Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LCZ696 Followed by a 52-week Study of LCZ696 Compared With Enalapril in Pediatric Patients With Heart Failure- PANORAMA
- Conditions
- Cardiovascular / Pediatric Heart FailureHFI50.9
- Registration Number
- LBCTR2019040224
- Lead Sponsor
- ovartis Pharma Services Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 10
Chronic heart failure resulting from left ventricular systolic dysfunction, and receiving chronic HF therapy (if not newly diagnosed)
NYHA classification II-IV (older children: 6 to <18 years old) or Ross CHF classification II-IV (younger children: < 6 years old)
Systemic left ventricular ejection fraction = 40% or fractional shortening =20%
For Part 1 study: Patients must be treated with an ACEI or ARB prior to screening. Patients in Group 1 and 2 must be currently treated with the dose equivalent of at least enalapril 0.2 mg/kg prior to the LCZ696 3.1 mg/kg administration. Group 3 patients will participate in LCZ696 0.8 mg/kg and not LCZ696 3.1 mg/kg.
Biventricular physiology with systemic left ventricle
Patient with single ventricle or systemic right ventricle
Patients listed for heart transplantation (as United Network for Organ Sharing status 1A) or hospitalized waiting for transplant (while on inotropes or with ventricular assist device)
Sustained or symptomatic dysrhythmias uncontrolled with drug or device therapy
Patients that have had cardiovascular surgery or percutaneous intervention to palliate or correct congenital cardiovascular malformations within 3 months of the screening visit. Patients anticipated to undergo corrective heart surgery during the 12 months after entry into Part 2
Patients with unoperated obstructive or severe regurgitant valvular (aortic, pulmonary, or tricuspid) disease, or significant systemic ventricular outflow obstruction or aortic arch obstruction
Patients with restrictive or hypertrophic cardiomyopathy
Active myocarditis
Renal vascular hypertension (including renal artery stenosis)
Moderate-to severe obstructive pulmonary disease
Serum potassium > 5.3 mmol/L
History of angioedema
Allergy or hypersensitivity to ACEI / ARB
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method ame: Percentage of patients falling into each category based on global ranking;Timepoints: 52 weeks;Measure: 52 weeks;Name: The global ranking is based on clinical events such as death, listing for urgent heart transplant, mechanical life support requirement at end of study, worsening heart failure (HF), New York Heart Association (NYHA)/Ross, Patient Global Impression of Severity (PGIS), Pediatric Quality of Life Inventory (PedsQL) physical functioning domain. The primary endpoint will be derived based on 5 categories ranking worst to best outcome;Timepoints: 52 weeks;Measure: 52 weeks
- Secondary Outcome Measures
Name Time Method ame: Time to first occurrence of Category 1 or Category 2 event;Timepoints: 52 weeks;Measure: 52 weeks;Name: Change from baseline in NYHA/Ross functional class ;Timepoints: 52 weeks;Measure: 52 weeks