There is no recommended INN ( 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
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agemin
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PhaseCountries
1EUCTR2017-001275-23-GB
(EUCTR)
21/09/201703/07/2017A clinical trial to study the effects of genetically modified patients' CD34+ cellsEfficacy 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 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: 20.1;Level: LLT;Classification code 10066372;Term: ADA deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: cryopreserved EFS-ADA LV transduced patient CD34+ cells
Product Code: OTL-101
INN or Proposed INN: There is no recommended INN
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 TrustNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
10Phase 2United Kingdom