A phase 2, randomised, double-blind, placebo-controlled, adaptive design study investigating the safety and effectiveness of orally administered BGP-15 on cardiac fibrosis and heart function in people with Non-Ischaemic Dilated Cardiomyopathy (NIDCM)
- Conditions
- on-ischemic dilated cardiomyopathyCardiac fibrosisNon-ischemic dilated cardiomyopathyCardiovascular - Other cardiovascular diseases
- Registration Number
- ACTRN12616000482460
- Lead Sponsor
- BakerIDI Heart and Diabetes Institute
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Withdrawn
- Sex
- All
- Target Recruitment
- 90
1.Males aged 18-80 years, inclusive
2.Females aged 18-80 years inclusive meeting the following criteria:
- A negative serum pregnancy test at Screening, not breast feeding and did not plan to be pregnant during the study, AND if one of the following three criteria was met:
-They had a hysterectomy or tubal ligation at least 6 months prior to signing the informed consent form;
-They had been postmenopausal for at least 1 year; or,
-They were of childbearing potential and practiced one of the following methods of birth control throughout the study: injectable or implantable hormonal contraception or intrauterine device; or two of the following methods of birth control throughout the study: oral or patch contraception plus a barrier contraceptive (e.g., diaphragm plus spermicide, male or female condom plus spermicide, or vasectomized male partner).
3.Known history of non-ischemic dilated cardiomyopathy as determined using appropriate invasive and/or non-invasive imaging assessments
4.Reduced left ventricular function, with a calculated LVEF between 35-50% based on CMR imaging
5.Evidence for significant LV dilation defined as > 2SD above the mean LVEDVi value for a gender compared normal population
6.Evidence of increased interstitial myocardial fibrosis, suggested by a native myocardial T1 time greater than 1218ms.
7.No evidence for maximum LV wall thickness > 15mm at end-diastole by cardiac magnetic resonance imaging or echocardiography
8.No evidence for resting LV outflow tract (LVOT) obstruction > 10mmHg at either valvular and/or sub-valvular level
9.Stable symptoms with no hospital admissions for heart failure in the preceding 3 months
10.Stable heart failure medical therapy with no changes in medications (other than diuretic dose) in preceding 3 months
11.Able to perform exercise testing but unable to achieve greater than the lower limit of normal VO2 max (20 mL/kg/min) at visit 2.
12.Expected life expectancy of at least 2 years.
13.Have given written informed consent
14.In the investigator’s opinion, the participant is able and willing to comply with study medication as required by the protocol.
1.Any contraindication to CMR scanning (non-CMR compatible pacemaker, other metallo-prosthetic implant, or severe claustrophobia).
2.Stage IIIB or worse renal dysfunction (glomerular filtration rate [GFR] <45ml/hour)
3.Plan for cardiac pacemaker of implantable cardioverter defibrillator (ICD) insertion.
4.Any severe comorbidity precluding study drug or successful completion of study protocol.
5.Previous myocardial infarction
6.Evidence of liver disease defined as aspartate transaminase (AST), alanine transaminase (ALT) or total bilirubin >2 x upper limit of normal at Visit 2
7.History of active malignancy within the past 5 years except for localized prostate cancer treated with hormone therapy, cervical carcinoma, or non-malignant melanoma
8.History or evidence of drug or alcohol abuse within the last 12 months of Visit 1
9.Use of other investigational drugs and devices at the time of enrolment, or within 90 days of Visit 1
10.History of non-compliance to medical regimens or unwillingness to comply with the study protocol
11.Any condition that in the opinion of the Investigators would confound the evaluation and interpretation of the data
12.Persons directly involved in the execution of the protocol
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Safety and tolerability compared between placebo and drug treatment. Safety will be assessed by blood tests (FBE, LFT, UEC) and the total number and severity of adverse events as well as the number and severity of treatment related adverse events. Tolerability refers to the ability of the participant to tolerate the adverse events. Tolerability will assessed by analysis of dropout rates specifically due to adverse events. Given the small number of previous studies and adverse event profile has not yet emerged from the recorded incidences of AE’s and SAE’s. The side effects previously reported in studies with placebo and BGP-15 include mild to moderate increases in appetite and weight gain, headache, gastrointestinal symptoms including nausea and diarrhoea, fatigue or sleepiness. Less common side effects include mild changes to ECG rhythm and moderate increases in liver enzymes. [The end of 3, 6 and 12 months treatment]
- Secondary Outcome Measures
Name Time Method