Clinical trial NCT03785405: Open-label Extension Study to Evaluate Long-term Safety of Sacubitril/Valsartan in Pediatric Patients With HF
Official title: A Multicenter Study to Evaluate Long-term Safety and Tolerability of Open Label Sacubitril/Valsartan in Pediatric Patients With Heart Failure Due to Systemic Left Ventricle Systolic Dysfunction Who Have Completed Study CLCZ696B2319
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Heart Failure
Keywords: heart failure
pediatric
LCZ696
sacubitril/valsartan
open-label study
angiotensin receptor neprilysin inhibitor
PANORAMA-HF
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2018-12-24
Enrollment: 216
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria:

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

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

Primary outcomes:
measure: Number of Participants With Adverse Events
timeFrame: to end of study, up to 4,5 years
measure: Number of Participants With Serious Adverse Events
timeFrame: to end of study, up to 4.5 years
measure: Duration of Drug Exposure
timeFrame: Up to 4.5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Loma Linda
country: United States
facility: Novartis Investigative Site
state: California
city: Los Angeles
country: United States
facility: Novartis Investigative Site
state: California
city: Palo Alto
country: United States
facility: Novartis Investigative Site
state: California
city: Saint Petersburg
country: United States
facility: Novartis Investigative Site
state: Florida
city: Atlanta
country: United States
facility: Novartis Investigative Site
state: Georgia
city: Indianapolis
country: United States
facility: Novartis Investigative Site
state: Indiana
city: Boston
country: United States
facility: Novartis Investigative Site
state: Massachusetts
city: Ann Arbor
country: United States
facility: Novartis Investigative Site
state: Michigan
city: Minneapolis
country: United States
facility: Novartis Investigative Site
state: Minnesota
city: Rochester
country: United States
facility: Mayo Clinic - Rochester
state: Minnesota
city: Saint Louis
country: United States
facility: Novartis Investigative Site
state: Missouri
city: New York
country: United States
facility: Novartis Investigative Site
state: New York
city: Charlotte
country: United States
facility: Novartis Investigative Site
state: North Carolina
city: Philadelphia
country: United States
facility: Novartis Investigative Site
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Novartis Investigative Site
state: Pennsylvania
city: Ciudad de Salta
country: Argentina
facility: Novartis Investigative Site
state: Provincia De Salta
city: Innsbruck
country: Austria
facility: Novartis Investigative Site
city: Sofia
country: Bulgaria
facility: Novartis Investigative Site
city: Edmonton
country: Canada
facility: Novartis Investigative Site
state: Alberta
city: Toronto
country: Canada
facility: Novartis Investigative Site
state: Ontario
city: Zagreb
country: Croatia
facility: Novartis Investigative Site
city: Praha 5
country: Czechia
facility: Novartis Investigative Site
city: Helsinki
country: Finland
facility: Novartis Investigative Site
city: Paris 15
country: France
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Erlangen
country: Germany
facility: Novartis Investigative Site
city: Freiburg
country: Germany
facility: Novartis Investigative Site
city: Heidelberg
country: Germany
facility: Novartis Investigative Site
city: Stuttgart
country: Germany
facility: Novartis Investigative Site
city: Budapest
country: Hungary
facility: Novartis Investigative Site
city: New Delhi
country: India
facility: Novartis Investigative Site
state: Delhi
city: Ahmedabad
country: India
facility: Novartis Investigative Site
state: Gujarat
city: Kochi
country: India
facility: Novartis Investigative Site
state: Kerala
city: New Delhi
country: India
facility: Novartis Investigative Site
city: Beer Sheva
country: Israel
facility: Novartis Investigative Site
city: Bergamo
country: Italy
facility: Novartis Investigative Site
state: BG
city: Bologna
country: Italy
facility: Novartis Investigative Site
state: BO
city: Firenze
country: Italy
facility: Novartis Investigative Site
state: FI
city: Milano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Roma
country: Italy
facility: Novartis Investigative Site
state: RM
city: Torino
country: Italy
facility: Novartis Investigative Site
state: TO
city: Napoli
country: Italy
facility: Novartis Investigative Site
city: Obu
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Sapporo city
country: Japan
facility: Novartis Investigative Site
state: Hokkaido
city: Omura
country: Japan
facility: Novartis Investigative Site
state: Nagasaki
city: Bunkyo ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Setagaya-ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Shinjuku ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Toyama-city
country: Japan
facility: Novartis Investigative Site
state: Toyama
city: Saitama
country: Japan
facility: Novartis Investigative Site
city: Yangsan Si
country: Korea, Republic of
facility: Novartis Investigative Site
state: Gyeongsangnam Do
city: Seoul
country: Korea, Republic of
facility: Novartis Investigative Site
city: Seoul
country: Korea, Republic of
facility: Novartis Investigative Site
city: Seoul
country: Korea, Republic of
facility: Novartis Investigative Site
city: Ashrafieh
country: Lebanon
facility: Novartis Investigative Site
city: Beirut
country: Lebanon
facility: Novartis Investigative Site
city: Warszawa
country: Poland
facility: Novartis Investigative Site
city: Wroclaw
country: Poland
facility: Novartis Investigative Site
city: Carnaxide
country: Portugal
facility: Novartis Investigative Site
state: Lisboa
city: Coimbra
country: Portugal
facility: Novartis Investigative Site
city: Lisboa
country: Portugal
facility: Novartis Investigative Site
city: Moscow
country: Russian Federation
facility: Novartis Investigative Site
city: Saint Petersburg
country: Russian Federation
facility: Novartis Investigative Site
city: Singapore
country: Singapore
facility: Novartis Investigative Site
city: Singapore
country: Singapore
facility: Novartis Investigative Site
city: Soweto
country: South Africa
facility: Novartis Investigative Site
state: Gauteng
city: Cordoba
country: Spain
facility: Novartis Investigative Site
state: Andalucia
city: Esplugues De Llobregat
country: Spain
facility: Novartis Investigative Site
state: Barcelona
city: Barcelona
country: Spain
facility: Novartis Investigative Site
state: Catalunya
city: Madrid
country: Spain
facility: Novartis Investigative Site
city: Madrid
country: Spain
facility: Novartis Investigative Site
city: Lausanne
country: Switzerland
facility: Novartis Investigative Site
city: Tainan
country: Taiwan
facility: Novartis Investigative Site
city: Taipei
country: Taiwan
facility: Novartis Investigative Site
city: Bangkoknoi
country: Thailand
facility: Novartis Investigative Site
state: Bangkok
city: Bangkok
country: Thailand
facility: Novartis Investigative Site
city: Ankara
country: Turkey
facility: Novartis Investigative Site
city: Izmir
country: Turkey
facility: Novartis Investigative Site
city: Konak-Izmir
country: Turkey
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: