Cardiovascular disease
Conditions
Interventions
No intervention.
Sponsors
Alexion Pharmaceuticals
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Participants must be 18 years or over (or the minimum country specific age of consent if >18), at Screening (Visit 1). Type of Participant and Disease Characteristics: 2. Participants who are willing and able to comply with all scheduled visits, laboratory tests, and other study procedures. 3. Documented BAG3 pathogenic or likely pathogenic mutation interpreted according to the American College of Medical Genetics Guidelines. 4. Heart failure Stage B-D, NYHA Class I-IV (See Section 10.7 for HF staging).5. LVEF <=50%, except if LVEF 45% to 50% and Stage B/NYHA Class I then NT-proBNP must be >=300 ng/dL. LVEF measurement must be within the last 12 month by either echocardiography or cMRI 6. Capable of giving signed informed consent as described in section 10.1.2, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
Exclusion criteria
Exclusion criteria: 1. Acute decompensated HF within 1 month prior to enrollment. Any of the following within 3 months prior to screening: myocardial infarction, cardiac surgical procedures (other than for pacemaker/ICD cardiac resynchronization therapy defibrillator 2. implantation, acute coronary syndrome or hospitalization for cardiac arrhythmia. 3. History of heart transplantation. 4. Estimated glomerular filtration rate <30 mL/min/1.73 m2 (using the chronic kidney disease epidemiology formula). 5. Malignancy that is active or has been diagnosed within 3 years prior to screening, except surgically curatively resected in situ malignancies or surgically cured early breast cancer, prostate cancer, skin cancer (basal cell carcinoma, squamous cell carcinoma), thyroid cancer, or cervical cancer, or, with prior review by the medical monitor, other early stage surgically curatively resected malignancies with greater than a 20% expected 2 year recurrence rate. 6. Noncardiac condition that limits lifespan to <1 year. 7. Presence of other form(s) of cardiomyopathy contributing to HF (eg, inflammatory or infiltrative cardiomyopathy), clinically significant cardiac anatomic abnormality (eg, LV aneurysm), clinically significant coronary artery disease (eg, coronary revascularization, exercise induced angina) per Investigator judgment or uncorrected, hemodynamically significant (ie, moderate to severe) primary structural valvular disease not due to HF. 8. Any severe concurrent disease or condition (eg, active systemic infection) that, in the judgment of the investigator, would make the participant inappropriate for study participation. 9. Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behaviour or laboratory abnormality that may increase the risk of study participation or, in the investigator*s judgment, make the participant inappropriate for the study. 10. Previous administration with an investigational drug within 30 days (or as determined by the local requirement). 11. Previously treated with gene therapy.12. In the Investigator’s opinion, there is a likelihood of undergoing cardiac transplantation, left ventricular assist device (LVAD) or other device implantation, or other cardiac surgery within the next 3 months. 13. No more than 3 first-degree members of the same family. 14. Investigator site staff or Alexion employees directly involved in the conduct of the study, site staff otherwise supervised by the investigator, and their respective family members.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Baseline and change over the course of one year follow up time measures of left ventricular volumes at end systole and diastole including LVEF, LVESVI, LVEDVI and LVMI using imaging over the course of one year follow up time. | — |
Secondary
| Measure | Time frame |
|---|---|
| Baseline, variance, and correlation of circulating biochemical biomarkers hsTNI, hsTNT and NT-proBNP to clinical markers of disease severity over the course of one year follow up time. | — |
Countries
Netherlands, Poland, Spain, United Kingdom, United States
Contacts
Public ContactE.V. Dolieslager
ICON Clinical Research
Outcome results
None listed