Open-label Extension Study to Evaluate Long-term Safety of Sacubitril/Valsartan in Pediatric Patients With HF

Phase 3

Completed

• Conditions: Heart Failure
• Interventions: Drug: sacubitril/valsartan

• Registration Number: NCT03785405
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

The purpose of this study was to evaluate long-term safety and tolerability and to provide post-trial access to sacubitril/valsartan to eligible participants who successfully completed CLCZ696B2319 (PANORAMA-HF) core study Part 2 as per protocol.

Detailed Description

This was a multicenter, open-label long-term extension study for participants who successfully completed PANORAMA-HF core study Part 2 of the trial or who discontinued study drug treatment early in Part 2 due to the implementation of Urgent safety measure (USM) of PANORAMA-HF core study. Provided that they fulfilled the protocol requirements, these patients were eligible to participate in the OLE. Depending on when and where the participant was enrolled, the duration of the study was of a minimum of 1 year, or until receipt of local marketing authorization and commercial availability, or reaching the maximum limit allowed by local regulations, or until Dec-2023, whichever occurred first.

Study Timeline

• Study First Submitted: 2018-12-20
• Study First Submitted QC: 2018-12-20
• Study First Posted: 2018-12-24
• Study Start: 2019-05-02
• Primary Completion: 2023-12-29
• Study Completion: 2023-12-29
• Status Verified: 2024-06
• Results First Submitted: 2024-06-20
• Results First Submitted QC: 2024-06-20
• Last Update Submitted: 2024-06-20
• Results First Posted: 2024-07-16
• Last Update Posted: 2024-07-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 216

Inclusion Criteria

• Signed informed consent
• On study drug at PANORAMA-HF Part 2 EOS visit. Does not have any significant safety issue

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Exclusion Criteria

• Subject only participated in PANORAMA-HF Part 1 or was a SF in PANORAMA-HF or permanently discontinued study drug in PANORMA-HF Part 2
• Use of investigational drugs within 5 half-lives of enrollment or within 30 days (longer duration); with the exception of PANORAMA-HF study drug (requires >/=36-hour washout before baseline visit)
• History of hypersensitivity or allergy to study treatment, its excipients or drugs of similar chemical class, ACEIs, ARBs, or NEP inhibitor and known/suspected contraindications to sacubitril/valsartan
• Renal vascular hypertension (including renal artery stenosis)
• Significant renal estimated glomerular filtration rate disorder (eGFR calculated using modified Schwartz formula <30% mean GFR for age); hepatic disorder (serum aspartate aminotransferase or alanine aminotransferase > 3 times upper limit of normal); gastrointestinal disorder or biliary disorder
• History of angioedema
• Parents or legal guardians of subject who do not give consent or allow the child to give assent, or inability of patient or 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
• Other protocol defined inclusion/exclusion criteria may apply

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
sacubitril/valsartan	sacubitril/valsartan	single arm, open label sacubitril/valsartan

Primary Outcome Measures

Name	Time	Method
Number of Participants With Serious Adverse Events	to end of study, up to 4.5 years	Number of participants with at least one Serious Adverse Events (SAEs)
Number of Participants With Adverse Events	to end of study, up to 4,5 years	Number of participants with at least one Adverse Events (AEs)
Duration of Drug Exposure	Up to 4.5 years	Median duration of exposure to sacubitril/valsartan (including temporary interruptions)

Trial Locations

Locations (2)

Novartis Investigative Site; 🇹🇷 Konak-Izmir, Turkey

Mayo Clinic - Rochester; 🇺🇸 Rochester, Minnesota, United States