Heart Failure
Conditions
Keywords
sacubitril/valsartan, post-trial access, pediatric, heart failure
Brief summary
The purpose of this open-label study was to collect additional safety information of sacubitril/valsartan and to provide post-trial access to sacubitril/valsartan for the eligible Japanese patients who completed CLCZ696B2319E1(NCT03785405) study until marketed product of pediatric formulation, film-coated granules in capsule, is available in Japan.
Detailed description
This trial (CLCZ696B2319E2) was a multicenter, open-label extension study for Japanese patients who have successfully completed the CLCZ696B2319E1 study. Only Japanese patients who successfully completed CLCZ696B2319E1 study and fulfilled protocol requirements were eligible to participate in this study. The first visit (Visit Day1) was the same day as the End of Study visit of CLCZ696B2319E1 study.
Interventions
DRUGsacubitril/valsartan
sacubiril/valsartan
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Signed informed consent * \<18 years of age (at the time of signing informed consent) * Completed CLCZ696B2319E1 study and safely enrolled
Exclusion criteria
* Permanently discontinued the study treatment during CLCZ696B2319E1 study * Renal vascular hypertension (including renal artery stenosis) * History of angioedema * Having parents or legal guardians who do not give consent or allow the child to give assent, or inability of patient or the parents/legal guardians to follow instructions or comply with follow-up procedures * Any medical condition(s) that may put the patient at risk in the Investigator's opinion or that the Investigator deems unsuitable for the study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of approximately 8 months. | Number of participants with treatment emergent adverse events (any AE regardless of seriousness), and SAEs. |
Countries
Japan
Participant flow
Recruitment details
Participants took part in 6 investigative sites in Japan.
Pre-assignment details
All consenting participants were assessed for eligibility into this study at the first visit (Visit Day 1) and the study medication was initiated.
Participants by arm
| Arm | Count |
|---|---|
| Sacubitril/Valsartan The starting dose of study drug was determined by the investigator in consideration of the participant´s condition. The dose level at the end of study visit of CLCZ696B2319E1 study could remain the same, or the dose level could be changed at the discretion of the investigator. | 8 |
| Total | 8 |
Baseline characteristics
| Characteristic | Sacubitril/Valsartan |
|---|---|
| Age, Continuous | 6.75 years STANDARD_DEVIATION 4.234 |
| Race/Ethnicity, Customized Asian | 8 Participants |
| Sex: Female, Male Female | 6 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 8 | 0 / 8 |
| other Total, other adverse events | 6 / 8 | 6 / 8 |
| serious Total, serious adverse events | 1 / 8 | 1 / 8 |
Outcome results
Primary
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Number of participants with treatment emergent adverse events (any AE regardless of seriousness), and SAEs.
Time frame: Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of approximately 8 months.
Population: Safety Set (SAF): all participants who received at least one dose of open-label study treatment.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Sacubitril/Valsartan | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Adverse Events | 6 Participants |
| Sacubitril/Valsartan | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Serious Adverse Events | 1 Participants |