EF1aS-ADA lentiviral vector gene modified autologous CD34+ cells ( DrugBank: - )

---

1 disease

告示番号	疾患名（ページ内リンク）	臨床試験数
65	原発性免疫不全症候群	1

65. 原発性免疫不全症候群

---

臨床試験数 : 500 /  薬物数 : 614 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 217

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
1	EUCTR2010-024253-36-GB (EUCTR)	03/08/2012	09/05/2012	A clinical trial to study the effects of genetically modified patients' CD34+ cells	Phase I/II, historical controlled, open-label, non-randomised, single-centre trial to assess the safety and efficacy of EF1aS-ADA lentiviral vector mediated gene modification of autologus CD34+ cells from ADA-deficient individuals - LV Gene Therapy for ADA Deficiency	Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protection from bacteria, viruses, and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. If not treated in a way that restores immune function, children with SCID usually live only a year or two. MedDRA version: 20.0;Level: LLT;Classification code 10066372;Term: ADA deficiency;System Organ Class: 100000012248;Therapeutic area: Diseases [C] - Immune System Diseases [C20]	Product Name: EF1aS-ADA lentiviral vector transduced patient CD34+ cells Product Code: transduced patient CD34+ cells INN or Proposed INN: EF1aS-ADA lentiviral vector gene modified autologous CD34+ cells Other descriptive name: Autologous CD34+ HSCs transduced ex vivo with EFS lentiviral vector encoding for the human ADA gene	Great Ormond Street Hospital for Children NHS Trust	NULL	Not Recruiting			Female: no Male: yes	10	Phase 1;Phase 2	United Kingdom