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 Type EXPERIMENTAL

NNC6019-0001

Intervention Type DRUG

Participants will receive i.v infusionof NNC6019-0001.

NNC6019-0001, Dose 2

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

Group Type EXPERIMENTAL

NNC6019-0001

Intervention Type DRUG

Participants will receive i.v infusionof NNC6019-0001.

Placebo

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

Group Type PLACEBO_COMPARATOR

Placebo (NNC6019-0001)

Intervention Type DRUG

Participants will receive i.v. infusion of placebo (NNC6019-0001).

Interventions

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NNC6019-0001

Participants will receive i.v infusionof NNC6019-0001.

Intervention Type DRUG

Placebo (NNC6019-0001)

Participants will receive i.v. infusion of placebo (NNC6019-0001).

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Male or female.
* Age greater than or equal to (\>=) 18 to less than (\<) 85 years at the time of signing informed consent.
* Have an established diagnosis of Transthyretin amyloid cardiomyopathy (ATTR CM) with either wild-type transhyretin (TTR) or hereditary transthyretin (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 greater than or equal to (\>=) 12 millimeters (mm).
* Presently classified as New York Heart Association (NYHA) Class II-III.
* N-terminal-pro brain natriuretic peptide (NT-proBNP) concentration greater than or equal to (\>=) 650 picograms per milliliter (pg/mL) in sinus cardiac rhythm and greater than (\>) 1000 pg/mL in atrial fibrillation at screening.
* Completed greater than or equal to (\>=) 150 meters to less than or equal to (\<=) 450 meters on the 6-minute walk test (MWT) at screening.
* Estimated glomerular filtration rate (eGFR) greater than or equal to (\>=) 25 milliliter per minute per 1.73 meter square (mL/min/1.73 m\^2) at screening.

Exclusion Criteria

* Cardiomyopathy not primarily caused by transthyretin amyloid cardiomyopathy transthyretin amyloid cardiomyopathy (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, insitu 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 (example \[e.g.\], 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 kilogram (kg) (264.6 pounds \[lb\]) at screening.
* History of contrast allergy or adverse reactions to gadolinium-containing agents.
Minimum Eligible Age

18 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novo Nordisk A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Transparency 2834

Role: STUDY_DIRECTOR

Novo Nordisk A/S

Locations

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Mayo Clinic Arizona

Phoenix, Arizona, United States

Site Status

Cedars-Sinai Medical Center_Los Angeles

Beverly Hills, California, United States

Site Status

Stanford Hlth Cre-Boswell Clin

Stanford, California, United States

Site Status

Mayo Clinic Jacksonville

Jacksonville, Florida, United States

Site Status

NW Univ-Bluhm Cardiovasc Inst

Evanston, Illinois, United States

Site Status

Univ of MD Schl of Med

Baltimore, Maryland, United States

Site Status

University of Maryland School of Medicine

Baltimore, Maryland, United States

Site Status

Mayo Clinic Rochester

Rochester, Minnesota, United States

Site Status

Oregon Hlth Sci Univ-Portland

Portland, Oregon, United States

Site Status

University of Calgary_Cardiology

Calgary, Alberta, Canada

Site Status

Ctr for Cardiovascular Innovation

Vancouver, British Columbia, Canada

Site Status

II. interni klinika VFN - Kardiologie a angiologie

Prague, , Czechia

Site Status

Ap-Hp-Hopital Henri Mondor

Créteil, , France

Site Status

Centre Hospitalier Universitaire de Toulouse-Hopital Rangueil-1

Toulouse, , France

Site Status

Centre Hospitalier Universitaire de Toulouse-Hopital Rangueil-1

Toulouse Cedex 9 TSA 50032, , France

Site Status

UniklinikHeidelberg - Innere Med. III - Kardiologie, Angiologie, Pneumologie

Heidelberg, , Germany

Site Status

LMU Klinikum München Klinik und Poliklinik 1

München, , Germany

Site Status

Universitätsklinikum Münster - Klinik für Kardiologie I

Münster, , Germany

Site Status

Universitatsklinikum Wurzburg AöR

Würzburg, , Germany

Site Status

Universitatsklinikum Würzburg - Zentrum für Herzinsuffizienz

Würzburg, , Germany

Site Status

Fondazione Toscana Gabriele Monasterio - Dipartimento Cardiotoracico - UOC Cardiologia e Medicina Cardiovascolare

Pisa, Pi, Italy

Site Status

Centro per lo Studio e la Cura delle Amiloidosi Sistemiche Fondazione IRCCS Policlinico San Matteo

Pavia, PV, Italy

Site Status

Hospital of the University of Occupational and Environmental Health, Cardiology, Nephrology

Fukuoka, , Japan

Site Status

Hiroshima University hospital, Cardiovascular Medicine

Hiroshima, , Japan

Site Status

Kumamoto University Hospital, Cardiovascular Medicine

Kumamoto-shi, Kumamoto, , Japan

Site Status

Shinshu University Hospital, Department of Neurology

Nagano, , Japan

Site Status

Nagasaki University Hospital, Cardiovascular Medicine

Nagasaki, , Japan

Site Status

Okayama University Hospital_Cardiovascular Medicine

Okayama-shi, Okayama, , Japan

Site Status

UMC Groningen

Groningen, , Netherlands

Site Status

Universitair Medisch Centrum Utrecht

Utrecht, , Netherlands

Site Status

Hospital da Senhora da Oliveira - Guimarães

Guimarães, , Portugal

Site Status

Unidade Local de Saude do Alto Ave, E.P.E.

Guimarães, , Portugal

Site Status

Unidade Local De Saude De Tras-Os-Montes E Alto Douro E.P.E.

Vila Real, , Portugal

Site Status

Hospital Universitario Puerta de Hierro Majadahonda

Majadahonda, Madrid, Spain

Site Status

Hospital Universitario Puerta de Hierro Majadahonda

Madrid, , Spain

Site Status

Countries

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United States Canada Czechia France Germany Italy Japan Netherlands Portugal Spain

References

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Suhr OB, Grogan M, Silva AMD, Karam C, Garcia-Pavia P, Drachman B, Zago W, Tripuraneni R, Kinney GG. PRX004 in variant amyloid transthyretin (ATTRv) amyloidosis: results of a phase 1, open-label, dose-escalation study. Amyloid. 2025 Mar;32(1):14-21. doi: 10.1080/13506129.2024.2420809. Epub 2024 Oct 29.

Reference Type DERIVED
PMID: 39472768 (View on PubMed)

Other Identifiers

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U1111-1271-3861

Identifier Type: OTHER

Identifier Source: secondary_id

2021-006226-49

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

NN6019-4940

Identifier Type: -

Identifier Source: org_study_id

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