Effect of intracoronary administration of AAV1/SERCA2a on ventricular remodelling in patients with advanced systolic heart failure: results from the AGENT-HF randomized phase 2 trial

European Journal of Heart Failure

Volumn 19, Issue 11, 2017, Pages 1534-1541

  Hulot, Jean Sébastien ^a   Salem, Joe Elie ^a   Redheuil, Alban ^a   Collet, Jean Philippe ^a   Varnous, Shaida ^a   Jourdain, Patrick ^b   Logeart, Damien ^c   Gandjbakhch, Estelle ^a   Bernard, Claude ^d   Hatem, Stéphane N ^a   Isnard, Richard ^a   Cluzel, Philippe ^a   Le Feuvre, Claude ^a   Leprince, Pascal ^a   Hammoudi, Nadjib ^a   Lemoine, François M ^d   Klatzmann, David ^d   Vicaut, Eric ^e   Komajda, Michel ^a   Montalescot, Gilles ^a,e   Lompré, Anne Marie ^a   Hajjar, Roger J ^f   more..

^a SORBONNE UNIVERSITÉ   (France)

^b Centre Hospitalier Pontoise   (France)

^c University of Paris   (France)

^d ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS   (France)

^e Centre de Recherches Cliniques Psychanalytique   (France)

^f MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

Clinical trial; Gene therapy; Heart failure; Sarcoplasmic reticulum calcium ATPase

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; DEOXYRIBONUCLEASE; PLACEBO; SARCOPLASMIC RETICULUM CALCIUM TRANSPORTING ADENOSINE TRIPHOSPHATASE;

ADULT; AGED; ARTICLE; CLINICAL ARTICLE; COMPUTER ASSISTED TOMOGRAPHY; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; FEMALE; FOLLOW UP; GENE THERAPY; GENE TRANSFER; HEART LEFT VENTRICLE EJECTION FRACTION; HEART LEFT VENTRICLE ENDSYSTOLIC VOLUME; HEART VENTRICLE REMODELING; HUMAN; ISCHEMIC CARDIOMYOPATHY; MALE; MIDDLE AGED; NEW YORK HEART ASSOCIATION CLASS; NONISCHEMIC CARDIOMYOPATHY; PHASE 2 CLINICAL TRIAL; PILOT STUDY; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; SYSTOLIC HEART FAILURE; TREATMENT DURATION; CLINICAL TRIAL; CORONARY BLOOD VESSEL; GENETICS; INTRAARTERIAL DRUG ADMINISTRATION; MULTICENTER STUDY; PATHOPHYSIOLOGY; PHYSIOLOGY; PROCEDURES; TREATMENT OUTCOME;

AGED; CORONARY VESSELS; DOUBLE-BLIND METHOD; FEMALE; GENETIC THERAPY; HEART FAILURE, SYSTOLIC; HUMANS; INFUSIONS, INTRA-ARTERIAL; MALE; MIDDLE AGED; SARCOPLASMIC RETICULUM CALCIUM-TRANSPORTING ATPASES; TREATMENT OUTCOME; VENTRICULAR REMODELING;

EID: 85017426351     PISSN: 13889842     EISSN: 18790844     Source Type: Journal    
DOI: 10.1002/ejhf.826     Document Type: Article

References (19)

1. Roger VL, Go AS, Lloyd-Jones DM, Benjamin EJ, Berry JD, Borden WB, Bravata DM, Dai S, Ford ES, Fox CS, Fullerton HJ, Gillespie C, Hailpern SM, Heit JA, Howard VJ, Kissela BM, Kittner SJ, Lackland DT, Lichtman JH, Lisabeth LD, Makuc DM, Marcus GM, Marelli A, Matchar DB, Moy CS, Mozaffarian D, Mussolino ME, Nichol G, Paynter NP, Soliman EZ, Sorlie PD, Sotoodehnia N, Turan TN, Virani SS, Wong ND, Woo D, Turner MB; American Heart Association Statistics Committee and Stroke Statistics Subcommittee. Heart disease and stroke statistics---2012 update: a report from the American Heart Association. Circulation 2012;125:e2–e220.
2. Maggioni AP, Dahlström U, Filippatos G, Chioncel O, Crespo Leiro M, Drozdz J, Fruhwald F, Gullestad L, Logeart D, Fabbri G, Urso R, Metra M, Parissis J, Persson H, Ponikowski P, Rauchhaus M, Voors AA, Nielsen OW, Zannad F, Tavazzi L; Heart Failure Association of the European Society of Cardiology (HFA). EURObservational Research Programme: regional differences and 1-year follow-up results of the Heart Failure Pilot Survey (ESC-HF Pilot). Eur J Heart Fail 2013;15:808–817.
3. Bers DM. Calcium cycling and signaling in cardiac myocytes. Annu Rev Physiol 2008;70:23–49.
4. Kho C, Lee A, Hajjar RJ. Altered sarcoplasmic reticulum calcium cycling---targets for heart failure therapy. Nat Rev Cardiol 2012;9:717–733.
5. Hasenfuss G, Pieske B. Calcium cycling in congestive heart failure. J Mol Cell Cardiol 2002;34:951–969.
6. 2+-ATPase in a rat model of heart failure. Circulation 2001;104:1424–1429.
7. Kawase Y, Ly HQ, Prunier F, Lebeche D, Shi Y, Jin H, Hadri L, Yoneyama R, Hoshino K, Takewa Y, Sakata S, Peluso R, Zsebo K, Gwathmey JK, Tardif JC, Tanguay JF, Hajjar RJ. Reversal of cardiac dysfunction after long-term expression of SERCA2a by gene transfer in a pre-clinical model of heart failure. J Am Coll Cardiol 2008;51:1112–1119.
8. Hulot JS, Ishikawa K, Hajjar RJ. Gene therapy for the treatment of heart failure: promise postponed. Eur Heart J 2016;37:1651–1658.
9. Zsebo K, Yaroshinsky A, Rudy JJ, Wagner K, Greenberg B, Jessup M, Hajjar RJ. Long-term effects of AAV1/SERCA2a gene transfer in patients with severe heart failure: analysis of recurrent cardiovascular events and mortality. Circ Res 2014;114:101–108.
10. 2+-ATPase in patients with advanced heart failure. Circulation 2011;124:304–313.
11. Greenberg B, Butler J, Felker GM, Ponikowski P, Voors AA, Desai AS, Barnard D, Bouchard A, Jaski B, Lyon AR, Pogoda JM, Rudy JJ, Zsebo KM. Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebo-controlled, phase 2b trial. Lancet 2016;387:1178–1186.
12. Karakikes I, Hadri L, Rapti K, Ladage D, Ishikawa K, Tilemann L, Yi GH, Morel C, Gwathmey JK, Zsebo K, Weber T, Kawase Y, Hajjar RJ. Concomitant intravenous nitroglycerin with intracoronary delivery of AAV1.SERCA2a enhances gene transfer in porcine hearts. Mol Ther 2012;20:565–571.
13. Lang RM, Badano LP, Mor-Avi V, Afilalo J, Armstrong A, Ernande L, Flachskampf FA, Foster E, Goldstein SA, Kuznetsova T, Lancellotti P, Muraru D, Picard MH, Rietzschel ER, Rudski L, Spencer KT, Tsang W, Voigt JU. Recommendations for cardiac chamber quantification by echocardiography in adults: an update from the American Society of Echocardiography and the European Association of Cardiovascular Imaging. Eur Heart J Cardiovasc Imaging 2015;16:233–270.
14. Salem JE, Aissaoui N, Paluszkiewicz L, Morshuis M, Gummert J, Leprince P, Fagon JY, Hulot JS, Diebold B. Impact of inodilator drugs on echocardiographic assessments of left ventricular filling pressure in patients with decompensated end-stage heart failure. Crit Care Med 2014;42:2508–2517.
15. Singh RM, Singh BM, Mehta JL. Role of cardiac CTA in estimating left ventricular volumes and ejection fraction. World J Radiol 2014;6:669–676.
16. Lin FY, Min JK. Assessment of cardiac volumes by multidetector computed tomography. J Cardiovasc Comput Tomogr 2008;2:256–262.
17. Mingozzi F, Anguela XM, Pavani G, Chen Y, Davidson RJ, Hui DJ, Yazicioglu M, Elkouby L, Hinderer CJ, Faella A, Howard C, Tai A, Podsakoff GM, Zhou S, Basner-Tschakarjan E, Wright JF, High KA. Overcoming preexisting humoral immunity to AAV using capsid decoys. Sci Transl Med 2013;5:194ra92.
18. Dismuke DJ, Tenenbaum L, Samulski RJ. Biosafety of recombinant adeno-associated virus vectors. Curr Gene Ther 2013;13:434–452.
19. Greenberg B, Butler J, Felker GM, Ponikowski P, Voors AA, Pogoda JM, Provost R, Guerrero J, Hajjar RJ, Zsebo KM. Prevalence of AAV1 neutralizing antibodies and consequences for a clinical trial of gene transfer for advanced heart failure. Gene Ther 2016;23:313–319.