Updated Follow-up of the Alta Study, a Phase 1/2, Open Label, Adaptive, Dose-Ranging Study to Assess the Safety and Tolerability of SB-525 Gene Therapy in Adult Patients with Severe Hemophilia A

被引:16
|
作者
Konkle, Barbara A. [1 ,2 ]
Stine, Kimo [3 ]
Visweshwar, Nathan [4 ]
Harrington, Thomas J. [5 ]
Leavitt, Andrew D. [6 ]
Giermasz, Adam [7 ]
Arkin, Steven [8 ]
Di Russo, Gregory [8 ]
Snyder, Ashley [9 ]
Woolfson, Adrian [9 ]
Rouy, Didier [9 ]
机构
[1] Bloodworks Northwest, Seattle, WA USA
[2] Univ Washington, Seattle, WA 98195 USA
[3] Arkansas Childrens Hosp, 800 Marshall St, Little Rock, AR 72202 USA
[4] Univ S Florida, Div Hematol & Med Oncol, Dept Internal Med, Tampa, FL 33620 USA
[5] Univ Miami, Miller Sch Med, Miami, FL 33136 USA
[6] Univ Calif San Francisco, Dept Lab Med, San Francisco, CA USA
[7] Univ Calif Davis, Sacramento, CA 95817 USA
[8] Pfizer Inc, Cambridge, MA USA
[9] Sangamo Therapeut, Brisbane, CA USA
关键词
D O I
10.1182/blood-2019-122143
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
2060
引用
收藏
页数:3
相关论文
共 50 条
  • [41] Macrophage Therapy for Acute Liver Injury (MAIL): a study protocol for a phase 1 randomised, open-label, dose-escalation study to evaluate safety, tolerability and activity of allogeneic alternatively activated macrophages in patients with paracetamol-induced acute liver injury in the UK
    Humphries, Christopher
    Addison, Melisande
    Aithal, Guruprasad
    Boyd, Julia
    Briody, Lesley
    Campbell, John D. M.
    Candela, Maria Elena
    Clarke, Ellise
    Coulson, James
    Downing-James, Nicholas
    Fontana, Robert John
    Geddes, Ailsa
    Grahamslaw, Julia
    Grant, Alison
    Heye, Anna
    Hutchinson, James A.
    Jones, Ashley
    Mitchell, Fiona
    Moore, Joanna
    Riddell, Alice
    Rodriguez, Aryelly
    Thomas, Angela
    Tucker, Garry
    Walker, Kim
    Weir, Christopher J.
    Woods, Rachel
    Zahra, Sharon
    Forbes, Stuart J.
    Dear, James W.
    BMJ OPEN, 2024, 14 (12):
  • [42] Phase 2, Open-Label Study of Ciltacabtagene Autoleucel, an Anti-BCMA CAR-T Cell Therapy, in Chinese Patients with Relapsed/Refractory Multiple Myeloma (CARTIFAN-1): 26-Month Median Follow-up
    Mi, Jian-Qing
    Zhao, Wanhong
    Jing, Hongmei
    Fu, Weijun
    Hu, Jianda
    Chen, Lijuan
    Zhang, Yiwen
    Yao, Dan
    Li, Hui
    Schecter, Jordan M.
    Yang, Fan
    Sun, Huabin
    Zhuang, Sen Hong
    Xu, Da
    Luo, Tracy
    Fan, Xiaohu
    Niu, Ting
    Jin, Jie
    Chen, Sai-Juan
    BLOOD, 2022, 140 : 7542 - 7544
  • [43] Phase 1/2 open-label, multi-center study to assess the safety, tolerability and efficacy of a single dose of PBGM01 delivered into the cisterna magna of subjects with type 1 (early onset) and type 2a (late onset) infantile GM1 gangliosidosis
    Jarnes, Jeanine R.
    Day-Salvatore, Debra L.
    Ficicioglu, Can
    Hastings, Caroline
    Bernard, Genevieve
    Baruteau, Julien
    Ezgu, Fatih
    Giugliani, Roberto
    Inbar-Feigenberg, Michal
    Whitley, Chester B.
    Ni, Yan
    Ballard, Victoria L.
    Haws, Thomas F.
    Weinstein, David A.
    MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S62 - S63
  • [44] CHANGE IN FUNCTIONAL ABILITIES IN A PATIENT RECEIVING INVESTIGATIONAL ENZYME REPLACEMENT THERAPY (ERT) FOR MUCOPOLYSACCHARIDOSIS IVA (MPS IVA) IN A PHASE 1/2 MULTICENTER, OPEN-LABEL, DOSE-ESCALATION STUDY TO EVALUATE THE SAFETY, TOLERABILITY AND EFFICACY OF BMN 110
    Hendriksz, C. J.
    Decker, C. J.
    Cheng, S.
    Hutchinson, J.
    Takkele, H.
    JOURNAL OF INHERITED METABOLIC DISEASE, 2011, 34 : S210 - S210
  • [45] A phase 1/2 study with open-label, dose escalation phase followed by single-arm expansion at the maximum tolerated dose to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of NT219 injection alone and in combination with cetuximab in adults with advanced solid tumors and head and neck cancer.
    Bessudo, Alberto
    Cohen, Ezra E. W.
    Gutierrez, Rodolfo
    Johnson, Daniel H.
    Rosenberg, Ari
    Sukari, Ammar
    Stemmer, Salomon M.
    Weinberg, Benjamin Adam
    Reuveni, Hadas
    Schickler, Michael
    Liang, Bertrand C.
    JOURNAL OF CLINICAL ONCOLOGY, 2021, 39 (15)
  • [46] A phase 2 open-label study to evaluate safety, tolerability, efficacy, and pharmacodynamics of JNJ-73763989, nucleos (t)ide analogs, and a low-dose PD-1 inhibitor in patients with chronic hepatitis B-Interim results of the OCTOPUS-1 study
    Asselah, Tarik
    Fung, Scott K.
    Akhan, Sila
    Chuang, Wan-Long
    Buti, Maria
    Brunetto, Maurizia
    Agarwal, Kosh
    Diba, Camellia
    Jerzorwski, John
    Kakuda, Thomas
    Nalpas, Catherine
    Guinard-Azadian, Carine
    Verbinnen, Thierry
    Lathouwers, Erkki
    De Creus, An
    Lenz, Oliver
    Biermer, Michael
    JOURNAL OF HEPATOLOGY, 2024, 80 : S27 - S28
  • [47] MP0274-CP101: A phase 1, first-in-human, single-arm, multi-center, open-label, dose escalation study to assess safety, tolerability, and pharmacokinetics of MP0274 in patients with advanced HER2-positive solid tumors
    Baird, Richard
    Omlin, Aurelius
    Kiemle-Kallee, Joachim
    Fiedler, Ulrike
    Zitt, Christof
    Feurstein, Daniel
    Herbst, Joerg
    Dawson, Keith
    vom Baur, Elmar
    Stumpp, Michael
    Hermann, Frank
    Harstrick, Andreas
    Schneeweiss, Andreas
    CANCER RESEARCH, 2018, 78 (04)
  • [48] HPN217-3001: A Phase 1/2 Open-Label, Multicenter, Dose Escalation and Dose Expansion Study of the Safety, Tolerability, and Pharmacokinetics of HPN217, a Bcma-Targeting T-Cell Engager, in Patients with Relapsed/Refractory Multiple Myeloma
    Schade, Henning
    Madan, Sumit
    Medvedova, Eva
    Nath, Rajneesh
    Knapp, Lisa
    Lemon, Bryan
    Sun, Liping Laura
    BLOOD, 2020, 136
  • [49] Update: a phase 1/2, open-label study to assess safety, tolerability, biodistribution, radiation dosimetry and PET imaging characteristics of [18F]FPyGal in comparison to in-vitro diagnostic for the assessment of senescence in oncological patients (NCT04536454)
    LaFougere, Christian
    Gueckel, Brigitte
    Dittmann, Helmut
    Trautwein, Nils
    Hinterleitner, Martina
    Cotton, Jonathan
    Reischl, Gerald
    Kienzle, Gabriele
    Singer, Stephan
    Gani, Cihan
    Lauer, Ulrich
    Pichler, Bernd
    Zender, Lars
    CANCER RESEARCH, 2023, 83 (08)
  • [50] A five-arm, open-label, phase I/lb study to assess safety and tolerability of the oral MEK1/MEK2 inhibitor trametinib (GSK1120212) in combination with chemotherapy or erlotinib in patients with advanced solid tumors.
    Becerra, Carlos
    Infante, Jeffrey R.
    Garbo, Lawrence E.
    Gordon, Michael S.
    Smith, David A.
    Braiteh, Fadi S.
    Gandara, David R.
    Jotte, Robert M.
    Reckamp, Karen L.
    Janku, Filip
    Burris, Howard A.
    Bellew, Kevin M.
    Gardner, Olivia S.
    Liu, Li
    Cox, Donna S.
    Peddareddigari, Vijay Gopal Reddy
    Blumenschein, George R.
    JOURNAL OF CLINICAL ONCOLOGY, 2012, 30 (15)