Design for the sacubitril/valsartan (LCZ696) compared with enalapril study of pediatric patients with heart failure due to systemic left ventricle systolic dysfunction (PANORAMA-HF study)
Sacubitril/valsartan (LCZ696) is an angiotensin receptor neprilysin inhibitor approved for the treatment of adult heart failure (HF); however, the benefit of sacubitril/valsartan in pediatric HF patients is unknown. This global multi-center study will use an adaptive, seamless two-part design. Part...
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| Veröffentlicht in: | The American heart journal 2017-11, Vol.193, p.23-34 |
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| container_title | The American heart journal |
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| creator | Shaddy, Robert Canter, Charles Halnon, Nancy Kochilas, Lazaros Rossano, Joseph Bonnet, Damien Bush, Christopher Zhao, Ziqiang Kantor, Paul Burch, Michael Chen, Fabian |
| description | Sacubitril/valsartan (LCZ696) is an angiotensin receptor neprilysin inhibitor approved for the treatment of adult heart failure (HF); however, the benefit of sacubitril/valsartan in pediatric HF patients is unknown.
This global multi-center study will use an adaptive, seamless two-part design. Part 1 will assess the pharmacokinetics/pharmacodynamics of single ascending doses of sacubitril/valsartan in pediatric (1 month to |
| doi_str_mv | 10.1016/j.ahj.2017.07.006 |
| format | Article |
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This global multi-center study will use an adaptive, seamless two-part design. Part 1 will assess the pharmacokinetics/pharmacodynamics of single ascending doses of sacubitril/valsartan in pediatric (1 month to <18 years) HF patients with systemic left ventricle and reduced left ventricular systolic function stratified into 3 age groups (Group 1: 6 to <18 years; Group 2: 1 to <6 years; Group 3: 1 month to <1 year). Part 2 is a 52-week, efficacy and safety study where 360 eligible patients will be randomized to sacubitril/valsartan or enalapril. A novel global rank primary endpoint derived by ranking patients (worst-to-best outcome) based on clinical events such as death, initiation of mechanical life support, listing for urgent heart transplant, worsening HF, measures of functional capacity (NYHA/Ross scores), and patient-reported HF symptoms will be used to assess efficacy.
The PANORAMA-HF study, which will be the largest prospective pediatric HF trial conducted to date and the first to use a global rank primary endpoint, will determine whether sacubitril/valsartan is superior to enalapril for treatment of pediatric HF patients with reduced systemic left ventricular systolic function.
Design of the two-stage, seamless, adaptive PANORAMA-HF study assessing the PK/PD, safety, and efficacy of sacubitril/valsartan versus enalapril in pediatric heart failure patients with systemic left ventricle and reduced left ventricular systolic function.
bid, twice-weekly; m, month; mg, milligram; kg, kilogram; PD, pharmacodynamics; PK, pharmacokinetics. [Display omitted]</description><identifier>ISSN: 0002-8703</identifier><identifier>EISSN: 1097-6744</identifier><identifier>DOI: 10.1016/j.ahj.2017.07.006</identifier><identifier>PMID: 29129252</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject><![CDATA[Adolescent ; Adults ; Aminobutyrates - administration & dosage ; Aminobutyrates - pharmacokinetics ; Angiotensin ; Angiotensin II Type 1 Receptor Blockers - administration & dosage ; Angiotensin II Type 1 Receptor Blockers - pharmacokinetics ; Angiotensin-Converting Enzyme Inhibitors - administration & dosage ; Angiotensin-Converting Enzyme Inhibitors - pharmacokinetics ; Cardiomyopathy ; Cardiovascular disease ; Child ; Child, Preschool ; Clinical trials ; Congenital diseases ; Design ; Dose-Response Relationship, Drug ; Drug Combinations ; Drug dosages ; Drug therapy ; Drug Therapy, Combination ; Enalapril - administration & dosage ; Enalapril - pharmacokinetics ; Enzymes ; FDA approval ; Female ; Follow-Up Studies ; Heart ; Heart diseases ; Heart failure ; Heart Failure - drug therapy ; Heart Failure - etiology ; Heart Failure - metabolism ; Heart transplantation ; Humans ; Infant ; Infant, Newborn ; Male ; Mortality ; Myocardial Ischemia - complications ; Myocardial Ischemia - diagnosis ; Myocardial Ischemia - physiopathology ; Neprilysin ; Patients ; Pediatrics ; Pharmacodynamics ; Pharmacokinetics ; Pharmacology ; Prospective Studies ; Quality of life ; Systole ; Tetrazoles - administration & dosage ; Tetrazoles - pharmacokinetics ; Time Factors ; Transplants & implants ; Treatment Outcome ; Valsartan - administration & dosage ; Valsartan - pharmacokinetics ; Ventricle ; Ventricular Dysfunction, Left - complications ; Ventricular Dysfunction, Left - diagnosis ; Ventricular Dysfunction, Left - physiopathology ; Ventricular Function, Left - physiology]]></subject><ispartof>The American heart journal, 2017-11, Vol.193, p.23-34</ispartof><rights>2017 The Authors</rights><rights>Copyright © 2017 The Authors. Published by Elsevier Inc. All rights reserved.</rights><rights>Copyright Elsevier Limited Nov 1, 2017</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c424t-87525bed9fe65eb0e6addba0b465074180bf8aab161fa58e4caf55eead8747c03</citedby><cites>FETCH-LOGICAL-c424t-87525bed9fe65eb0e6addba0b465074180bf8aab161fa58e4caf55eead8747c03</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://www.proquest.com/docview/1965447487?pq-origsite=primo$$EHTML$$P50$$Gproquest$$H</linktohtml><link.rule.ids>314,778,782,3539,27907,27908,45978,64366,64370,72220</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/29129252$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Shaddy, Robert</creatorcontrib><creatorcontrib>Canter, Charles</creatorcontrib><creatorcontrib>Halnon, Nancy</creatorcontrib><creatorcontrib>Kochilas, Lazaros</creatorcontrib><creatorcontrib>Rossano, Joseph</creatorcontrib><creatorcontrib>Bonnet, Damien</creatorcontrib><creatorcontrib>Bush, Christopher</creatorcontrib><creatorcontrib>Zhao, Ziqiang</creatorcontrib><creatorcontrib>Kantor, Paul</creatorcontrib><creatorcontrib>Burch, Michael</creatorcontrib><creatorcontrib>Chen, Fabian</creatorcontrib><title>Design for the sacubitril/valsartan (LCZ696) compared with enalapril study of pediatric patients with heart failure due to systemic left ventricle systolic dysfunction (PANORAMA-HF study)</title><title>The American heart journal</title><addtitle>Am Heart J</addtitle><description>Sacubitril/valsartan (LCZ696) is an angiotensin receptor neprilysin inhibitor approved for the treatment of adult heart failure (HF); however, the benefit of sacubitril/valsartan in pediatric HF patients is unknown.
This global multi-center study will use an adaptive, seamless two-part design. Part 1 will assess the pharmacokinetics/pharmacodynamics of single ascending doses of sacubitril/valsartan in pediatric (1 month to <18 years) HF patients with systemic left ventricle and reduced left ventricular systolic function stratified into 3 age groups (Group 1: 6 to <18 years; Group 2: 1 to <6 years; Group 3: 1 month to <1 year). Part 2 is a 52-week, efficacy and safety study where 360 eligible patients will be randomized to sacubitril/valsartan or enalapril. A novel global rank primary endpoint derived by ranking patients (worst-to-best outcome) based on clinical events such as death, initiation of mechanical life support, listing for urgent heart transplant, worsening HF, measures of functional capacity (NYHA/Ross scores), and patient-reported HF symptoms will be used to assess efficacy.
The PANORAMA-HF study, which will be the largest prospective pediatric HF trial conducted to date and the first to use a global rank primary endpoint, will determine whether sacubitril/valsartan is superior to enalapril for treatment of pediatric HF patients with reduced systemic left ventricular systolic function.
Design of the two-stage, seamless, adaptive PANORAMA-HF study assessing the PK/PD, safety, and efficacy of sacubitril/valsartan versus enalapril in pediatric heart failure patients with systemic left ventricle and reduced left ventricular systolic function.
bid, twice-weekly; m, month; mg, milligram; kg, kilogram; PD, pharmacodynamics; PK, pharmacokinetics. [Display omitted]</description><subject>Adolescent</subject><subject>Adults</subject><subject>Aminobutyrates - administration & dosage</subject><subject>Aminobutyrates - pharmacokinetics</subject><subject>Angiotensin</subject><subject>Angiotensin II Type 1 Receptor Blockers - administration & dosage</subject><subject>Angiotensin II Type 1 Receptor Blockers - pharmacokinetics</subject><subject>Angiotensin-Converting Enzyme Inhibitors - administration & dosage</subject><subject>Angiotensin-Converting Enzyme Inhibitors - pharmacokinetics</subject><subject>Cardiomyopathy</subject><subject>Cardiovascular disease</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Clinical trials</subject><subject>Congenital diseases</subject><subject>Design</subject><subject>Dose-Response Relationship, Drug</subject><subject>Drug Combinations</subject><subject>Drug dosages</subject><subject>Drug therapy</subject><subject>Drug Therapy, Combination</subject><subject>Enalapril - administration & dosage</subject><subject>Enalapril - pharmacokinetics</subject><subject>Enzymes</subject><subject>FDA approval</subject><subject>Female</subject><subject>Follow-Up Studies</subject><subject>Heart</subject><subject>Heart diseases</subject><subject>Heart failure</subject><subject>Heart Failure - drug therapy</subject><subject>Heart Failure - etiology</subject><subject>Heart Failure - metabolism</subject><subject>Heart transplantation</subject><subject>Humans</subject><subject>Infant</subject><subject>Infant, Newborn</subject><subject>Male</subject><subject>Mortality</subject><subject>Myocardial Ischemia - complications</subject><subject>Myocardial Ischemia - diagnosis</subject><subject>Myocardial Ischemia - physiopathology</subject><subject>Neprilysin</subject><subject>Patients</subject><subject>Pediatrics</subject><subject>Pharmacodynamics</subject><subject>Pharmacokinetics</subject><subject>Pharmacology</subject><subject>Prospective Studies</subject><subject>Quality of life</subject><subject>Systole</subject><subject>Tetrazoles - 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administration & dosage</topic><topic>Aminobutyrates - pharmacokinetics</topic><topic>Angiotensin</topic><topic>Angiotensin II Type 1 Receptor Blockers - administration & dosage</topic><topic>Angiotensin II Type 1 Receptor Blockers - pharmacokinetics</topic><topic>Angiotensin-Converting Enzyme Inhibitors - administration & dosage</topic><topic>Angiotensin-Converting Enzyme Inhibitors - pharmacokinetics</topic><topic>Cardiomyopathy</topic><topic>Cardiovascular disease</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>Clinical trials</topic><topic>Congenital diseases</topic><topic>Design</topic><topic>Dose-Response Relationship, Drug</topic><topic>Drug Combinations</topic><topic>Drug dosages</topic><topic>Drug therapy</topic><topic>Drug Therapy, Combination</topic><topic>Enalapril - administration & dosage</topic><topic>Enalapril - pharmacokinetics</topic><topic>Enzymes</topic><topic>FDA approval</topic><topic>Female</topic><topic>Follow-Up Studies</topic><topic>Heart</topic><topic>Heart diseases</topic><topic>Heart failure</topic><topic>Heart Failure - drug therapy</topic><topic>Heart Failure - etiology</topic><topic>Heart Failure - metabolism</topic><topic>Heart transplantation</topic><topic>Humans</topic><topic>Infant</topic><topic>Infant, Newborn</topic><topic>Male</topic><topic>Mortality</topic><topic>Myocardial Ischemia - complications</topic><topic>Myocardial Ischemia - diagnosis</topic><topic>Myocardial Ischemia - physiopathology</topic><topic>Neprilysin</topic><topic>Patients</topic><topic>Pediatrics</topic><topic>Pharmacodynamics</topic><topic>Pharmacokinetics</topic><topic>Pharmacology</topic><topic>Prospective Studies</topic><topic>Quality of life</topic><topic>Systole</topic><topic>Tetrazoles - administration & dosage</topic><topic>Tetrazoles - pharmacokinetics</topic><topic>Time Factors</topic><topic>Transplants & implants</topic><topic>Treatment Outcome</topic><topic>Valsartan - administration & dosage</topic><topic>Valsartan - pharmacokinetics</topic><topic>Ventricle</topic><topic>Ventricular Dysfunction, Left - complications</topic><topic>Ventricular Dysfunction, Left - diagnosis</topic><topic>Ventricular Dysfunction, Left - physiopathology</topic><topic>Ventricular Function, Left - physiology</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Shaddy, Robert</creatorcontrib><creatorcontrib>Canter, Charles</creatorcontrib><creatorcontrib>Halnon, Nancy</creatorcontrib><creatorcontrib>Kochilas, Lazaros</creatorcontrib><creatorcontrib>Rossano, Joseph</creatorcontrib><creatorcontrib>Bonnet, Damien</creatorcontrib><creatorcontrib>Bush, Christopher</creatorcontrib><creatorcontrib>Zhao, Ziqiang</creatorcontrib><creatorcontrib>Kantor, Paul</creatorcontrib><creatorcontrib>Burch, Michael</creatorcontrib><creatorcontrib>Chen, Fabian</creatorcontrib><collection>ScienceDirect Open Access Titles</collection><collection>Elsevier:ScienceDirect:Open Access</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Central (Corporate)</collection><collection>Biotechnology Research Abstracts</collection><collection>Nursing & Allied Health Database</collection><collection>Physical Education Index</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>Healthcare Administration Database (Alumni)</collection><collection>Medical Database (Alumni Edition)</collection><collection>ProQuest Pharma Collection</collection><collection>Public Health Database</collection><collection>Technology Research Database</collection><collection>Hospital Premium Collection</collection><collection>Hospital Premium Collection (Alumni Edition)</collection><collection>ProQuest Central (Alumni) (purchase pre-March 2016)</collection><collection>Research Library (Alumni Edition)</collection><collection>ProQuest Central (Alumni Edition)</collection><collection>ProQuest Central UK/Ireland</collection><collection>British Nursing Database</collection><collection>ProQuest Central Essentials</collection><collection>ProQuest Central</collection><collection>ProQuest One Community College</collection><collection>ProQuest Central Korea</collection><collection>Engineering Research Database</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>ProQuest Central Student</collection><collection>Research Library Prep</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Nursing & Allied Health Database (Alumni Edition)</collection><collection>Health & Medical Collection (Alumni Edition)</collection><collection>Healthcare Administration Database</collection><collection>Medical Database</collection><collection>Research Library</collection><collection>Research Library (Corporate)</collection><collection>Nursing & Allied Health Premium</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>ProQuest Central China</collection><collection>ProQuest Central Basic</collection><jtitle>The American heart journal</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Shaddy, Robert</au><au>Canter, Charles</au><au>Halnon, Nancy</au><au>Kochilas, Lazaros</au><au>Rossano, Joseph</au><au>Bonnet, Damien</au><au>Bush, Christopher</au><au>Zhao, Ziqiang</au><au>Kantor, Paul</au><au>Burch, Michael</au><au>Chen, Fabian</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Design for the sacubitril/valsartan (LCZ696) compared with enalapril study of pediatric patients with heart failure due to systemic left ventricle systolic dysfunction (PANORAMA-HF study)</atitle><jtitle>The American heart journal</jtitle><addtitle>Am Heart J</addtitle><date>2017-11</date><risdate>2017</risdate><volume>193</volume><spage>23</spage><epage>34</epage><pages>23-34</pages><issn>0002-8703</issn><eissn>1097-6744</eissn><abstract>Sacubitril/valsartan (LCZ696) is an angiotensin receptor neprilysin inhibitor approved for the treatment of adult heart failure (HF); however, the benefit of sacubitril/valsartan in pediatric HF patients is unknown.
This global multi-center study will use an adaptive, seamless two-part design. Part 1 will assess the pharmacokinetics/pharmacodynamics of single ascending doses of sacubitril/valsartan in pediatric (1 month to <18 years) HF patients with systemic left ventricle and reduced left ventricular systolic function stratified into 3 age groups (Group 1: 6 to <18 years; Group 2: 1 to <6 years; Group 3: 1 month to <1 year). Part 2 is a 52-week, efficacy and safety study where 360 eligible patients will be randomized to sacubitril/valsartan or enalapril. A novel global rank primary endpoint derived by ranking patients (worst-to-best outcome) based on clinical events such as death, initiation of mechanical life support, listing for urgent heart transplant, worsening HF, measures of functional capacity (NYHA/Ross scores), and patient-reported HF symptoms will be used to assess efficacy.
The PANORAMA-HF study, which will be the largest prospective pediatric HF trial conducted to date and the first to use a global rank primary endpoint, will determine whether sacubitril/valsartan is superior to enalapril for treatment of pediatric HF patients with reduced systemic left ventricular systolic function.
Design of the two-stage, seamless, adaptive PANORAMA-HF study assessing the PK/PD, safety, and efficacy of sacubitril/valsartan versus enalapril in pediatric heart failure patients with systemic left ventricle and reduced left ventricular systolic function.
bid, twice-weekly; m, month; mg, milligram; kg, kilogram; PD, pharmacodynamics; PK, pharmacokinetics. [Display omitted]</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>29129252</pmid><doi>10.1016/j.ahj.2017.07.006</doi><tpages>12</tpages><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0002-8703 |
| ispartof | The American heart journal, 2017-11, Vol.193, p.23-34 |
| issn | 0002-8703 1097-6744 |
| language | eng |
| recordid | cdi_proquest_journals_1965447487 |
| source | MEDLINE; Elsevier ScienceDirect Journals; ProQuest Central UK/Ireland |
| subjects | Adolescent Adults Aminobutyrates - administration & dosage Aminobutyrates - pharmacokinetics Angiotensin Angiotensin II Type 1 Receptor Blockers - administration & dosage Angiotensin II Type 1 Receptor Blockers - pharmacokinetics Angiotensin-Converting Enzyme Inhibitors - administration & dosage Angiotensin-Converting Enzyme Inhibitors - pharmacokinetics Cardiomyopathy Cardiovascular disease Child Child, Preschool Clinical trials Congenital diseases Design Dose-Response Relationship, Drug Drug Combinations Drug dosages Drug therapy Drug Therapy, Combination Enalapril - administration & dosage Enalapril - pharmacokinetics Enzymes FDA approval Female Follow-Up Studies Heart Heart diseases Heart failure Heart Failure - drug therapy Heart Failure - etiology Heart Failure - metabolism Heart transplantation Humans Infant Infant, Newborn Male Mortality Myocardial Ischemia - complications Myocardial Ischemia - diagnosis Myocardial Ischemia - physiopathology Neprilysin Patients Pediatrics Pharmacodynamics Pharmacokinetics Pharmacology Prospective Studies Quality of life Systole Tetrazoles - administration & dosage Tetrazoles - pharmacokinetics Time Factors Transplants & implants Treatment Outcome Valsartan - administration & dosage Valsartan - pharmacokinetics Ventricle Ventricular Dysfunction, Left - complications Ventricular Dysfunction, Left - diagnosis Ventricular Dysfunction, Left - physiopathology Ventricular Function, Left - physiology |
| title | Design for the sacubitril/valsartan (LCZ696) compared with enalapril study of pediatric patients with heart failure due to systemic left ventricle systolic dysfunction (PANORAMA-HF study) |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-01-17T08%3A37%3A16IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_cross&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Design%20for%20the%20sacubitril/valsartan%20(LCZ696)%20compared%20with%20enalapril%20study%20of%20pediatric%20patients%20with%20heart%20failure%20due%20to%20systemic%20left%20ventricle%20systolic%20dysfunction%20(PANORAMA-HF%20study)&rft.jtitle=The%20American%20heart%20journal&rft.au=Shaddy,%20Robert&rft.date=2017-11&rft.volume=193&rft.spage=23&rft.epage=34&rft.pages=23-34&rft.issn=0002-8703&rft.eissn=1097-6744&rft_id=info:doi/10.1016/j.ahj.2017.07.006&rft_dat=%3Cproquest_cross%3E1965447487%3C/proquest_cross%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=1965447487&rft_id=info:pmid/29129252&rft_els_id=S0002870317302053&rfr_iscdi=true |