A research study to look at long-term treatment with a medicine called NNC6019-0001 for people who have heart failure due to transthyretin amyloidosis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novo Nordisk A/S

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

16 Sep 2024 → ongoing

Decision date (initial)

2024-05-30

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Novo Nordisk A/S

External identifiers

EU CT number

2022-502605-15-00

WHO UTN

U1111-1284-5820

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To assess the long-term safety and tolerability of NNC6019-0001 from baseline up to week 156 in participants with ATTRv-CM or ATTRwt-CM

Secondary objectives 1

1. To assess the long-term effect of NNC6019-0001 on: •Functional test •Participant-reported QoL •Biomarkers •Imaging (echocardiography and cardiac MRI) from baseline to week 104 in participants with ATTRv-CM or ATTRwt-CM

Conditions and MedDRA coding

Participants with transthyretin amyloid cardiomyopathy (ATTR CM)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Informed consent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
2. Completed study intervention in NN6019-4940 (a) and attended the last study visit (week 64; visit 16), and no later than 12 weeks after visit 16. (a)- A participant who has not permanently discontinued study intervention as defined in NN6019-4940
3. Expected to be on stable doses of cardiovascular medical therapy 6 weeks prior to the enrolment visit.

Exclusion criteria 12

1. Known or suspected hypersensitivity to study intervention(s) or related products.
2. Previous enrolment in this study.
3. Female who is pregnant, breast-feeding or intends to become pregnant or is of childbearing potential and not using highly effective contraceptive method, as defined in Appendix 4 (Section ‎10.4). Spain: For country-specific requirements, please refer to Appendix 8 (Section ‎10.8).
4. Use of another approved or non-approved investigational medicinal product within 30 days or 5 half-lives of the investigational medicinal product (whichever is longer) before screening, except study intervention used in study NN6019-4940
5. Participation (i.e., signed informed consent) in any interventional clinical study from visit 16 (week 64) in NN6019-4940 until visit 1 (screening) in the current study NN6019-7565.
6. Any disorder, which in the investigator’s opinion might jeopardise participant’s safety or compliance with the protocol.
7. A prior solid organ transplant.
8. 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 or low-grade prostate cancer) within 5 years before screening.
9. 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.
10. Aspartate transaminase (AST) or alanine transaminase (ALT) levels ≥ 2.5 × the upper limit of normal (ULN) or total bilirubin ≥ 2 × ULN at screening.
11. Body weight > 120 kg (264.6 lb) at screening.
12. International normalised ratio (INR)>1.5 (unless participant is on anticoagulant therapy, in which case excluded if INR>3.5).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Number of treatment emergent adverse events from baseline (week 0) up to visit 39 (week 156)

Secondary endpoints 6

1. Change in 6-minute walk test (6MWT) from baseline (week 0) to visit 28 (week 104)
2. Change in NT-proBNP from baseline (week 0) to visit 28 (week 104)
3. Change in myocardial extracellular volume from baseline (week 0) to visit 28 (week 104)
4. Change in KCCQ-(CSS)2 from baseline (week 0) to visit 28 (week 104)
5. Change in troponin I from baseline (week 0) to visit 28 (week 104)
6. Change in GLS on echocardiography from baseline (week 0) to visit 28 (week 104)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Auxiliary 4

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novo Nordisk A/S

Sponsor organisation

Novo Nordisk A/S

Address

Novo Alle 1

City

Bagsvaerd

Postcode

2880

Country

Denmark

Scientific contact point

Organisation

Novo Nordisk A/S

Contact name

EU Submission Hub

Public contact point

Organisation

Novo Nordisk A/S

Contact name

EU Submission Hub

Third parties 6

Locations

7 EU/EEA countries · 13 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 77 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

11 submissions — initial application plus substantial / non-substantial modifications