65. 原発性免疫不全症候群 Primary immunodeficiency Clinical trials / Disease details
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
Showing 1 to 4 of 4 diseases
| 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) Gene therapy study using a frozen formulation of OTL-103 in patients with Wiskott-Aldrich Syndrome ( ... | 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. A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved ... | 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] Wiskott-Aldrich Syndrome MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodef ... | 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: Product Name: OTL-103Dispersion for Infusion Product Code: OTL-103 INN or Proposed INN: Other hematolo ... | Orchard Therapeutics Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 6 | Phase 3 | Italy | ||
| 2 | JPRN-UMIN000030806 | 2018/01/17 | 15/01/2018 | A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome | A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome - Hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome - H ... | Wiskott-Aldrich syndrome | WASP cDNA-transduced autologous hematopoietic stem cells are administered to patients affected by WAS after the administration of rituximab and preconditioning chemotherapy including Fludarabine and Busulfan. 1. Rituximab (day-22) 375 mg/m2 2. Preconditioning chemotherapy Fludarabine 30mg/m2 x 2 (day-3, day-2) Busulfan cumulative target AUC 48000 ng/mL*h (day-3 to -1, every 6 hours) 3. Infusion of WASP cDNA-transduced CD34 positive HSC 5 x 10^6/kg (at least 3 x 10^6/kg) WASP cDNA-transduced autologous hematopoietic stem cells are administered to patients affected by WA ... | National Center for Child Heath and Development | NULL | Complete: follow-up continuing | Not applicable | Not applicable | Male | 3 | Phase 1,2 | Japan |
| 3 | NCT02789397 (ClinicalTrials.gov) | May 2, 2016 | 16/5/2016 | Treatment of Granulomatous and Lymphocytic Interstitial Lung Disease in Patients With Common Variable Immunodeficiency Treatment of Granulomatous and Lymphocytic Interstitial Lung Disease in Patients With Common Variabl ... | Clinical Trial to Assess the Efficacy of Rituximab and Azathioprine in the Treatment of Granulomatous and Lymphocytic Interstitial Lung Disease (GLILD) in Adult Patients With Common Variable Immunodeficiency (CVID) Clinical Trial to Assess the Efficacy of Rituximaband Azathioprine in the Treatment of Granulomatous ... | Granulomatous and Lymphocytic Interstitial Lung Disease | Drug: Rituximab (RTX) and Azathioprine (AZA);Drug: Placebos | Medical College of Wisconsin | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 2 | NULL |
| 4 | EUCTR2009-017346-32-IT (EUCTR) | 15/03/2010 | 26/04/2010 | HAEMATOPOIETIC STEM CELL GENE THERAPY | A PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME - TIGET-WAS A PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROM ... | Wiskott-Aldrich Syndrom 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] Wiskott-Aldrich Syndrom MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodefi ... | Trade Name: FLUDARABINA TEVA - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 1 FLACONCINO DI VETRO DA 2 ML Product Name: na Product Code: [na] INN or Proposed INN: FLUDARABINA Other descriptive name: FLUDARABINA Trade Name: MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - FLACONCINO (VETRO) - 24 MG/1.2 ML 1 FLACONCINO Product Name: PLERIXAFOR Product Code: [na] INN or Proposed INN: PLERIXAFOR Other descriptive name: Plerixafor Trade Name: MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML Product Name: na Product Code: [na] INN or Proposed INN: LENOGRASTIM Trade Name: MABTHERA - 2 FIALE 100 MG 10 ML Product Name: RITUXIMAB Product Code: [na] INN or Proposed INN: RITUXIMAB Other descriptive name: RITUXIMAB Trade Name: BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI Product Name: na Product Code: [na] Trade Name: FLUDARABINATEVA - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 1 FLACO ... | Orchard Therapeutics (Europe) Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 8 | Phase 1;Phase 2 | Italy |