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.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-001145-14-DE
(EUCTR)
23/01/202027/11/2018Clinical 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
35Phase 3France;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.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03547830
(ClinicalTrials.gov)
April 13, 201924/5/2018Plerixafor/G-CSF as Additional Agents for Conditioning Before HSCT in CGD PatientsA Clinical Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure in Patients With Chronic Granulomatous DiseaseChronic Granulomatous DiseaseDrug: Plerixafor;Drug: GcsfFederal Research Institute of Pediatric Hematology, Oncology and ImmunologyNULLRecruiting1 Month24 YearsAll17Phase 2Russian Federation
2EUCTR2018-003842-18-IT
(EUCTR)
08/01/201919/11/2018Gene 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
6Phase 3Italy
3NCT03019809
(ClinicalTrials.gov)
June 201611/1/2017A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS PatientsA 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 FailureBiological: G-CSF for Conditioning before HSCT.;Biological: Plerixafor for Conditioning before HSCT.Federal Research Institute of Pediatric Hematology, Oncology and ImmunologyNULLUnknown status1 Month19 YearsAll30Phase 2Russian Federation
4NCT03055247
(ClinicalTrials.gov)
November 6, 201521/7/2016Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGDA 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-CGDChronic Granulomatous Disease X-linked (X-CGD)Drug: Ibuprofen;Drug: Myelostim;Drug: MozobilIRCCS San RaffaeleFondazione TelethonRecruiting18 Years45 YearsMale3Phase 2Italy
5EUCTR2015-002356-27-IT
(EUCTR)
16/10/201529/07/2015Evaluation of safety and efficacy of the combination of Ibuprofen (IBU), G-CSF and Plerixafor as a stem cell mobilization regimen in patients affected by XCGDA 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 RaffaeleNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
3Phase 2Italy
6NCT02231879
(ClinicalTrials.gov)
October 14, 20143/9/2014Plerixafor Versus G-CSF in the Treatment of People With WHIM SyndromeA Phase III Double-Blind Randomized Crossover Study of Plerixafor Versus G-CSF in the Treatment of Patients With WHIM Syndrome.Myelokathexis;Infections;Neutropenia;Warts;HypogammaglobulinemiaDrug: Plerixafor;Drug: G-CSFNational Institute of Allergy and Infectious Diseases (NIAID)NULLCompleted10 Years75 YearsAll20Phase 2/Phase 3United States
7NCT01182675
(ClinicalTrials.gov)
August 20109/8/2010Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & FilgrastimHematopoietic Stem Cell Transplantation for Children With Severe Combined Immunodeficiency Disease Utilizing Alemtuzumab and Mobilization With Plerixafor & FilgrastimSevere Combined ImmunodeficiencyDrug: Transplant Conditioning with Mobilization Only;Drug: Transplant Conditioning with Mobilization and AlemtuzumabUniversity of California, San FranciscoNULLTerminatedN/A3 YearsAll7Phase 2United States
8EUCTR2009-017346-32-IT
(EUCTR)
15/03/201026/04/2010HAEMATOPOIETIC STEM CELL GENE THERAPYA 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) LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
8Phase 1;Phase 2Italy

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.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02678533
(ClinicalTrials.gov)
February 10, 20175/2/2016Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and PlerixaforPilot 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 TherapyFanconi AnemiaDrug: G-CSF;Drug: PlerixaforAssistance Publique - Hôpitaux de ParisEuroFancolenCompleted2 Years17 YearsAll4Phase 1/Phase 2France
2NCT03157804
(ClinicalTrials.gov)
January 7, 201626/4/2017Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype AClinical 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 AnemiaProcedure: 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 AspirationHospital Infantil Universitario Niño Jesús, Madrid, SpainCentro 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 BarcelonaActive, not recruiting1 Year21 YearsAll9Phase 1/Phase 2Spain
3EUCTR2014-004272-29-GB
(EUCTR)
30/03/201515/07/2015Phase 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 AnaemiaPhase 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 TrustNULLNot 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): noFrance;Spain;United Kingdom
4NCT02931071
(ClinicalTrials.gov)
September 20136/9/2016Clinical 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-1Clinical 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 PatientFanconi AnemiaDrug: filgrastim;Drug: plerixaforHospital Universitari Vall d'Hebron Research InstituteCIEMAT;CIBERERCompleted2 Years64 YearsAll13Phase 2Spain
5NCT01331018
(ClinicalTrials.gov)
February 22, 201216/3/2011Gene Therapy for Fanconi AnemiaGene Transfer for Patients With Fanconi Anemia Complementation Group A (FANCA)Fanconi AnemiaProcedure: Bone Marrow Aspiration;Biological: Filgrastim;Biological: Genetically Engineered Hematopoietic Stem Progenitor Cells;Other: Laboratory Biomarker Analysis;Procedure: Leukapheresis;Drug: Methylprednisolone;Drug: Plerixafor;Drug: PrednisoneFred Hutchinson Cancer CenterNational Heart, Lung, and Blood Institute (NHLBI);Rocket Pharma LimitedActive, not recruiting4 YearsN/AAll3Phase 1United 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.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT01916577
(ClinicalTrials.gov)
August 201329/7/2013Autologous CD117+ Progenitor Cell Mobilization for Lung TransplantationAutologous CD117+ Progenitor Cell Mobilization for Lung TransplantationCOPD;Cystic Fibrosis;Pulmonary FibrosisDrug: Plerixafor mobilization of autologous CD117 stem cellsUniversity of Colorado, DenverSanofiCompleted18 Years70 YearsAll15Phase 1United States