Efficacy and safety of a cryopreserved formulation of autologous CD34+ haematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for human ADA gene in subjects with Severe Combined Immunodeficiency (SCID) due to Adenosine Deaminase Deficiency

Great Ormond Street Hospital for Children NHS Trust0 个研究点目标入组 10 人开始时间: 2017年7月3日最近更新: 2019年6月30日

适应症

试验速览

暂无简介。

•1.Provision of written informed consent prior to any study related procedures. In this study consent must be provided by the parents/legal guardians and, where applicable according to local laws, a signed assent from the child,
•2.Subjects =30 days and <18 years of age,
•3.With a diagnosis of ADA-SCID based on evidence of ADA deficiency or evidence of ADA-SCID,
•4.Ineligible for or with no available matched family donor for allogeneic BM transplantation, defined as the absence of a medically eligible HLA-identical sibling or family donor, with normal immune function, who could serve as an allogeneic bone marrow donor.
•5.Females of child-bearing age will be required to provide a negative pregnancy test 30 days prior to Visit 2,
•6.Subjects and their parents/legal guardians must be willing and able to comply with study restrictions and to remain at the clinic for the required duration during the study period and willing to return to the clinic for the follow up evaluation as specified in the protocol.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 10
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1.Ineligible for autologous haematopoietic stem cell (HSC) procedure.
•2.Other conditions which in the opinion of the Principal Investigator and/or Co Investigators, contraindicate the harvest of bone marrow, the administration of busulfan and the infusion of transduced cells, or which indicate an inability of the subject or subject’s parent/legal guardian to comply with the protocol
•3.Haematologic abnormality,
•4.Pulmonary abnormality,
•5.Cardiac abnormality,
•6.Neurologic abnormality,
•7.Renal abnormality,
•8.Hepatic/gastrointestinal abnormality,
•9.Oncologic disease,
•10.Known sensitivity to Busulfan,
•11.Confirmation of an infectious disease by deoxyribonucleic acid (DNA) PCR positive at time of assessment for the following: HIV-1, Hepatitis B, Parvovirus B19,
•12.The subject is pregnant or has a major congenital anomaly,
•13.Is likely to require treatment during the study with drugs that are not permitted by the study protocol,
•14.The subject has previously received another form of gene therapy.

发起方

Great Ormond Street Hospital for Children NHS Trust