Other hematological Agents ( 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 | EUCTR2018-003842-18-IT (EUCTR) | 08/01/2019 | 19/11/2018 | Gene therapy study using a frozen formulation of OTL-103 in patients with Wiskott-Aldrich Syndrome (WAS) | A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved Autologous CD34+ Cells Transduced with Lentiviral Vector encoding WAS cDNA in Subjects with Wiskott-Aldrich Syndrome (WAS). - Clinical study using cryopreserved OTL-103 for treatment of WAS. | Wiskott-Aldrich Syndrome MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: OTL-103 Dispersion for Infusion Product Code: OTL-103 INN or Proposed INN: Other hematological Agents Other descriptive name: Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with lentiviral vector that encodes for the human Wiskott Aldrich Syndrome (WAS) cDNA sequence Trade Name: Busilvex INN or Proposed INN: BUSULFAN Other descriptive name: NA Trade Name: Fludarabina Accord INN or Proposed INN: FLUDARABINE Other descriptive name: NA Trade Name: MabThera INN or Proposed INN: RITUXIMAB Other descriptive name: NA Trade Name: Mozobil, INN or Proposed INN: plerixafor Other descriptive name: PLERIXAFOR Trade Name: MYELOSTIM Product Name: granulocyte colony stimulating factor (G-CSF) INN or Proposed INN: | Orchard Therapeutics Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 6 | Phase 3 | Italy |