i-Care; Quantitative-imaging in cardiac transthyretin amyloidosis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Universitair Medisch Centrum Groningen

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Decision date (initial)

2024-09-19

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2024-513310-36-01

EudraCT number

2020-003350-72

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Diagnosis, Others

Quantification of myocardial microcalcification on 18F-sodiumfluoride PET/CT

Mean difference in [123I]MIBG uptake between early and progressive ATTR-CM at baseline and after one years on tafamidis.

Secondary objectives 4

1. Change in myocardial microcalcification activity on 18F-sodiumfluoride PET/CT;
2. Cardiac indices on magnetic resonance imaging, such as left ventricular ejection fraction, left ventricular mass, extracellular volume and global longitudinal strain
3. Cardiac biomarkers including NT-ProBNP and high-sensitivity cardiac troponin I
4. Clinical measures such as 6-minute walk test (6MWT), Kansas City Cardiomyopathy Questionnaire – Overall Summary (KCCQ-OS) score, and Norfolk Quality of Life–Diabetic Neuropathy questionnaire total score (Norfolk QOL‐DN)

Conditions and MedDRA coding

Transthyretin amyloid cardiomyopathy

Regulatory references

Plan to share IPD

No

IPD plan description

No sharing of IPD

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Completion of informed consent
2. Age > 40 years for patients with ATTR or AL cardiac amyloidosis and age >30 years for patients with HCM
3. ATTR cardiac amyloid according to Expert Consensus Recommendations OR AL amyloidosis according to Expert Consensus Recommendations OR Hypertrophic cardiomyopathy according to European Society of Cardiology guidelines

Exclusion criteria 11

1. Inability or unwilling to give informed consent
2. Women who are pregnant, breastfeeding or of child-bearing potential (women who have experienced menarche, are pre-menopausal and have not been sterilised) will not be enrolled into the trial
3. Renal dysfunction (eGFR ≤30 mL/min/1.73m2)
4. NYHA Class IV heart failure
5. Patients with atrial fibrillation and poor rate control
6. Contraindications to MR
7. Previous history of contrast allergy of adverse reactions (gadolinium)
8. Contraindications to tafamidis therapy
9. Only for MIBG-scan: Tricyclic antidepressants (TCA) usage (most importantly amitriptyline, clomipramine, imipramine, nortriptyline, chlorpromazine or perphenazine)
10. Only for MIBG-scan: Parkinson’s disease as mentioned in patients’ medical history
11. Only for MIBG-scan: Insulin dependent diabetes mellitus as shown in patients’ actual medication list

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Quantification of myocardial microcalcification on 18F-sodiumfluoride PET/CT and quantification of cardiac autonomic function on [123I]MIBG imaging.

Secondary endpoints 4

1. Change in myocardial microcalcification activity and myocardial innervation on 18F-sodiumfluoride PET/CT and [123I]-MIBG respectively
2. Cardiac indices on magnetic resonance imaging, such as left ventricular ejection fraction, left ventricular mass, extracellular volume and global longitudinal strain
3. Cardiac biomarkers including NT-ProBNP and high-sensitivity cardiac troponin I
4. Clinical measures, such as 6MWT test, KCCQ-OS and Norfolk QOL‐DN

Investigational products

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

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Universitair Medisch Centrum Groningen

Sponsor organisation

Universitair Medisch Centrum Groningen

Address

Hanzeplein 1

City

Groningen

Postcode

9713 GZ

Country

Netherlands

Scientific contact point

Organisation

Universitair Medisch Centrum Groningen

Contact name

RHJA Slart

Public contact point

Organisation

Universitair Medisch Centrum Groningen

Contact name

RHJA Slart

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Results and documents

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

Documents 7 files

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

Application history

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