A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study of Mitapivat in Patients with Sickle Cell Disease: RISE UP Phase 2 Results

被引:2
|
作者
Idowu, Modupe [1 ]
Otieno, Lucas [2 ]
Dumitriu, Bogdan [3 ]
Lobo, Clarisse L. C. [4 ]
Thein, Swee Lay [5 ]
Andemariam, Biree [6 ]
Nnodu, Obiageli E. [7 ]
Inati, Adlette [8 ]
Glaros, Alexander K. [9 ]
Olalla Saad, Sara Teresinha [10 ,11 ]
Bartolucci, Pablo [12 ]
Colombatti, Raffaella [13 ]
Taher, Ali T. [14 ]
Abboud, Miguel R. [15 ]
Oluyadi, Abdulafeez [16 ]
Iyer, Varsha [16 ]
Yin, Ophelia [17 ,18 ]
Morris, Susan [16 ]
Yates, Amber M. [16 ]
Shao, Hui [16 ]
Patil, Spurthi [16 ]
Urbstonaitis, Rolandas [16 ]
Zaidi, Ahmar U. [16 ]
Smith, Wally R. [19 ]
机构
[1] UTHealth, McGovern Med Sch, Houston, TX USA
[2] Victoria Biomed Res Inst, Kisumu, Kenya
[3] Mid Atlantic Permanente Med Grp, Largo, MD USA
[4] HEMORIO, Div Res, Rio De Janeiro, Brazil
[5] NHLBI, Sickle Cell Branch, Bethesda, MD USA
[6] Univ Connecticut Hlth, New England Sickle Cell Inst, Farmington, CT USA
[7] Univ Abuja, Dept Haematol & Blood Transfus, Coll Hlth Sci, Ctr Excellence Sickle Cell Dis Res & Training, Abuja, Nigeria
[8] Lebanese Amer Univ, Gilbert & Rose Marie Chagoury Sch Med, Byblos & NINI Hosp, Tripoli, Lebanon
[9] Childrens Hosp Michigan, Div Hematol Oncol, Detroit, MI USA
[10] Univ Estadual Campinas, Hematol & Transfus Med Ctr, Hemoctr, Campinas, SP, Brazil
[11] Univ Campinas Unicamp, Campinas, Brazil
[12] Mondor Univ Hosp, Reference Ctr Sickle Cell Dis, Creteil, France
[13] Univ Padua, Pediat Hematol Oncol Unit, Padua, Italy
[14] Amer Univ Beirut, Med Ctr, Beirut, Lebanon
[15] Amer Univ, Dept Pediat & Adolescent Med, Beirut Med Ctr, Beirut, Lebanon
[16] Agios Pharmaceut Inc, Cambridge, MA USA
[17] ITeos Therapeut, Watertown, MA USA
[18] Agios Pharmaceut Inc, Time Res, Cambridge, MA USA
[19] Virginia Commonwealth Univ, Richmond, VA USA
关键词
D O I
10.1182/blood-2023-187033
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
引用
收藏
页数:6
相关论文
共 50 条
  • [31] Isoquercetin for thromboinflammation in sickle cell disease: a randomized double-blind placebo-controlled trial
    Lizarralde-Iragorri, Maria A.
    Gopalan, Bindu Parachalil
    Merriweather, Brenda
    Brooks, Jennifer
    Hill, Mai
    Lovins, Dianna
    Pierre-Charles, Ruth
    Cullinane, Ann
    Dulau-Florea, Alina
    Lee, Duck-Yeon
    Villasmil, Rafael
    Jeffries, Neal
    Shet, Arun S.
    BLOOD ADVANCES, 2024, 8 (01) : 172 - 182
  • [32] Transdermal buprenorphine in the treatment of chronic pain: Results of a Phase III, multicenter, randomized, double-blind, placebo-controlled study
    Sorge, J
    Sittl, R
    CLINICAL THERAPEUTICS, 2004, 26 (11) : 1808 - 1820
  • [33] A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Talactoferrin in Patients With Severe Sepsis
    Guntupalli, Kalpalatha
    Dean, Nathan
    Morris, Peter E.
    Bandi, Venkata
    Margolis, Benjamin
    Rivers, Emanuel
    Levy, Mitchell
    Lodato, Robert F.
    Ismail, Preeti M.
    Reese, Amber
    Schaumberg, John P.
    Malik, Rajesh
    Dellinger, R. Phillip
    CRITICAL CARE MEDICINE, 2013, 41 (03) : 706 - 716
  • [34] Secukinumab in Giant Cell Arteritis: A Randomized, Parallel-group, Double-blind, Placebo-controlled, Multicenter Phase 2 Trial
    Venhoff, Nils
    Schmidt, Wolfgang
    Bergner, Raoul
    Rech, Juergen
    Unger, Leonore
    Tony, Hans-Peter
    Mendelson, Meryl
    Sieder, Christian
    Maricos, Meron
    Thiel, Jens
    ARTHRITIS & RHEUMATOLOGY, 2021, 73 : 4130 - 4133
  • [35] Daridorexant in Japanese patients with insomnia disorder: A phase 3, randomized, double-blind, placebo-controlled study
    Uchimura, Naohisa
    Taniguchi, Mitsutaka
    Ariyoshi, Yu
    Oka, Yasunori
    Togo, Osamu
    Uchiyama, Makoto
    SLEEP MEDICINE, 2024, 122 : 27 - 34
  • [36] Ustekinumab in Patients with Active Psoriatic Arthritis: Results of the Phase 3, Multicenter, Double-Blind, Placebo-Controlled Psummit I Study
    Kavanaugh, Arthur
    McInnes, Iain B.
    Gottlieb, Alice B.
    Puig, Lluis
    Rahman, Proton
    Ritchlin, Christopher T.
    Li, Shu
    Wang, Yuhua
    Mendelsohn, Alan
    Doyle, Mittie K.
    ARTHRITIS AND RHEUMATISM, 2012, 64 (10): : S1083 - S1084
  • [37] USTEKINUMAB IN PATIENTS WITH ACTIVE PSORIATIC ARTHRITIS: RESULTS OF THE PHASE 3, MULTICENTER, DOUBLE-BLIND, PLACEBO-CONTROLLED PSUMMIT I STUDY
    McInnes, I. B.
    Kavanaugh, A.
    Gottlieb, A. B.
    Puig, L.
    Rahman, P.
    Ritchlin, C.
    Li, S.
    Wang, Y.
    Mendelsohn, A. M.
    Doyle, M. K.
    ANNALS OF THE RHEUMATIC DISEASES, 2013, 71 : 107 - 107
  • [38] Doxycycline in Creutzfeldt-Jakob disease: A phase 2, randomized, double-blind, placebo-controlled trial
    Brandel, Jean-Philippe
    Marcon, Gabriella
    Coudert, Mathieu
    Tettamanti, Mauro
    Welaratne, Arlette
    Giacone, Georgio
    Azimi, Shohreh
    Pietrini, Vladimiro
    Fabreguettes, Jean-Roch
    Imperiale, Daniele
    Aucan, Christophe
    Lucca, Ugo
    Mallet, Alain
    Salmona, Mario
    Zerr, Inga
    Redaelli, Veronica
    Forloni, Gianluigi
    Haik, Stephane
    Tagliavini, Fabrizio
    PRION, 2013, 7 : 62 - 62
  • [39] A Phase II, Randomized, Double-blind, Placebo-controlled Multicenter Trial of Endostar in Patients With Metastatic Melanoma
    Cui, Chuanliang
    Mao, Lili
    Chi, Zhihong
    Si, Lu
    Sheng, Xinan
    Kong, Yan
    Li, Siming
    Lian, Bin
    Gu, Kangsheng
    Tao, Min
    Song, Xin
    Lin, Tongyu
    Ren, Xiubao
    Qin, Shukui
    Guo, Jun
    MOLECULAR THERAPY, 2013, 21 (07) : 1456 - 1463
  • [40] Efficacy and safety of anamorelin HCl in NSCLC patients: Results from a randomized, double-blind, placebo-controlled, multicenter phase II study
    Temel, J.
    Bondarde, S.
    Jain, M.
    Allen, S.
    Mann, W.
    EUROPEAN JOURNAL OF CANCER, 2013, 49 : S269 - S270