Heart Failure due to Dilated Cardiomyopathy MedDRA version: 18.1 Level: LLT Classification code 10056419 Term: Dilated cardiomyopathy System Organ Class: 100000004849
Conditions
Sponsors
Ethicare GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Age 1 month to ? P95 and/or LV shortening fraction (SF) =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Severe HF and/or end stage heart failure requiring ICU support precluding introduction or continuation of ACEI. • Too low blood pressure, e.g. ?P5 • Restrictive and hypertrophic cardiomyopathies. • Obstructive valvular disease (peak echocardiographic gradient more than 30 mm Hg). • Uncorrected severe peripheral stenosis of large arteries including severe coarctation of the aorta. • Severe renal impairment with a GFR below 30 ml/1.73 m2 serum creatinine >2x ULN (Upper Limit of Normal) according to the hospital’s test methodology). • History of Angioedema. • Hypersensitivity to ACEI. • Concomittant medication: o Dual ACEI therapy o Renin inhibitors o Angiotensin II antagonists o NSAIDs except for aspirin acetylsalicylic acid only for antiplatelet therapy • Already enrolled in interventional trial with an investigational drug, unless no interference with current study can be shown.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To obtain paediatric pharmacokinetic data of enalapril and its active metabolite enalaprilat in paediatric patients treated with enalapril ODMTs to describe the dose exposure in the paediatric population with DCM.;Secondary Objective: 1. To demonstrate safety, in particular renal safety, of enalapril ODMTs in children with DCM. 2. To characterise the dose/safety relationship from a starting dose to an optimal maintenance dose. 3. To explore the dose exposure/response relationship with pharmacodynamic parameters in the paediatric population with DCM. 4. To investigate the Shortening Fraction (SF) of the heart muscle by echocardiography. 5. To investigate the acceptability and palatability of enalapril ODMTs in the paediatric population with DCM. ;Primary end point(s): The bioavailability of enalapril and its active metabolite enalaprilat in the paediatric population (AUC from 0 to time of last sampling point, Cmax and Tmax); descriptive pharmacokinetic investigation.;Timepoint(s) of evaluation of this end point: PK/PD profile sampling timepoint: Visit day 1: pre-dose, 0h, 2, 4, 6, 8, 12 hours | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. The bioavailability of enalapril and its active metabolite enalaprilat in the different age subsets (1 months to ?12 months, 12 months to ?6 years, 6 years to ?12 years) of the paediatric population (AUC from 0 to time of last sampling point, Cmax and Tmax); descriptive pharmacokinetic investigation. 2. Markers of the renin-angiotensin-aldosterone system as exploratory pharmacodynamic investigation. 3. Brain natriuretic peptides (BNPs). 4. Acceptability and palatability of the novel formulation. 5. Safety parameters including blood pressure and renal function. 6. Echocardiography (Shortening Fraction) 7. Rehospitalisation due to heart failure. 8. Death due to worsening of the underlying disease 9. Pharmacodynamic and efficacy endpoints analysis to differentiate high and low output disease ;Timepoint(s) of evaluation of this end point: Biochemistry, Urine and BNP collected during each visit in the hospital to ensure renal function. PK and PD 1 sample collected during each visit, unless the patient terminated use of the IMP, then only PD sample is collected. Accepútability and Palatability questionnaire is completed during initial visit, once stable dose is reached and during end of study visit. | — |
Countries
Austria, Germany, Hungary, Netherlands, Serbia, United Kingdom
Contacts
Public ContactLead Clinical Project Manager
Pharmaplex bvba
Outcome results
None listed