Other: (DrugBank: -)
2 diseases| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 2 | 筋萎縮性側索硬化症 | 25 |
| 65 | 原発性免疫不全症候群 | 13 |
| 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 | NCT04390386 (ClinicalTrials.gov) | January 1, 2021 | 13/5/2020 | Controlled Study of IC14 for Treatment of ALS | Amyotrophic Lateral Sclerosis Trial: A Randomized, Double-Blind, Placebo-Controlled Study: IC14, a Monoclonal Antibody Against CD14 | Amyotrophic Lateral Sclerosis | Biological: IC14;Other: Placebo | Implicit Bioscience | NULL | Withdrawn | 18 Years | 70 Years | All | 0 | Phase 2 | United States |
| 2 | NCT04654689 (ClinicalTrials.gov) | January 2021 | 13/11/2020 | Impact of the Combined Treatment of Liposomed Polyphenols With Dutasteride on the ALS Patients | Impact of the Combined Treatment of Curcumin and Resveratrol Liposomed Polyphenols With Dutasteride on the Clinical Improvement of ALS Patients | Amyotrophic Lateral Sclerosis | Dietary Supplement: Liposomed polyphenols resveratrol and curcumin;Other: Placebo for liposomed resveratrol and curcumin;Dietary Supplement: Isocaloric Diet;Drug: Dutasteride 0.5 mg;Other: Placebo microcrystalline methylcellulose | Fundación Universidad Católica de Valencia San Vicente Mártir | NULL | Not yet recruiting | 18 Years | 75 Years | All | 100 | Phase 2 | Spain |
| 3 | NCT04632225 (ClinicalTrials.gov) | December 1, 2020 | 29/10/2020 | Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis | A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis | Amyotrophic Lateral Sclerosis | Biological: Engensis;Other: Placebo | Helixmith Co., Ltd. | NULL | Not yet recruiting | 20 Years | 80 Years | All | 18 | Phase 2 | NULL |
| 4 | NCT04579666 (ClinicalTrials.gov) | September 30, 2020 | 22/9/2020 | MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS) | A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Subjects With Amyotrophic Lateral Sclerosis (ALS) | Amyotrophic Lateral Sclerosis;Motor Neuron Disease | Drug: Pegcetacoplan (APL-2);Other: Placebo | Apellis Pharmaceuticals, Inc. | NULL | Recruiting | 18 Years | N/A | All | 228 | Phase 2 | United States |
| 5 | NCT03508453 (ClinicalTrials.gov) | August 15, 2019 | 16/4/2018 | IC14 for Treatment of Amyotrophic Lateral Sclerosis | A Phase 2, Randomised, Double-Blind, Placebo-Controlled Study of IC14 for Treatment of Patients With Rapidly Progressive Motor Neuron Disease | Amyotrophic Lateral Sclerosis;Motor Neuron Disease | Biological: IC14;Other: Placebo | Implicit Bioscience | NULL | Withdrawn | 18 Years | 75 Years | All | 0 | Phase 2 | Australia |
| 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 | NCT04055623 (ClinicalTrials.gov) | August 7, 2019 | 12/8/2019 | T-regulatory Cells in ALS | Phase 2a Study of the Expansion and Infusion of Autologous T-Regulatory Cells in Amyotrophic Lateral Sclerosis | ALS (Amyotrophic Lateral Sclerosis) | Biological: Monthly autologous Treg cells infusions + 3 times per week Interleukin-2 injections;Other: Monthly placebo infusions + 3 times per week placebo injections | The Methodist Hospital System | Massachusetts General Hospital;The Center for Clinical and Translational Sciences (CCTS) Clinical Research Unit at The University of Texas Health Science Center at Houston;North East Amyotrophic Lateral Sclerosis Consortium | Active, not recruiting | 18 Years | N/A | All | 12 | Phase 2 | United States |
| 7 | NCT03793868 (ClinicalTrials.gov) | December 4, 2018 | 28/12/2018 | Perampanel Single Ascending Dose Transcranial Magnetic Stimulation Biomarker Study in Amyotrophic Lateral Sclerosis | Perampanel Single Ascending Dose Transcranial Magnetic Stimulation Biomarker Study in Amyotrophic Lateral Sclerosis | Amyotrophic Lateral Sclerosis | Drug: Perampanel;Other: Placebo | Mayo Clinic | NULL | Completed | 18 Years | 70 Years | All | 22 | Early Phase 1 | United States |
| 8 | NCT03280056 (ClinicalTrials.gov) | August 28, 2017 | 29/8/2017 | Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients | A Phase 3, Randomized Double-Blind, Placebo-Controlled Multicenter Study to Evaluate Efficacy and Safety of Repeated Administration of NurOwn® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors) in Participants With ALS | Amyotrophic Lateral Sclerosis (ALS) | Biological: NurOwn® (MSC-NTF cells);Other: Placebo | Brainstorm-Cell Therapeutics | California Institute for Regenerative Medicine (CIRM) | Active, not recruiting | 18 Years | 60 Years | All | 261 | Phase 3 | United States |
| 9 | NCT03127514 (ClinicalTrials.gov) | June 22, 2017 | 12/4/2017 | AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) | Evaluation of the Safety, Tolerability, Efficacy and Activity of AMX0035, a Fixed Combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for the Treatment of ALS | Amyotrophic Lateral Sclerosis;Motor Neuron Disease;Neuromuscular Diseases;Neurodegenerative Diseases;Spinal Cord Diseases;TDP-43 Proteinopathies;Nervous System Diseases;Central Nervous System Diseases | Drug: AMX0035;Other: Placebo | Amylyx Pharmaceuticals Inc. | ALS Finding a Cure Foundation;ALS Association;Northeast ALS Consortium;Massachusetts General Hospital Neurology Clinical Research Institute;Leandro P. Rizzuto Foundation | Completed | 18 Years | 80 Years | All | 137 | Phase 2;Phase 3 | United States |
| 10 | NCT02962050 (ClinicalTrials.gov) | May 31, 2017 | 4/11/2016 | Delineating Swallowing Impairment and Decline in ALS | Delineating Physiologic Mechanisms of Swallowing Impairment and Decline in ALS | Amyotrophic Lateral Sclerosis | Procedure: Videofluoroscopic Swallowing Study (VFSS);Device: Voluntary Peak Cough Flow Testing;Device: Iowa Oral Performance Instrument;Drug: Capsaicin Challenge;Device: Pulmonary Function Testing;Other: Eating Assessment Tool 10;Other: The Center for Neurologic Study Bulbar Function Scale | University of Florida | National Institute of Neurological Disorders and Stroke (NINDS) | Recruiting | 18 Years | 90 Years | All | 110 | United States | |
| 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 |
| 11 | NCT02710110 (ClinicalTrials.gov) | April 2016 | 26/2/2016 | Respiratory Strength Training in Persons With Amyotrophic Lateral Sclerosis (ALS) | The Impact of Respiratory Strength Training in Individuals With Amyotrophic Lateral Sclerosis (ALS) | Amyotrophic Lateral Sclerosis | Device: PowerLung trainer;Device: Micro Mouth Pressure Meter;Procedure: Pulmonary Function Testing;Procedure: Videofluoroscopic swallowing study;Other: Swallowing Quality of Life Questionnaire;Device: Iowa Oral Pressure Instrument;Drug: Capsaicin | University of Florida | ALS Association | Completed | 21 Years | 85 Years | All | 50 | Early Phase 1 | United States |
| 12 | NCT02709330 (ClinicalTrials.gov) | April 2016 | 5/3/2016 | ALS Reversals - Lunasin Regimen | An Open-label, Single-center, 12-month Trial of a Lunasin Regimen for Patients With Amyotrophic Lateral Sclerosis (ALS) | ALS (Amyotrophic Lateral Sclerosis) | Drug: Lunasin Regimen;Other: Historical control | Richard Bedlack, M.D., Ph.D. | NULL | Completed | 18 Years | N/A | All | 60 | Phase 2 | United States |
| 13 | NCT02710162 (ClinicalTrials.gov) | April 2016 | 29/2/2016 | Accurate Screening Tools for Dysphagia in Amyotrophic Lateral Sclerosis (ALS) | Determination of Accurate Screening Tools for Dysphagia in Amyotrophic Lateral Sclerosis (ALS) | Amyotrophic Lateral Sclerosis | Device: Micro Mouth Pressure Meter;Device: Iowa Oral Performance Instrument;Device: Electrical Impedance Myography;Drug: Capsaicin;Procedure: Videofluoroscopic Swallowing Study;Procedure: Pulmonary Function Testing;Other: Swallowing Related Quality of Life Questionnaire;Other: Functional Oral Intake Scale;Other: Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised;Other: Eating Assessment Tool-10;Other: Communicative Effectiveness Survey;Other: The Center for Neurologic Studies Bulbar Function Scale | University of Florida | NULL | Completed | 21 Years | 85 Years | All | 21 | Early Phase 1 | United States |
| 14 | NCT02623699 (ClinicalTrials.gov) | January 20, 2016 | 24/11/2015 | An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS) | A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB067 Administered to Adult Subjects With Amyotrophic Lateral Sclerosis and Confirmed Superoxide Dismutase 1 Mutation | Amyotrophic Lateral Sclerosis | Drug: BIIB067;Other: Placebo | Biogen | Ionis Pharmaceuticals, Inc. | Recruiting | 18 Years | N/A | All | 183 | Phase 3 | United States;Australia;Belgium;Canada;Denmark;France;Germany;Italy;Japan;Korea, Republic of;United Kingdom;Sweden |
| 15 | NCT02469896 (ClinicalTrials.gov) | November 2015 | 3/6/2015 | A Trial of Tocilizumab in ALS Subjects | A Phase 2 Randomized, Placebo Controlled Trial of Tocilizumab in ALS Subjects | ALS;Amyotrophic Lateral Sclerosis;Lou Gehrig's Disease;Motor Neuron Disease | Drug: Tocilizumab;Other: Placebo | Barrow Neurological Institute | ALS Association;Barrow Neurological Foundation;Massachusetts General Hospital;Genentech, Inc. | Completed | 18 Years | 75 Years | All | 22 | Phase 2 | United States |
| 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 |
| 16 | NCT02881476 (ClinicalTrials.gov) | November 2015 | 22/4/2016 | Therapeutic Treatment of Amyotrophic Lateral Sclerosis | Application of Wharton's Jelly-derived Mesenchymal Stem Cells in the Treatment of Amyotrophic Lateral Sclerosis | Amyotrophic Lateral Sclerosis | Other: Biological: Cell-based therapy | University of Warmia and Mazury | NULL | Enrolling by invitation | 18 Years | 65 Years | Both | 30 | Phase 1 | NULL |
| 17 | NCT02881489 (ClinicalTrials.gov) | November 2015 | 22/4/2016 | Autologous Bone Marrow Mesenchymal Stem Cells in the Treatment of Patients With Amyotrophic Lateral Sclerosis | Evaluation of Mesenchymal Stem Cell Culturing Protocols in the Treatment of Amyotrophic Lateral Sclerosis | Amyotrophic Lateral Sclerosis | Other: Biological: Cell-based therapy | University of Warmia and Mazury | NULL | Enrolling by invitation | 18 Years | 65 Years | Both | 30 | Phase 1 | NULL |
| 18 | NCT02286011 (ClinicalTrials.gov) | November 2014 | 24/10/2014 | Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis | Phase I Clinical Trial on Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis. | ALS (Amyotrophic Lateral Sclerosis) | Biological: MNC (Mononuclear cells);Other: Saline | Red de Terapia Celular | Spanish National Health System;Hospital Universitario Virgen de la Arrixaca;Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia;Public Health Service, Murcia;Instituto Murciano de Investigación Biosanitaria Virgen de la Arrixaca | Active, not recruiting | 18 Years | 70 Years | All | 20 | Phase 1 | Spain |
| 19 | NCT02290886 (ClinicalTrials.gov) | July 2014 | 9/10/2014 | A Multicenter Phase I/II Clinical Trial to Evaluate Safety of Mesenchymal Stem Cell in Patients With Amyotrophic Sclerosis Lateral | A Multicenter Phase I/II Clinical Trial, Randomized, Controlled With Placebo, Triple Blind to Evaluate Safety, and Indications of Efficiency of the Intravenous Administration of the Therapy With 3 Doses of MSC in Patients With ASL Moderated to Severe | Amyotrophic Lateral Sclerosis | Other: Intravenous administration of placebo;Drug: Intravenous administration of 1 million of MSC;Drug: Intravenous administration of 2 million of MSC;Drug: Intravenous administration of 4 million of MSC | Andalusian Initiative for Advanced Therapies - Fundación Pública Andaluza Progreso y Salud | Iniciativa Andaluza en Terapias Avanzadas | Active, not recruiting | 18 Years | N/A | All | 52 | Phase 1;Phase 2 | Spain |
| 20 | NCT01786174 (ClinicalTrials.gov) | August 2013 | 4/2/2013 | Gilenya in Amyotrophic Lateral Sclerosis (ALS) | Phase IIa Double-Blind, Placebo-Controlled Study to Evaluate the Safety of Oral Fingolimod in Patients With Amyotrophic Lateral Sclerosis (ALS) | Amyotrophic Lateral Sclerosis | Drug: Gilenya;Other: Placebo | Massachusetts General Hospital | ALS Therapy Development Institute | Completed | 18 Years | N/A | All | 30 | Phase 2 | United States |
| 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 |
| 21 | NCT01709149 (ClinicalTrials.gov) | October 2012 | 16/10/2012 | Study of Safety, Tolerability & Efficacy of CK-2017357 in Amyotrophic Lateral Sclerosis (ALS) | A Phase IIb, Multi-National, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Efficacy of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS) (BENEFIT-ALS) | Amyotrophic Lateral Sclerosis | Drug: CK-2017357;Other: Placebo tablets;Drug: Riluzole | Cytokinetics | NULL | Completed | 18 Years | N/A | All | 711 | Phase 2 | United States;Canada;France;Germany;Ireland;Netherlands;Spain;United Kingdom |
| 22 | NCT01363401 (ClinicalTrials.gov) | February 2011 | 30/5/2011 | Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis | An Open-label, Phase I/II Trial for Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis | Amyotrophic Lateral Sclerosis;ALS | Biological: HYNR-CS inj;Other: Control group | Corestem, Inc. | NULL | Completed | 25 Years | 75 Years | All | 72 | Phase 1;Phase 2 | Korea, Republic of |
| 23 | NCT02193893 (ClinicalTrials.gov) | January 2010 | 16/7/2014 | Biological Treatment of Amyotrophic Lateral Sclerosis | Safety/Efficacy Study for the Biological Treatment of Amyotrophic Lateral Sclerosis With Autologous Stem/Progenitor Cells | Amyotrophic Lateral Sclerosis | Other: Biological: Cell-based therapeutics;Other: Symptomatic treatment of ALS | Pomeranian Medical University Szczecin | NULL | Enrolling by invitation | 18 Years | 65 Years | Both | 50 | Phase 1 | Poland |
| 24 | NCT00696332 (ClinicalTrials.gov) | September 2008 | 10/6/2008 | Talampanel for Amyotrophic Lateral Sclerosis (ALS) | A Multinational, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Tolerability and Safety of Talampanel in Subjects With Amyotrophic Lateral Sclerosis (ALS) | ALS | Drug: Talampanel;Other: placebo | Teva Pharmaceutical Industries | NULL | Completed | 18 Years | 80 Years | Both | 559 | Phase 2 | United States;Belgium;Canada;France;Germany;Hungary;Israel;Italy;Netherlands;Spain |
| 25 | NCT00349622 (ClinicalTrials.gov) | July 2006 | 5/7/2006 | Clinical Trial Ceftriaxone in Subjects With ALS | Clinical Trial Ceftriaxone in Subjects With Amyotrophic Lateral Sclerosis (ALS) | Amyotrophic Lateral Sclerosis;ALS | Drug: ceftriaxone;Other: placebo | Massachusetts General Hospital | National Institute of Neurological Disorders and Stroke (NINDS) | Completed | 18 Years | N/A | All | 513 | Phase 3 | United States;Canada;Puerto Rico |
| 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 | NCT03597594 (ClinicalTrials.gov) | December 2020 | 19/6/2018 | Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) | Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) | Severe Combined Immunodeficiency | Drug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: Thiotepa;Device: CliniMACS;Other: Donor Lymphocyte Infusion | St. Jude Children's Research Hospital | NULL | Recruiting | 2 Months | N/A | All | 42 | Phase 1;Phase 2 | United States |
| 2 | NCT04275479 (ClinicalTrials.gov) | January 10, 2020 | 10/2/2020 | Diabetes/ Endocrine Surveillance in SDS | Endocrine Diabetes Screening in Patients With Shwachman-Diamond Syndrome DIABETES/ ENDOCRINE SURVEILLANCE IN SDS | Shwachman-Diamond Syndrome | Diagnostic Test: Oral Glucose Tolerance Test;Other: Modified Oral Glucose Tolerance Test;Other: Modified Mixed Meal Tolerance Test;Device: Continuous Glucose Monitor;Other: Food Diary;Other: Medical History Questionnaires | Washington University School of Medicine | Shwachman Diamond Syndrome Foundation;Barnes-Jewish Hospital | Recruiting | 3 Years | N/A | All | 60 | United States | |
| 3 | NCT03333486 (ClinicalTrials.gov) | December 7, 2017 | 2/11/2017 | Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer | A Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem Cells | Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Acute Leukemia in Remission;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Acute Myeloid Leukemia With FLT3/ITD Mutation;Acute Myeloid Leukemia With Gene Mutations;Aplastic Anemia;B-Cell Non-Hodgkin Lymphoma;CD40 Ligand Deficiency;Chronic Granulomatous Disease;Chronic Leukemia in Remission;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Congenital Amegakaryocytic Thrombocytopenia;Congenital Neutropenia;Congenital Pure Red Cell Aplasia;Glanzmann Thrombasthenia;Immunodeficiency Syndrome;Myelodysplastic Syndrome;Myelofibrosis;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Plasma Cell Myeloma;Polycythemia Vera;Recurrent Non-Hodgkin Lymphoma;Refractory Non-Hodgkin Lymphoma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndrome;Severe Aplastic Anemia;Shwachman-Diamond Syndrome;Sickle Cell Disease;T-Cell Non-Hodgkin Lymphoma;Thalassemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich Syndrome | Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body Irradiation | Roswell Park Cancer Institute | National Cancer Institute (NCI) | Recruiting | 1 Year | 75 Years | All | 58 | Phase 2 | United States |
| 4 | NCT03113760 (ClinicalTrials.gov) | July 21, 2017 | 28/2/2017 | Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency | Multicenter, Double-blind, Placebo-controlled, Randomized Withdrawal Trial With Tadekinig Alfa (r-hIL-18BP) in Patients With IL-18 Driven Monogenic Autoinflammatory Conditions: NLRC4 Mutation and XIAP Deficiency | NLRC4-MAS;XIAP Deficiency | Drug: Tadekinig alfa;Other: 0.9% sodium chloride | AB2 Bio Ltd. | NULL | Recruiting | N/A | 17 Years | All | 10 | Phase 3 | United States;Canada;Germany |
| 5 | NCT02787486 (ClinicalTrials.gov) | October 2015 | 26/5/2016 | Expanded Noninvasive Genomic Medical Assessment: The Enigma Study | A Clinical Study to Evaluate the Relative Clinical Sensitivity, Specificity, and Performance of the a Laboratory Developed Test as a Screening Test for Fetal Chromosomal Aneuploidy, Infectious and Other Diseases, and RhD Genotyping in the General Population of Pregnant Women | Down Syndrome;Edwards Syndrome;Patau Syndrome;Klinefelter Syndrome;Turner Syndrome;DiGeorge Syndrome;Chromosome Deletion;Aneuploidy | Other: Blood sampling for Laboratory Developed Test (LDT) analysis | Progenity, Inc. | NULL | Completed | 18 Years | 54 Years | Female | 760 | United States | |
| 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 | NCT01380990 (ClinicalTrials.gov) | November 15, 2012 | 23/6/2011 | Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency | Phase I/II, Historical Controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1aS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient Individuals | Adenosine Deaminase Deficiency;Severe Combined Immunodeficiencies (SCID) | Genetic: Infusion of autologous EFS-ADA LV CD34+ cells;Other: Haematopoietic Stem Cell Transplantation (HSCT);Drug: Busulfan;Drug: Peg-Ada | Great Ormond Street Hospital for Children NHS Foundation Trust | Orchard Therapeutics | Completed | N/A | 15 Years | All | 36 | Phase 1;Phase 2 | United Kingdom |
| 7 | 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 |
| 8 | 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 |
| 9 | NCT03333200 (ClinicalTrials.gov) | January 11, 2012 | 24/4/2017 | Longitudinal Study of Neurodegenerative Disorders | Longitudinal Study of Neurodegenerative Disorders | MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency Disease | Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation | University of Pittsburgh | NULL | Recruiting | N/A | N/A | All | 1500 | United States | |
| 10 | 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 |
| 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 |
| 11 | NCT00579527 (ClinicalTrials.gov) | December 19, 2005 | 20/12/2007 | Phase I/II Thymus Transplantation With Immunosuppression #950 | Phase I/II Trial of Thymus Transplantation With Immunosuppression, #950 | DiGeorge Anomaly;Complete DiGeorge Anomaly;Complete Atypical DiGeorge Anomaly;Complete DiGeorge Syndrome;Complete Atypical DiGeorge Syndrome | Biological: Cultured Thymus Tissue for Implantation (CTTI);Other: Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation;Procedure: Blood Draw;Drug: Rabbit anti-thymocyte globulin;Drug: Cyclosporine;Drug: Tacrolimus;Drug: Methylprednisolone or Prednisolone;Drug: Daclizumab;Drug: Mycophenolate mofetil | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Enzyvant Therapeutics GmbH;Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Completed | N/A | N/A | All | 14 | Phase 1;Phase 2 | United States |
| 12 | NCT00576836 (ClinicalTrials.gov) | September 2, 2004 | 17/12/2007 | Thymus Transplantation Dose in DiGeorge #932 | Dose Study of Thymus Transplantation in DiGeorge Anomaly, IND 9836, #932.1 | DiGeorge Anomaly;DiGeorge Syndrome;Complete DiGeorge Anomaly;Complete DiGeorge Syndrome | Biological: Cultured Thymus Tissue Implantation (CTTI);Other: Cultured Thymus Tissue Implantation with Parathyroid Transplantation | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbH | Completed | N/A | N/A | All | 7 | Phase 2 | United States |
| 13 | 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 |