PLERIXAFOR ( DrugBank: Plerixafor )
4 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 20 | 副腎白質ジストロフィー | 1 |
| 65 | 原発性免疫不全症候群 | 8 |
| 285 | ファンコニ貧血 | 5 |
| 299 | 嚢胞性線維症 | 1 |
20. 副腎白質ジストロフィー
臨床試験数 : 61 / 薬物数 : 90 - (DrugBank : 31) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 126
| 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 | EUCTR2018-001145-14-DE (EUCTR) | 23/01/2020 | 27/11/2018 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD) | A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) | Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lenti-D Drug Product INN or Proposed INN: elivaldogene autotemcel Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA INN or Proposed INN: plerixafor Other descriptive name: PLERIXAFOR | bluebird bio, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 35 | Phase 3 | France;United States;Netherlands;Germany;United Kingdom;Italy |
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 | NCT03547830 (ClinicalTrials.gov) | April 13, 2019 | 24/5/2018 | Plerixafor/G-CSF as Additional Agents for Conditioning Before HSCT in CGD Patients | A Clinical Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure in Patients With Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Plerixafor;Drug: Gcsf | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Recruiting | 1 Month | 24 Years | All | 17 | Phase 2 | Russian Federation |
| 2 | 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 | ||
| 3 | NCT03019809 (ClinicalTrials.gov) | June 2016 | 11/1/2017 | A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients | A Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure After Transplantation With TCR Alpha/Beta Grafts Depletion in Patients With Wiskott-Aldrich Syndrome. | Wiskott-Aldrich Syndrome;Hematopoietic Stem Cell Transplantation;Graft Failure | Biological: G-CSF for Conditioning before HSCT.;Biological: Plerixafor for Conditioning before HSCT. | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Unknown status | 1 Month | 19 Years | All | 30 | Phase 2 | Russian Federation |
| 4 | NCT03055247 (ClinicalTrials.gov) | November 6, 2015 | 21/7/2016 | Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD | A Multicentric, Exploratory, Non-randomised, Non-controlled, Prospective, Open-label Phase II Study Evaluating Safety and Efficacy of IBU, G-CSF and Plerixafor as Stem Cell Mobilization Regimen in Patients Affected by X-CGD | Chronic Granulomatous Disease X-linked (X-CGD) | Drug: Ibuprofen;Drug: Myelostim;Drug: Mozobil | IRCCS San Raffaele | Fondazione Telethon | Recruiting | 18 Years | 45 Years | Male | 3 | Phase 2 | Italy |
| 5 | EUCTR2015-002356-27-IT (EUCTR) | 16/10/2015 | 29/07/2015 | Evaluation of safety and efficacy of the combination of Ibuprofen (IBU), G-CSF and Plerixafor as a stem cell mobilization regimen in patients affected by XCGD | A multicentric, exploratory, non-randomised, non-controlled, prospective, open-label phase II, study evaluating safety and efficacy of IBU, G-CSF and Plerixafor as a stem cell mobilization regimen in patients affected by X-CGD. | X-linked chronic granulomatous disease MedDRA version: 18.0;Level: PT;Classification code 10008906;Term: Chronic granulomatous disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Ibuprofen INN or Proposed INN: IBUPROFEN Other descriptive name: NA Trade Name: MYELOSTIM 34 milions UI/ml, powder and solvent for solution for injection or infusion Product Name: MYELOSTIM 34 milions UI/ml - powder and solvent for solution for injection/infusion INN or Proposed INN: LENOGRASTIM Trade Name: Mozobil 20mg/mL vial (injectable solution for subcutaneous use) Product Name: Mozobil 20mg/mL vial (injectable solution, subcutaneous use) INN or Proposed INN: Plerixafor Other descriptive name: Plerixafor Product Name: Pantoprazolo 20 mg gastro-resistant tablets INN or Proposed INN: Pantoprazole Other descriptive name: PANTOPRAZOLE | Ospedale San Raffaele | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 3 | Phase 2 | Italy | ||
| 6 | NCT02231879 (ClinicalTrials.gov) | October 14, 2014 | 3/9/2014 | Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome | A Phase III Double-Blind Randomized Crossover Study of Plerixafor Versus G-CSF in the Treatment of Patients With WHIM Syndrome. | Myelokathexis;Infections;Neutropenia;Warts;Hypogammaglobulinemia | Drug: Plerixafor;Drug: G-CSF | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | 10 Years | 75 Years | All | 20 | Phase 2/Phase 3 | United States |
| 7 | NCT01182675 (ClinicalTrials.gov) | August 2010 | 9/8/2010 | Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim | Hematopoietic Stem Cell Transplantation for Children With Severe Combined Immunodeficiency Disease Utilizing Alemtuzumab and Mobilization With Plerixafor & Filgrastim | Severe Combined Immunodeficiency | Drug: Transplant Conditioning with Mobilization Only;Drug: Transplant Conditioning with Mobilization and Alemtuzumab | University of California, San Francisco | NULL | Terminated | N/A | 3 Years | All | 7 | Phase 2 | United States |
| 8 | 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 | 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] | 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] | Orchard Therapeutics (Europe) Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 8 | Phase 1;Phase 2 | Italy |
285. ファンコニ貧血
臨床試験数 : 62 / 薬物数 : 93 - (DrugBank : 30) / 標的遺伝子数 : 30 - 標的パスウェイ数 : 144
| 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 | NCT02678533 (ClinicalTrials.gov) | February 10, 2017 | 5/2/2016 | Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor | Pilot Study Assessing the Feasibility of CD34+ Cells Mobilization and Collection After Treatment With G-CSF and Plerixafor in Patients With Fanconi Anemia for Subsequent Treatment by Gene Therapy | Fanconi Anemia | Drug: G-CSF;Drug: Plerixafor | Assistance Publique - Hôpitaux de Paris | EuroFancolen | Completed | 2 Years | 17 Years | All | 4 | Phase 1/Phase 2 | France |
| 2 | NCT03157804 (ClinicalTrials.gov) | January 7, 2016 | 26/4/2017 | Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A | Clinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A (FANCOLEN-1) | Fanconi Anemia | Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs);Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells;Other: Laboratory Biomarker Analysis;Biological: Filgrastim;Drug: Plerixafor;Procedure: Bone Marrow Aspiration | Hospital Infantil Universitario Niño Jesús, Madrid, Spain | Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT);Centro de Investigación en Red de Enfermedades Raras (CIBERER);Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz;Hospital Vall d'Hebron;Universitat Autonoma de Barcelona | Active, not recruiting | 1 Year | 21 Years | All | 9 | Phase 1/Phase 2 | Spain |
| 3 | EUCTR2014-004272-29-GB (EUCTR) | 30/03/2015 | 15/07/2015 | Phase II clinical trial to evaluate safety and efficacy of mobilisation and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia for subsequent transduction with a lentiviral vector carrying the FANCA gene and reinfusion into the patient - Fancostem-Plerixafor & Filgrastim mobilisation in Fanconi Anaemia | Phase II clinical trial to evaluate safety and efficacy of mobilisation and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia for subsequent transduction with a lentiviral vector carrying the FANCA gene and reinfusion into the patient - Fancostem-Plerixafor & Filgrastim mobilisation in Fanconi Anaemia | Fanconi Anaemia;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Mozobil Product Name: Mozobil INN or Proposed INN: Mozobil Other descriptive name: Plerixafor Trade Name: Neupogen Product Name: Neupogen INN or Proposed INN: Neupogen Other descriptive name: Filgrastim | Great Ormond Street Hospital NHS foundation Trust | NULL | Not Recruiting | Female: yes Male: yes | 20 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Spain;United Kingdom | ||
| 4 | NCT02931071 (ClinicalTrials.gov) | September 2013 | 6/9/2016 | Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1 | Clinical Phase II Trial to Evaluate Efficacy and Safety of CD34+ Cells Mobilization and Collection After Treatment With Plerixafor and Filgrastim in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene and Reinfusion in the Patient | Fanconi Anemia | Drug: filgrastim;Drug: plerixafor | Hospital Universitari Vall d'Hebron Research Institute | CIEMAT;CIBERER | Completed | 2 Years | 64 Years | All | 13 | Phase 2 | Spain |
| 5 | NCT01331018 (ClinicalTrials.gov) | February 22, 2012 | 16/3/2011 | Gene Therapy for Fanconi Anemia | Gene Transfer for Patients With Fanconi Anemia Complementation Group A (FANCA) | Fanconi Anemia | Procedure: Bone Marrow Aspiration;Biological: Filgrastim;Biological: Genetically Engineered Hematopoietic Stem Progenitor Cells;Other: Laboratory Biomarker Analysis;Procedure: Leukapheresis;Drug: Methylprednisolone;Drug: Plerixafor;Drug: Prednisone | Fred Hutchinson Cancer Center | National Heart, Lung, and Blood Institute (NHLBI);Rocket Pharma Limited | Active, not recruiting | 4 Years | N/A | All | 3 | Phase 1 | United States |
299. 嚢胞性線維症
臨床試験数 : 1,695 / 薬物数 : 1,527 - (DrugBank : 268) / 標的遺伝子数 : 111 - 標的パスウェイ数 : 174
| 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 | NCT01916577 (ClinicalTrials.gov) | August 2013 | 29/7/2013 | Autologous CD117+ Progenitor Cell Mobilization for Lung Transplantation | Autologous CD117+ Progenitor Cell Mobilization for Lung Transplantation | COPD;Cystic Fibrosis;Pulmonary Fibrosis | Drug: Plerixafor mobilization of autologous CD117 stem cells | University of Colorado, Denver | Sanofi | Completed | 18 Years | 70 Years | All | 15 | Phase 1 | United States |