Autologous ex vivo gene therapy products based on the efs lv encoding for the human adenosine deaminase (ada) gene (efs-ada lv) (DrugBank: Adenosine)
3 diseases告示番号 | 疾患名(ページ内リンク) | 臨床試験数 |
---|---|---|
13 | 多発性硬化症/視神経脊髄炎 | 0 |
58 | 肥大型心筋症 | 0 |
65 | 原発性免疫不全症候群 | 1 |
No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
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1 | NCT04049084 (ClinicalTrials.gov) | September 26, 2019 | 6/8/2019 | An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID | An Observational Long-term Follow-up Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID) | Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency (SCID) | Biological: autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV) | Orchard Therapeutics | University of California, Los Angeles;Great Ormond Street Hospital for Children NHS Foundation Trust | Enrolling by invitation | N/A | N/A | All | 70 | United States;United Kingdom |