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

NCT07210723 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: DAN-301
• Study Type: interventional
• Target: 332
• Locations: 8 countries
• Sites: 33

Brief Summary

The Sponsor is studying an investigational medication called danicamtiv to determine if it can help people with genetic and familial dilated cardiomyopathy (DCM). Investigational means that the safety and effectiveness of danicamtiv have not been established. Currently, there are no approved drugs that are designed specifically to treat genetic or familial DCM. The purpose of this study is to evaluate how well danicamtiv works compared to a placebo (sugar pill that looks like danicamtiv pill but does not contain any danicamtiv) and see how safe it is for people with genetic and familial DCM. In DCM, the heart muscle weakens and enlarges, making it harder for the heart to pump blood; this can happen for different reasons. Some people have DCM because of a change in a gene (called genetic DCM). Others may have DCM that runs in their family, even if no specific gene change is found (called familial DCM). The main goals of the study are:

• To assess the effect of danicamtiv on cardiac function using echocardiogram.
• To evaluate the impact of danicamtiv on exercise capacity
• To evaluate the safety and tolerability of danicamtiv Participants will:
• Take danicamtiv or placebo every day for approximately 6 months
• Visit the clinic about 12 times for initial evaluation, checkups, tests and follow up

Conditions

Symptomatic Genetic Dilated Cardiomyopathy

Outcome Measures

Primary Outcomes (2)

• Part 1: Change from Baseline (Day 1) to Week 26 in left atrial function index in Cohort 1

  26 weeks

• Part 2: Change from Baseline (Day 1) to Week 26 in peak VO2 in Cohort 1

  26 weeks

Secondary Outcomes (14)

• Part 1: Change from Baseline (Day 1) to Week 26 in peak VO2

  26 weeks

• Part 1: Association between response in left ventricular global longitudinal strain and/or left atrial function index after 2 weeks of active treatment with placebo-adjusted Week 26 response in peak VO2

  26 weeks

• Part 1: Change from Baseline (Day 1) to Week 26 in left atrial function index in overall population

  26 weeks

• Part 1 and 2: Change from Baseline (Day 1) to Week 26 in left ventricular ejection fraction

  26 weeks

• Part 1 and 2: Change from Baseline (Day 1) to Week 26 in ventilatory efficiency

  26 weeks

Other Outcomes (2)

• Part 1 and 2: Incidence and severity of treatment emergent adverse events, adverse events of special interest, serious adverse events and disease related events

  26 weeks

• Incidence of abnormal findings in laboratory assessments, physical examinations, 12-lead ECGs, and vital signs

  26 weeks

Study Arms (2)

danicamtiv

EXPERIMENTAL

Drug: danicamtiv

placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

danicamtiv DRUG

Danicamtiv will be administrated twice daily for up to 26 weeks

danicamtiv

Placebo DRUG

Placebo will be administrated twice daily for up to 26 weeks

placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Has a diagnosis of DCM due to probable disease-causing variants of MYH7, TTN, or other identified genetic DCM variants, or with familial DCM
• Has New York Heart Association (NYHA) Class II-IV at Screening with stable symptoms for ≥1 month.
• Has at least mild left ventricular enlargement (LVE) and has adequate acoustic windows to enable accurate TTEs according to the Echocardiography Core Laboratory.
• Has a LVEF of ≤45%.
• Is on stable doses of maximally tolerated standard-of-care heart failure (HF) therapies reflecting current guidelines for at least 4 weeks prior to the first visit.
• Has DCM not attributed to substance abuse, amyloidosis, sarcoidosis, or any other secondary form of cardiomyopathy per the Investigator.
• Can perform an upright cardiopulmonary exercise training (CPET) with a peak oxygen uptake (pV̇O2) of 80% or less of predicted for a healthy individual and respiratory exchange ratio (RER) of ≥1.05

You may not qualify if:

• Has heart failure with reduced ejection (HFrEF) caused primarily by ischemic heart disease, chronic valvulopathy, or another condition, as determined by the Investigator.
• Recent (\<90 days) clinically significant cardiac events, including acute coronary syndrome, hemodynamically significant epicardial coronary disease (per Investigator), coronary revascularization (percutaneous coronary intervention \[PCI\] or coronary artery bypass graft \[CABG\]), or hospitalization for heart failure/intravenous (IV) diuretic use.
• Presence of disqualifying cardiac rhythms that might interfere with reliable echocardiographic measurements of left ventricle (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.
• Unstable or untreated severe ventricular arrythmia (eg, ventricular tachycardia or ventricular fibrillation).
• History of malignancy of any type within 5 years prior to Screening
• Severe renal insufficiency (defined at the time of Screening as current estimated glomerular filtration rate \[eGFR\] \<15 mL/min/1.73m2 by simplified Modification of Diet in Renal Disease equation \[sMDRD\]).
• History or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the Investigator or Sponsor, would pose a risk to participant safety or interfere with the study evaluation
• History of heart transplantation or anticipated heart transplantation in the next 6 months.
• Ongoing or anticipated advanced cardiac interventions, including chronic IV inotropic therapy, planned cardiac resynchronization therapy (CRT) or major surgery, or current/anticipated ventricular assist device placement within 6 months
• Clinically significant laboratory abnormalities at Screening

Sponsors & Collaborators

• Kardigan, Inc.: lead

Study Sites (33)

University of Alabama Birmingham

Birmingham, Alabama, 35233, United States

RECRUITING

Cedars Sinai

Los Angeles, California, 90048, United States

RECRUITING

UCSF

San Francisco, California, 94158, United States

RECRUITING

MedStar Washington Hospital Center

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Louisiana State University (LSU) Health Sciences Center

New Orleans, Louisiana, 70112, United States

RECRUITING

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

Henry Ford Health System

Detroit, Michigan, 48202, United States

RECRUITING

Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States

RECRUITING

Washington University in St. Louis

St Louis, Missouri, 63110, United States

RECRUITING

University of Nebraska Medical Center

Omaha, Nebraska, 68106, United States

RECRUITING

NYU Langone Health

New York, New York, 10016, United States

RECRUITING

The Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States

RECRUITING

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Baylor College of Medicine

Houston, Texas, 77030, United States

RECRUITING

Aarhus University Hospital

Aarhus, 8200, Denmark

RECRUITING

Rigshospitalet

Copenhagen, 1050, Denmark

RECRUITING

AP-HP Hopital Pitie-Salpetriere

Paris, 75013, France

RECRUITING

Semmelweis Egyetem - Varosmajori Sziv es Ergyogyaszati Klinika

Budapest, 1122, Hungary

RECRUITING

Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont

Szeged, 6724, Hungary

RECRUITING

Azienda sanitaria universitaria Giuliano Isontina (ASU GI) - Ospedale di Cattinara

Trieste, 34100, Italy

RECRUITING

Hospital Universitario Vall d'Hebron

Horta-Guinardó, Barcelona, 08035, Spain

RECRUITING

Hospital Universitario Puerta de Hierro

Majadahonda, Madrid, 20222, Spain

RECRUITING

Hospital Universitario de A Coruña

A Coruña, 15006, Spain

RECRUITING

Hospital Universitari de Bellvitge

Barcelona, 08907, Spain

RECRUITING

Hospital Clinico Universitario Virgen de la Arrixaca

Murcia, 30120 El Palmar,, Spain

RECRUITING

Hospital Universitario Son Llatzer

Palma de Mallorca, 7198, Spain

RECRUITING

Hospital Universitario Virgen del Rocio

Seville, 41013, Spain

RECRUITING

Sodersjukhuset AB

Stockholm, 11883, Sweden

RECRUITING

NHS Greater Glasgow and Clyde

Glasgow, G12 0YN, United Kingdom

RECRUITING

St. Bartholomew's Hospital

London, EC1A, United Kingdom

RECRUITING

Royal Brompton Hospital

London, SW3 6NP, United Kingdom

RECRUITING

Hammersmith Hospital, Imperial College of London

London, W12 0HS, United Kingdom

RECRUITING

Cardiovascular Clinical Research Facility (CCRF)

Oxford, OX3 9DU, United Kingdom

RECRUITING

Central Study Contacts

Kardigan Clinical Trial Information Team

CONTACT

1-877-310-5135; clinicaltrials@kardigan.bio

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2025
• First Posted: October 7, 2025
• Study Start: February 13, 2026
• Primary Completion (Estimated): September 1, 2027
• Study Completion (Estimated): January 1, 2028
• Last Updated: April 28, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1); HU (2); ES (7); DK (2); FR (1); SE (1); US (14); GB (5)