65. 原発性免疫不全症候群
［臨床試験数：413，薬物数：581（DrugBank：97），標的遺伝子数：68，標的パスウェイ数：202］
Searched query = "Primary immunodeficiency", "X-SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase def ... show all
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | NCT04370795 (ClinicalTrials.gov) | December 17, 2020 | 30/4/2020 | Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus | Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus | Severe Combined Immunodeficiency (SCID) | Drug: Sirolimus;Drug: Busulfan;Drug: Horse -Anti-thymocyte;Drug: G-CSF;Radiation: Total Body Irradiation (TBI) | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Enrolling by invitation | 3 Years | 40 Years | All | 30 | Phase 1;Phase 2 | United States |
| 2 | NCT04558736 (ClinicalTrials.gov) | November 2020 | 9/9/2020 | Haploidentical HCT for Severe Aplastic Anemia | Haploidentical Donor Hematopoietic Cell Transplantation for Patients With Severe Aplastic Anemia | Aplastic Anemia;Bone Marrow Failure Syndrome;Dyskeratosis Congenita | Drug: Anti-Thymocyte Globulin (Rabbit);Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Mesna;Drug: G-CSF;Radiation: Total Lymphoid Irradiation (TLI);Device: CliniMACS;Biological: HPC, A Infusion;Biological: CD45RA-depleted DLI | St. Jude Children's Research Hospital | NULL | Recruiting | N/A | 21 Years | All | 21 | Phase 2 | United States |
| 3 | 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 |
| 4 | 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 | ||
| 5 | 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 |
| 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 |
| 6 | NCT03055247 (ClinicalTrials.gov) | November 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 |
| 7 | 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 | ||
| 8 | 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 | Active, not recruiting | 10 Years | 75 Years | All | 19 | Phase 2;Phase 3 | United States |