65. Primary immunodeficiency Clinical trials / Disease details
Clinical trials : 482 / Drugs : 653 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 212
| 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 | NCT04400045 (ClinicalTrials.gov) | May 22, 2020 | 19/5/2020 | Low Dose Treosulfan Based Conditioning Regimen in HSCT for Nijmegen Breakage Syndrome | Clinical Open-label Phase 2 Study of Low Dose Treosulfan Based Conditioning Regimen Efficacy in Hematopoietic Stem Cell Transplantation for Children With Nijmegen Breakage Syndrome | Nijmegen Breakage Syndrome | Drug: Treosulfan | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Recruiting | 3 Months | 21 Years | All | 10 | Phase 2 | Russian Federation |
| 2 | NCT03879876 (ClinicalTrials.gov) | May 13, 2020 | 15/3/2019 | Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients | A Phase I/II Study Evaluating the Safety and the Efficacy of Human T Lymphoid Progenitor (HTLP) Injection to Accelerate Immune Reconstitution After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients | Pediatric Patients;Any Type of Severe Combined Immunodeficiency (SCID);Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) | Drug: Human T Lymphoid Progenitor (HTLP) injection | Assistance Publique - Hôpitaux de Paris | NULL | Recruiting | 2 Years | 18 Years | All | 12 | Phase 1/Phase 2 | France |
| 3 | EUCTR2018-001029-14-FR (EUCTR) | 25/10/2018 | 02/07/2018 | HTLP | A phase I/II Study evaluating the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients - HTLP NECKER | SCID pediatric patients (n=12 for analysis) requiring an HLA partially compatible allogeneic HSCT. MedDRA version: 20.0;Level: PT;Classification code 10010099;Term: Combined immunodeficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: HTLP Product Code: HTLP | ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP) | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 1;Phase 2 | France | ||
| 4 | NCT01821781 (ClinicalTrials.gov) | March 2013 | 19/3/2013 | Immune Disorder HSCT Protocol | A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime | Immune Deficiency Disorders;Severe Combined Immunodeficiency;Chronic Granulomatous Disease;X-linked Agammaglobulinemia;Wiskott-Aldrich Syndrome;Hyper-IgM;DiGeorge Syndrome;Chediak-Higashi Syndrome;Common Variable Immune Deficiency;Immune Dysregulatory Disorders;Hemophagocytic Lymphohistiocytosis;IPEX;Autoimmune Lymphoproliferative Syndrome;X-linked Lymphoproliferative Syndrome | Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan | Washington University School of Medicine | NULL | Recruiting | N/A | 21 Years | All | 20 | Phase 2 | United States |
| 5 | NCT02327351 (ClinicalTrials.gov) | July 2012 | 18/12/2014 | TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PID | Phase II/III Study of Allogeneic Hematopoietic Stem Cell Transplantation From Unrelated and Haploidentical Donors After TCR Alfa Beta Negative Selection in Pediatric Patients With Primary Immunodeficiency Diseases | Primary Immune Deficiency Disorder;Hematopoietic Stem Cell Transplantation | Other: Biological: TCR alfa beta T cell depletion | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Recruiting | 1 Month | 18 Years | Both | 60 | Phase 2/Phase 3 | Russian Federation |
| 6 | NCT01529827 (ClinicalTrials.gov) | February 28, 2012 | 6/2/2012 | Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies | A Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body Irradiation | Accelerated Phase Chronic Myelogenous Leukemia;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Burkitt Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;Congenital Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;Previously Treated Myelodysplastic Syndromes;Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Secondary Myelofibrosis;Severe Combined Immunodeficiency;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich Syndrome | Drug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Drug: methotrexate;Other: laboratory biomarker analysis;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation | Roswell Park Cancer Institute | NULL | Completed | 3 Years | 75 Years | All | 94 | Phase 2 | United States |
| 7 | NCT01338675 (ClinicalTrials.gov) | January 2011 | 4/3/2011 | Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD) | Chronic Granulomatous Disease | Drug: Busulfan | Seoul National University Hospital | NULL | Recruiting | N/A | N/A | Both | 5 | Phase 1/Phase 2 | Korea, Republic of | |
| 8 | NCT01319851 (ClinicalTrials.gov) | September 2010 | 15/9/2010 | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot Study | Thalassemia;Sickle Cell Disease;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic-granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Schwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Fanconi Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia | Drug: Alefacept | Emory University | Children's Healthcare of Atlanta | Terminated | N/A | 21 Years | All | 3 | N/A | United States |
| 9 | 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 |
| 10 | NCT02512679 (ClinicalTrials.gov) | February 2007 | 21/5/2015 | Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells | Protocol for Related Donor Hematopoietic Stem Cell Transplantation (HSCT) for Treatment of Symptomatic Genetic Lymphohematological Diseases | Stem Cell Transplantation;Bone Marrow Transplantation;Peripheral Blood Stem Cell Transplantation;Allogeneic Transplantation;Genetic Diseases;Thalassemia;Pediatrics;Diamond-Blackfan Anemia;Combined Immune Deficiency;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;X-linked Lymphoproliferative Disease;Metabolic Diseases | Drug: Cyclophosphamide Dose Level 1;Drug: Cyclophosphamide Dose Level 2;Drug: Cyclophosphamide Dose Level 3;Drug: Cyclophosphamide Dose Level 4 | Children's Hospital Los Angeles | Lucile Packard Children's Hospital | Terminated | 3 Months | N/A | All | 20 | Phase 2 | NULL |
| 11 | NCT00358657 (ClinicalTrials.gov) | May 24, 2006 | 28/7/2006 | Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders | HLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting Cyclophosphamide | Immunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;Donor | Procedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Sirolimus;Drug: Tacrolimus;Radiation: Total-Body Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Active, not recruiting | N/A | 55 Years | All | 14 | Phase 2 | United States |
| 12 | NCT00160355 (ClinicalTrials.gov) | May 2005 | 8/9/2005 | Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome | Haploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot Study | Wiskott-Aldrich Syndrome | Procedure: Hematopoietic stem cell transplantation;Device: Miltenyi CliniMACS selection system;Drug: Fludarabine, Melphalan, Thiotepa | St. Jude Children's Research Hospital | NULL | Completed | N/A | 18 Years | Male | 4 | Phase 1 | United States |
| 13 | NCT00295971 (ClinicalTrials.gov) | April 2005 | 23/2/2006 | Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease | Stem Cell Enriched, T Cell Depleted Haplocompatible Peripheral Blood Transplantation for Children With Myelodysplastic Disease, Leukemia, Marrow Failure Syndromes, or Severe Immunodeficiency Diseases | Congenital Amegakaryocytic Thrombocytopenia;Leukemia;Myelodysplastic Syndromes;Severe Congenital Neutropenia | Biological: anti-thymocyte globulin;Biological: therapeutic allogeneic lymphocytes;Drug: fludarabine phosphate;Drug: thiotepa;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: in vitro-treated peripheral blood stem cell transplantation;Radiation: total-body irradiation | University of California, San Francisco | National Cancer Institute (NCI) | Completed | 1 Year | 17 Years | Both | 21 | Phase 1 | United States |
| 14 | NCT00301834 (ClinicalTrials.gov) | January 2005 | 9/3/2006 | Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders | Evaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/Leukemia | Congenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Leukemia;Myelodysplastic Syndromes;Severe Congenital Neutropenia | Biological: alemtuzumab;Drug: busulfan;Drug: cyclosporine;Drug: fludarabine phosphate;Drug: methotrexate;Drug: methylprednisolone;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation | University of California, San Francisco | National Cancer Institute (NCI) | Completed | N/A | 21 Years | All | 35 | Phase 2 | United States |
| 15 | NCT00006056 (ClinicalTrials.gov) | March 2000 | 5/7/2000 | Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders | Chediak-Higashi Syndrome;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;Virus-Associated Hemophagocytic Syndrome | Drug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: filgrastim;Drug: methotrexate;Procedure: allogeneic hematopoietic stem cell transplantation | Fairview University Medical Center | NULL | Active, not recruiting | N/A | 55 Years | Both | 40 | N/A | United States | |
| 16 | NCT00008450 (ClinicalTrials.gov) | August 11, 1997 | 6/1/2001 | Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant | Induction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil | Adenosine Deaminase Deficiency;Autosomal Recessive Disorder;Immune System Disorder;Purine-Nucleoside Phosphorylase Deficiency;Severe Combined Immunodeficiency;Severe Combined Immunodeficiency With Absence of T and B Cells;X-Linked Severe Combined Immunodeficiency | Procedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclosporine;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Radiation: Total-Body Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Completed | N/A | N/A | All | 6 | Phase 1 | United States |