A Study of the Efficacy and Safety of Danicamtiv in Participants with Symptomatic Genetic and Familial Dilated Cardiomyopathy.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Kardigan Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

25 Feb 2026 → ongoing

Decision date (initial)

2026-02-03

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Kardigan, Inc.

External identifiers

EU CT number

2025-522553-19-00

WHO UTN

U1111-1327-3512

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacokinetic, Efficacy, Therapy

Part 1: To assess the effect of danicamtiv on cardiac function using transthoracic echocardiogram (TTE) in Cohort 1.
Part 2: To evaluate the impact of danicamtiv on exercise capacity using cardiopulmonary exercise testing (CPET) in Cohort 1.

Conditions and MedDRA coding

Symptomatic genetic and familial dilated cardiomyopathy.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. 1. Is an adult ≥18 years of age at the Screening visit.
2. 10. Is able to understand and comply with the study procedures, understand the risks involved in the study, and provide written informed consent according to local and institutional guidelines before the first study-specific procedure.
3. 2. Has a diagnosis of dilated cardiomyopathy (DCM) due to probable disease-causing variants of myosin heavy chain 7(MYH7), titin (TTN), or other identified genetic DCM variants, or with familial DCM.
4. 3. Has New York Heart Association (NYHA) Class II-IV at Screening with stable symptoms for ≥1 month.
5. 4. Has chronic DCM, defined as not due to acute or possibly reversible conditions (e.g., hyper/hypothyroidism, infectious cause, tachyarrhythmia, iron overload), according to the Investigator.
6. 5. Has DCM not attributed to substance abuse, amyloidosis, sarcoidosis, or any other secondary form of cardiomyopathy per the Investigator.
7. 6. Has sinus rhythm, stable atrial or ventricular pacing or paroxysmal atrial fibrillation that is adequately rate-controlled when in the arrhythmia, to allow assessments by TTE.
8. 7. Can perform an upright cardiopulmonary exercise testing (CPET) with a peak oxygen consumption (pVO2) of 80% or less of predicted for a healthy individual and RER of ≥1.05 on the Screening CPET, as confirmed by the CPET Core Laboratory.
9. 8. Contraceptive use by participants should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. All participants, if sexually active, must use 1 of the highly effective birth control methods listed in the protocol from the Screening visit through 3 months after the last dose of IMP.

Exclusion criteria 31

1. 1. Has heart failure with reduced ejection (HFrEF) caused primarily by ischemic heart disease, chronic valvulopathy, or another condition, as determined by the Investigator.
2. 18. Has planned cardiac resynchronization therapy (CRT) or major surgery planned in the next 6 months.
3. 19. Has a ventricular assist device or anticipated ventricular assist device placement planned in the next 6 months.
4. 2. Recent (<90 days) acute coronary syndrome or hemodynamically significant epicardial coronary disease, at the discretion of the Investigator.
5. 20. Participated in a clinical trial in which the participant received any investigational drug (or is currently using an investigational device) within 30 days prior to Screening, or less than 5 times the respective elimination half-life (whichever is longer).
6. 21. Serum potassium <3.5 or >5.5 mEq/L at the Screening Visit.
7. 22. Any persistent (2 or more) out-of-range safety laboratory parameters (including chemistry, hematology), considered by the Investigator and Medical Monitor to be clinically significant.
8. 23. Hemoglobin <10g/L at Screening, confirmed on repeat testing if initial result is borderline or deemed not clinically significant by the Investigator.
9. 24. Has active liver disease, defined as any known current infectious, neoplastic, or metabolic, pathology of the liver; unexplained elevations in alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >3 × upper limit of normal (ULN); or total bilirubin (TBL) >2 × ULN at Screening.
10. 25. Thyroid-stimulating hormone >1.5 × ULN at Screening.
11. 26. Serum creatine kinase >3 × ULN at Screening.
12. 10. History of malignancy of any type within 5 years prior to Screening, except for in situ cervical cancer, non-melanomatous skin cancers, ductal carcinoma in situ, and nonmetastatic prostate cancer, which resolved one year prior to screening.
13. 27. Positive for hepatitis B surface antigen or hepatitis C virus (HCV) antibody (anti-HCV) plus HCV-RNA. Participants who are anti-HCV positive but HCV-RNA negative (secondary to treatment or natural viral clearance) are eligible with at least a 1-year period since documented date of RNA polymerase chain reaction negative confirmation following conclusion of treatment.
14. 28. Has hypersensitivity to danicamtiv or any of the components of the danicamtiv formulation.
15. 29. Has life expectancy <6 months.
16. 3. Had coronary revascularization (percutaneous coronary intervention [PCI] or coronary artery bypass graft [CABG]) within prior 90 days.
17. 30. Is pregnant or breastfeeding.
18. 31. Is employed by or is a first-degree relative of someone employed by the Sponsor, the Investigator, or his/her staff or family.
19. 4. Recent (<90 days) hospitalization for heart failure (HF) or use of intravenous (IV) diuretic.
20. 6. Known aortic stenosis of moderate or greater severity, at the discretion of the Investigator.
21. 7. Presence of disqualifying cardiac rhythms that might interfere with reliable echocardiographic measurements of LV function, as determined by the Investigator including: (a) inadequately rate-controlled atrial fibrillation, (b) ectopic beats (atrial, junctional or ventricular) of sufficient frequency (e.g. > 10% of total beats) that the participant's cardiac rhythm is irregular potentially interfering with reliable echocardiographic measurements of LV function.
22. 11. Severe renal insufficiency (defined at the time of Screening as current estimated glomerular filtration rate [eGFR] <15 mL/min/1.73m2 by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation.
23. 8. Unstable or untreated severe ventricular arrythmia (eg, ventricular tachycardia or ventricular fibrillation).
24. 9. Has active infection indicated clinically as determined by the Investigator. In the case of SARS-CoV-2 (COVID-19) infection within 4 weeks prior to and during Screening, symptoms must have completely resolved and based on Investigator assessment in consultation with the Medical Monitor, there are no sequelae that would place the participant at a higher risk if receiving investigational treatment. The methods to assess SARS-CoV-2 (COVID-19) infection include polymerase chain reaction (PCR), antigen test and serology tests.
25. 5. Other recent (<90 days) cardiovascular event (e.g., cerebrovascular accident).
26. 12. History or evidence of any other clinically significant disorder, condition, or disease (including substance abuse) that, in the opinion of the Investigator or Sponsor, would pose a risk to participant safety or interfere with the study evaluation, procedures, completion, or lead to premature withdrawal from the study.
27. 13. Dependent on continuous oxygen supplementation, severe chronic obstructive pulmonary disease (COPD) or restrictive lung disease that may impact CPET performance.
28. 14. Significant hematologic, orthopedic, neuromuscular, or rheumatologic conditions limiting exercise capacity.
29. 15. History of heart transplantation or anticipated heart transplantation in the next 6 months.
30. 16. Any condition deemed by the Investigator to pose an unacceptable risk for CPET or interfere with study assessments.
31. 17. Receives chronic IV inotropic therapy.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Change from Baseline (Day 1) to Week 26 in left atrial function index (LAFI) in Cohort 1.
2. Change from Baseline to Week 26 in peak oxygen consumption (pVO2) in Cohort 1.

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Kardigan Inc.

Sponsor organisation

Kardigan Inc.

Address

131 Oyster Point Boulevard Second Floor

City

South San Francisco

Postcode

94080-2029

Country

United States

Scientific contact point

Organisation

Kardigan Inc.

Contact name

SM Kardigan Medical Information

Public contact point

Organisation

Kardigan Inc.

Contact name

SM Kardigan Medical Information

Third parties 13

Locations

9 EU/EEA countries · 39 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 80 files

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

Application history

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