A study to learn more about the change in the blood levels of transthyretin when participants with transthyretin amyloidosis with cardiomyopathy switched from tafamidis to acoramidis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Bayer Consumer Care AG

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

12 Feb 2026 → ongoing

Decision date (initial)

2026-02-07

Transition trial

No

Low-intervention

Yes

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Bayer Consumer Care AG

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic

To evaluate the change of serum TTR level associated with acoramidis treatment in patients with ATTR-CM switching from tafamidis.

Secondary objectives 5

1. To characterize the course of serum TTR level associated with acoramidis treatment in patients with ATTR-CM switching from tafamidis
2. To evaluate overall reported safety in patients with ATTR-CM switching from tafamidis to acoramidis.
3. To evaluate biomarkers and functional capacity in patients with ATTR-CM switching from tafamidis to acoramidis.
4. To evaluate the course of laboratory parameters related to kidney and thyroid function in patients with ATTR-CM switching from tafamidis to acoramidis.
5. To evaluate the course of quality of life in patients with ATTR-CM switching from tafamidis to acoramidis.

Conditions and MedDRA coding

Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Participant must be 18 to 90 years of age inclusive, at the time of signing the informed consent.
2. Diagnosis of ATTR-CM; disease defining examination, i.e., SPECT or SPECT/CT or biopsy, within 24 months prior to V1
3. Participants must currently be treated with tafamidis and have used tafamidis for at least the previous 3 months prior to V1 and have adhered to tafamidis therapy
4. NYHA class ≤ II at V1
5. eGFR ≥ 30 mL/min/1.73m2 at V1
6. NT-proBNP > 300 and ≤ 7000 pg/mL at V1

Exclusion criteria 16

1. Prior liver or heart transplantation or planned within the next 12 months
2. Current or planned use of ventricular assist device
3. Active cancer or other disease that decreases the life expectancy to less than one year
4. Heart failure due to ischemic heart disease
5. Myocardial infarction, CV surgery, or unstable angina within the last 90 days prior to V1
6. Confirmed diagnosis of light-chain amyloidosis
7. Dialysis or severe renal impairment as reflected by UACR > 300 mg/g at V1
8. Major surgery 90 days prior to V1
9. Recent initiation of SGLT2i within 3 months before V1
10. Initiation of treatment with a diuretic or increase in diuretic dose within 3 months before V1
11. Treatment with calcium channel blockers (e.g., verapamil, diltiazem) or digitalis required
12. Recent CV hospitalization within 3 months before V1
13. Known hypersensitivity to acoramidis or to any of the excipients
14. A condition that, as judged by the investigator, would preclude compliance with the study protocol, such as a history of substance abuse, alcoholism, or a psychiatric condition
15. Known or suspected liver disorder and bile secretion/flow (cholestasis, also history of it)
16. Abnormal liver function tests at V1, defined as ALT (GPT) or AST (GOT) ≥ 3 x ULN or total bilirubin ≥ 3 x ULN at V1

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change in serum TTR level from baseline to month 6 or premature discontinuation of treatment

Secondary endpoints 7

1. Serum TTR level at baseline, week 1, 2, 3, 4 and at month 3.
2. Occurrence of TEAEs and type of TEAEs and serious TEAEs.
3. Change from baseline to month 6 of NT-proBNP, hs-TnT, hs-CRP, RBP4.
4. Signs of remodeling from baseline to month 6 in ECHO.
5. Change from baseline to month 6 in 6MWD
6. Change from baseline to month 6 in kidney and thyroid biomarkers: eGFR, Creatinine, Cystatin C, UACR, and TSH.
7. Change from baseline to month 6 in KCCQ-OS score and in EQ-5D-5L index score.

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Bayer Consumer Care AG

Sponsor organisation

Bayer Consumer Care AG

Address

Peter Merian-Strasse 84

City

Basel

Postcode

4052

Country

Switzerland

Scientific contact point

Organisation

Bayer Consumer Care AG

Contact name

Chief Medical officer

Public contact point

Organisation

Bayer Consumer Care AG

Contact name

Chief Medical officer

Third parties 1

Locations

4 EU/EEA countries · 19 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 30 files

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

Application history

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