GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

被引：31

作者：

Nathwani, Amit C. ^{[1
,2
,3
,4
]}

Tuddenham, Edward ^{[5
]}

Chowdary, Pratima ^{[6
]}

McIntosh, Jenny ^{[4
]}

Lee, Doyoung ^{[5
]}

Rosales, Cecilia ^{[5
]}

Phillips, Mark ^{[7
]}

Pie, Jun ^{[8
]}

Zhou Junfang ^{[9
]}

Meagher, Michael M. ^{[10
]}

Reiss, Ulrike ^{[11
]}

Davidoff, Andrew M. ^{[12
]}

Morton, Chris L. ^{[9
]}

Riddell, Anne ^{[2
,3
]}

机构：

[1] Freeline, Stevenage, Herts, England

[2] Royal Free NHS Fdn Trust, Katharine Dormandy Haemophilia Ctr, London, England

[3] Royal Free NHS Fdn Trust, Thrombosis Unit, London, England

[4] UCL, Canc Inst, Dept Haematol, London, England

[5] UCL Canc Inst, Dept Haematol, London, England

[6] Royal Free Hosp, KD Haemophilia & Thrombosis Ctr, London, England

[7] UCL, UCL Canc Inst, London, England

[8] KD Haemophilia & Thrombosis Ctr, London, England

[9] St Jude Childrens Res Hosp, Dept Surg, 332 N Lauderdale St, Memphis, TN 38105 USA

[10] St Jude Childrens Res Hosp, Childrens GMP LLC, 332 N Lauderdale St, Memphis, TN 38105 USA

[11] St Jude Childrens Rsch Hosp, Dept Surg, Memphis, TN USA

[12] St Jude Childrens Res Hosp, 332 N Lauderdale St, Memphis, TN 38105 USA

来源：

BLOOD | 2018年 / 132卷

关键词：

D O I：

10.1182/blood-2018-99-118256

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

489

引用

页数：3

共 50 条

[41] High dose samarium-153 EDTMP (ethylenediaminetetramethylene phosphonic acid) and Melphalan chemotherapy with peripheral blood stem cell rescue for therapy of patients with multiple myeloma: Phase I and II clinical trial preliminary results.
Wiseman, GA
Dispensieri, A
Lacy, MQ
Gertz, MA
JOURNAL OF NUCLEAR MEDICINE, 2003, 44 (05) : 174P - 175P
[42] HIGH-DOSE PLASMID-MEDIATED VASCULAR ENDOTHELIAL GROWTH FACTOR GENE TRANSFER IN PATIENTS WITH SEVERE CORONARY ARTERY DISEASE (GENESIS-I). SIX MONTHS RESULTS OF THE FIRST PHASE I LATIN AMERICAN TRIAL OF GENE THERAPY IN MYOCARDIAL ISCHEMIA
Favaloro, Liliana Ethel
Diez, Mirta
Mendiz, Oscar
Valdivieso, Leon
Bercovich, Andres
Criscuolo, Marcelo E.
Janavel, Gustavo Vera
Crottogini, Alberto J.
JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY, 2010, 55 (10)
[43] Preliminary updated results of a phase I/II trial using trimodality therapy in patients with post-prostatectomy high risk pathologic (p) T2-3N0M0 prostate cancer
Kumar, Parvesh
Van Veldhuizen, Peter J.
Thompson, Mark
Shen, Xinglei
Coster, James
Pinskl, Jacek
JOURNAL OF CLINICAL ONCOLOGY, 2014, 32 (15)
[44] Dose escalation results from a first-in-human, phase I/II study of GB263T, a novel EGFR/cMET/cMET trispecific antibody, in patients with advanced EGFR-mutated (EGFRm) non-small cell lung cancer (NSCLC)
Yang, Jin-Ji
Pavlakis, Nick
Xie, Fan
Li, Tong
Wu, Yi-Long
MOLECULAR CANCER THERAPEUTICS, 2023, 22 (12)
[45] Results of a Phase I Dose Escalation Trial (WJOG3305) Using Accelerated Hyperfractionated 3D Conformal Radiation Therapy (AHF-3DCRT) and Concurrent Chemotherapy for Unresectable Stage III Non-Small Cell Lung Cancer (NSCLC)
Tsujino, Kayoko
Tada, Takuhito
Fukuda, Haruyuki
Nishimura, Yasumasa
Kokubo, Masaki
Soejima, Toshimori
Takada, Yoshie
Nishikawa, Tatsuyuki
Nakagawa, Kazuhiko
JOURNAL OF THORACIC ONCOLOGY, 2010, 5 (12) : S381 - S381
[46] Three-Year Safety Results of SAR422459 (EIAV-ABCA4) Gene Therapy in Patients With ABCA4-Associated Stargardt Disease: An Open-Label Dose-Escalation Phase I/IIa Clinical Trial, Cohorts 1-5
Parker, Maria a.
Erker, Laura r.
Audo, I. S. A. B. E. L. L. E.
Choi, D. O. N. G. S. E. O. K.
Mohand-said, S. A. D. D. E. K.
Sestakauskas, K. A. S. T. Y. T. I. S.
Benoit, P. A. T. R. I. C. K.
Appelqvist, T. E. R. E. N. C. E.
Krahmer, M. E. L. I. S. S. A.
Segaut-prevost, C. A. R. O. L. I. N. E.
Lujan, Brandon j.
Faridi, A. M. B. A. R.
Chegarnov, Elvira n.
Steinkamp, Peter n.
Ku, C. R. I. S. T. Y.
Palma, Mariana matioli da
Barale, Pierre-olivier
Ayelo-scheer, S. A. R. A. H.
Lauer, A. N. D. R. E. A. S.
Stout, T. I. M.
Wilson, David j.
Weleber, Richard g.
Pennesi, Mark e.
Sahel, JOSe A. L. A. I. N.
Yang, P. A. U. L.
AMERICAN JOURNAL OF OPHTHALMOLOGY, 2022, 240 : 285 - 301
[47] A first-in-human, phase 1, open-label trial of FX201, an intra-articular, helper-dependent adenoviral gene therapy for osteoarthritis - preliminary evaluation of clinical activity in 8 patients from the mid-dose cohort of a single ascending dose study design
Kivitz, A.
Senter, B.
Golod, D.
Parenti, D.
Cinar, A.
Hong, W.
HUMAN GENE THERAPY, 2021, 32 (19-20) : A73 - A73
[48] Preliminary Results from a First-in-human Phase I/II Gene Therapy Study (FOCUS) of Subretinally Delivered GT005, an Investigational AAV2 Vector, in Patients with Geographic Atrophy Secondary to Age-related Macular Degeneration
Nielsen, Jared
MacLaren, Robert E.
Heier, Jeffrey S.
Steel, David
Ivanova, Tsveta
Sivaprasad, Sobha
Stanga, Paulo
Bailey, Clare
Issa, Peter Charbel
Mendonca, Luisa
Francis, James
Curtiss, Darin
Hughes, Jane
Waheed, Nadia K.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2022, 63 (07)
[49] MBS303, a Novel 2:1 CD20xCD3 Bispecific Antibody, Demonstrated a Favorable Safety and Potent Antitumor Activity in Patients with Relapsed/Refractory (R/R) B-Cell Non-Hodgkin Lymphoma: Preliminary Results from a Phase I , Dose-Escalation Trial
Song, Yuqin
Yang, Haiyan
Jia, Youchao
Sheng, Lixia
Deng, Lijuan
Chen, Xi
Tang, Yongjing
Liu, Xifang
Liu, Sijun
Wei, Min
Li, Feng
BLOOD, 2024, 144 : 6481 - 6482
[50] A novel fully human monoclonal antibody against epidermal growth factor receptor (EGFR). First clinical and FDG-PET imaging results from a phase I/II trial conducted by the Danish Head and Neck Cancer Study Group (DAHANCA) in patients with squamous cell carcinoma of the head and neck (SCCHN).
Bastholt, L
Specht, L
Jensen, K
Brun, E
Loft, A
Petersen, J
Kastberg, H
Eriksen, JG
JOURNAL OF CLINICAL ONCOLOGY, 2005, 23 (16) : 507S - 507S

← 1 2 3 4 5 →