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
来源：
BLOOD | 2019年 / 134卷
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
  • [21] ELOQUENT-2: Extended safety and efficacy follow-up of the phase 3, randomized, open-label study of elotuzumab in combination with lenalidomide/dexamethasone in patients with relapsed/refractory multiple myeloma
    Dimopoulos, M.
    Lonial, S.
    White, D.
    Moreau, P.
    Palumbo, A.
    San Miguel, J.
    Shpilberg, O.
    Anderson, K.
    Grosicki, S.
    Spicka, I.
    Walter-Croneck, A.
    Magen-Nativ, H.
    Mateos, M-V
    Reece, D.
    Beksac, M.
    Bleickardt, E.
    Poulart, V.
    Katz, J.
    Singhal, A.
    Richardson, P.
    BRITISH JOURNAL OF HAEMATOLOGY, 2016, 173 : 6 - 7
  • [22] The safety, tolerability, and pharmacokinetics of BAT8001 in patients with human epidermal growth factor receptor-2 (HER2)-positive metastatic breast cancer: An open-label, dose-up, phase 1 study
    Wang, Shusen
    Hong, Ruoxi
    Wang, Liye
    Xu, Fei
    Xia, Wen
    CANCER RESEARCH, 2020, 80 (04)
  • [23] AN OPEN-LABEL, DOSE ESCALATION STUDY TO ASSESS THE SAFETY AND EFFICACY OF AAV1-LPLS447X GENE THERAPY WITH ALIPOGENE TIPARVOVEC (AMT-011) FOR PATIENTS WITH SEVERE HYPERTRIGLYCERIDEMIA DUE TO LIPOPROTEIN LIPASE DEFICIENCY (LPLD)
    Gaudet, D.
    Brisson, D.
    Methot, J.
    van Deventer, S.
    ATHEROSCLEROSIS SUPPLEMENTS, 2009, 10 (02)
  • [24] Long Term Outcomes of a Phase 1/2, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)
    Hendriksz, Christian
    Vellodi, Ashok
    Jones, Simon
    Takkele, Hiwot
    Lee, S.
    Chesler, Sarah
    Decker, Celeste
    MOLECULAR GENETICS AND METABOLISM, 2012, 105 (02) : S35 - S35
  • [25] Phase 2 open-label, multicenter, single-arm study of cemiplimab in patients with locally advanced basal cell carcinoma after hedgehog inhibitor therapy: Extended follow-up
    Stratigos, Alexander J.
    Sekulic, Aleksandar
    Peris, Ketty
    Bechter, Oliver
    Prey, Sorilla
    Lewis, Karl D.
    Basset-Seguin, Nicole
    Chang, Anne Lynn S.
    Dalle, Stephane
    Orland, Almudena Fernandez
    Licitra, Lisa
    Robert, Caroline
    Ulrich, Claas
    Hauschild, Axel
    Migden, Michael R.
    Dummer, Reinhard
    Yoo, Suk-Young
    Okoye, Emmanuel
    Bassukas, Ioannis
    Loquai, Carmen
    De Giorgi, Vincenzo
    Eroglu, Zeynep
    Gutzmer, Ralf
    Ulrich, Jens
    Puig, Susana
    Inocencio, Timothy J.
    Chen, Chieh-, I
    Lafontaine, Patrick R.
    Seebach, Frank
    Lowy, Israel
    Fury, Matthew G.
    JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY, 2021, 90 (02) : 414 - 418
  • [26] Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study
    Sardh, Eliane
    Balwani, Manisha
    Rees, David C.
    Anderson, Karl E.
    Jia, Gang
    Sweetser, Marianne T.
    Wang, Bruce
    ORPHANET JOURNAL OF RARE DISEASES, 2024, 19 (01)
  • [27] A phase 1, open-label, dose-finding study to assess the safety and tolerability of U3 1287 (AMG 888), a human monoclonal antibody targeting HER3 in patients with advanced solid tumors
    LoRusso, P.
    Keedy, V.
    Yee, L.
    Oliveira, M.
    Rizvi, N.
    Berlin, J.
    Jin, X.
    Hettmann, T.
    Copigneaux, C.
    Beaupre, D.
    EJC SUPPLEMENTS, 2010, 8 (07): : 76 - 76
  • [28] Results of a phase 1, open-label, dose-escalation study evaluating the safety and tolerability of EZN-3042, a survivin mRNA antagonist, administered with or without docetaxel in adult patients with advanced solid tumors or lymphoma
    Tolcher, Anthony W.
    Patnaik, Amita
    Papadopoulos, Kyriakos P.
    Agnew, James
    Lokiec, Francois M.
    Rezai, Keyvan
    Kalambakas, Stacey
    Buchbinder, Aby
    CANCER RESEARCH, 2011, 71
  • [29] Tremelimumab plus durvalumab retreatment and 4-year outcomes in patients with mesothelioma: a follow-up of the open label, non-randomised, phase 2 NIBIT-MESO-1 study
    Calabro, Luana
    Rossi, Giulia
    Morra, Aldo
    Rosati, Claudio
    Cutaia, Ornella
    Daffina, Maria Grazia
    Altomonte, Maresa
    Giacomo, Anna Maria Di
    Casula, Milena
    Fazio, Carolina
    Palmieri, Giuseppe
    Giannarelli, Diana
    Covre, Alessia
    Maio, Michele
    LANCET RESPIRATORY MEDICINE, 2021, 9 (09): : 969 - 976
  • [30] A first-in- human Phase 1/1b multicenter, open-label dose escalation study to assess safety and tolerability of PMD-026, a first-in-class oral RSK inhibitor, in metastatic breast cancer patients
    Beeram, Murali
    Wang, Judy S.
    Chalasani, Pavani
    Mina, Lida
    Patnaik, Amita
    Pambid, Mary Rose
    Jayanthan, Aarthi
    Huynh, My-my
    Los, Gerrit
    Dunn, Sandra E.
    Dorr, F. Andrew
    CANCER RESEARCH, 2021, 81 (04)
12345