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

2. 筋萎縮性側索硬化症 [臨床試験数:508,薬物数:530(DrugBank:146),標的遺伝子数:170,標的パスウェイ数:221
Searched query = "Amyotrophic lateral sclerosis", "ALS"
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.
25 / 508 trials found
No.TrialIDDate_
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PhaseCountries
1NCT04390386
(ClinicalTrials.gov)
January 1, 202113/5/2020Controlled Study of IC14 for Treatment of ALSAmyotrophic Lateral Sclerosis Trial: A Randomized, Double-Blind, Placebo-Controlled Study: IC14, a Monoclonal Antibody Against CD14Amyotrophic Lateral SclerosisBiological: IC14;Other: PlaceboImplicit BioscienceNULLWithdrawn18 Years70 YearsAll0Phase 2United States
2NCT04654689
(ClinicalTrials.gov)
January 202113/11/2020Impact of the Combined Treatment of Liposomed Polyphenols With Dutasteride on the ALS PatientsImpact of the Combined Treatment of Curcumin and Resveratrol Liposomed Polyphenols With Dutasteride on the Clinical Improvement of ALS PatientsAmyotrophic Lateral SclerosisDietary 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 methylcelluloseFundación Universidad Católica de Valencia San Vicente MártirNULLNot yet recruiting18 Years75 YearsAll100Phase 2Spain
3NCT04632225
(ClinicalTrials.gov)
December 1, 202029/10/2020Safety of Engensis in Participants With Amyotrophic Lateral SclerosisA Phase 2a, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Safety of Engensis in Participants With Amyotrophic Lateral SclerosisAmyotrophic Lateral SclerosisBiological: Engensis;Other: PlaceboHelixmith Co., Ltd.NULLNot yet recruiting20 Years80 YearsAll18Phase 2NULL
4NCT04579666
(ClinicalTrials.gov)
September 30, 202022/9/2020MERIDIAN: 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 DiseaseDrug: Pegcetacoplan (APL-2);Other: PlaceboApellis Pharmaceuticals, Inc.NULLRecruiting18 YearsN/AAll228Phase 2United States
5NCT03508453
(ClinicalTrials.gov)
August 15, 201916/4/2018IC14 for Treatment of Amyotrophic Lateral SclerosisA Phase 2, Randomised, Double-Blind, Placebo-Controlled Study of IC14 for Treatment of Patients With Rapidly Progressive Motor Neuron DiseaseAmyotrophic Lateral Sclerosis;Motor Neuron DiseaseBiological: IC14;Other: PlaceboImplicit BioscienceNULLWithdrawn18 Years75 YearsAll0Phase 2Australia
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
6NCT04055623
(ClinicalTrials.gov)
August 7, 201912/8/2019T-regulatory Cells in ALSPhase 2a Study of the Expansion and Infusion of Autologous T-Regulatory Cells in Amyotrophic Lateral SclerosisALS (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 injectionsThe Methodist Hospital SystemMassachusetts 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 ConsortiumActive, not recruiting18 YearsN/AAll12Phase 2United States
7NCT03793868
(ClinicalTrials.gov)
December 4, 201828/12/2018Perampanel Single Ascending Dose Transcranial Magnetic Stimulation Biomarker Study in Amyotrophic Lateral SclerosisPerampanel Single Ascending Dose Transcranial Magnetic Stimulation Biomarker Study in Amyotrophic Lateral SclerosisAmyotrophic Lateral SclerosisDrug: Perampanel;Other: PlaceboMayo ClinicNULLCompleted18 Years70 YearsAll22Early Phase 1United States
8NCT03280056
(ClinicalTrials.gov)
August 28, 201729/8/2017Safety and Efficacy of Repeated Administrations of NurOwn® in ALS PatientsA 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 ALSAmyotrophic Lateral Sclerosis (ALS)Biological: NurOwn® (MSC-NTF cells);Other: PlaceboBrainstorm-Cell TherapeuticsCalifornia Institute for Regenerative Medicine (CIRM)Active, not recruiting18 Years60 YearsAll261Phase 3United States
9NCT03127514
(ClinicalTrials.gov)
June 22, 201712/4/2017AMX0035 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 ALSAmyotrophic Lateral Sclerosis;Motor Neuron Disease;Neuromuscular Diseases;Neurodegenerative Diseases;Spinal Cord Diseases;TDP-43 Proteinopathies;Nervous System Diseases;Central Nervous System DiseasesDrug: AMX0035;Other: PlaceboAmylyx Pharmaceuticals Inc.ALS Finding a Cure Foundation;ALS Association;Northeast ALS Consortium;Massachusetts General Hospital Neurology Clinical Research Institute;Leandro P. Rizzuto FoundationCompleted18 Years80 YearsAll137Phase 2;Phase 3United States
10NCT02962050
(ClinicalTrials.gov)
May 31, 20174/11/2016Delineating Swallowing Impairment and Decline in ALSDelineating Physiologic Mechanisms of Swallowing Impairment and Decline in ALSAmyotrophic Lateral SclerosisProcedure: 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 ScaleUniversity of FloridaNational Institute of Neurological Disorders and Stroke (NINDS)Recruiting18 Years90 YearsAll110United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
11NCT02710110
(ClinicalTrials.gov)
April 201626/2/2016Respiratory Strength Training in Persons With Amyotrophic Lateral Sclerosis (ALS)The Impact of Respiratory Strength Training in Individuals With Amyotrophic Lateral Sclerosis (ALS)Amyotrophic Lateral SclerosisDevice: 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: CapsaicinUniversity of FloridaALS AssociationCompleted21 Years85 YearsAll50Early Phase 1United States
12NCT02710162
(ClinicalTrials.gov)
April 201629/2/2016Accurate Screening Tools for Dysphagia in Amyotrophic Lateral Sclerosis (ALS)Determination of Accurate Screening Tools for Dysphagia in Amyotrophic Lateral Sclerosis (ALS)Amyotrophic Lateral SclerosisDevice: 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 ScaleUniversity of FloridaNULLCompleted21 Years85 YearsAll21Early Phase 1United States
13NCT02709330
(ClinicalTrials.gov)
April 20165/3/2016ALS Reversals - Lunasin RegimenAn 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 controlRichard Bedlack, M.D., Ph.D.NULLCompleted18 YearsN/AAll60Phase 2United States
14NCT02623699
(ClinicalTrials.gov)
January 20, 201624/11/2015An 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 MutationAmyotrophic Lateral SclerosisDrug: BIIB067;Other: PlaceboBiogenIonis Pharmaceuticals, Inc.Recruiting18 YearsN/AAll183Phase 3United States;Australia;Belgium;Canada;Denmark;France;Germany;Italy;Japan;Korea, Republic of;United Kingdom;Sweden
15NCT02881489
(ClinicalTrials.gov)
November 201522/4/2016Autologous Bone Marrow Mesenchymal Stem Cells in the Treatment of Patients With Amyotrophic Lateral SclerosisEvaluation of Mesenchymal Stem Cell Culturing Protocols in the Treatment of Amyotrophic Lateral SclerosisAmyotrophic Lateral SclerosisOther: Biological: Cell-based therapyUniversity of Warmia and MazuryNULLEnrolling by invitation18 Years65 YearsBoth30Phase 1NULL
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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16NCT02881476
(ClinicalTrials.gov)
November 201522/4/2016Therapeutic Treatment of Amyotrophic Lateral SclerosisApplication of Wharton's Jelly-derived Mesenchymal Stem Cells in the Treatment of Amyotrophic Lateral SclerosisAmyotrophic Lateral SclerosisOther: Biological: Cell-based therapyUniversity of Warmia and MazuryNULLEnrolling by invitation18 Years65 YearsBoth30Phase 1NULL
17NCT02469896
(ClinicalTrials.gov)
November 20153/6/2015A Trial of Tocilizumab in ALS SubjectsA Phase 2 Randomized, Placebo Controlled Trial of Tocilizumab in ALS SubjectsALS;Amyotrophic Lateral Sclerosis;Lou Gehrig's Disease;Motor Neuron DiseaseDrug: Tocilizumab;Other: PlaceboBarrow Neurological InstituteALS Association;Barrow Neurological Foundation;Massachusetts General Hospital;Genentech, Inc.Completed18 Years75 YearsAll22Phase 2United States
18NCT02286011
(ClinicalTrials.gov)
November 201424/10/2014Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral SclerosisPhase 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: SalineRed de Terapia CelularSpanish 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 ArrixacaActive, not recruiting18 Years70 YearsAll20Phase 1Spain
19NCT02290886
(ClinicalTrials.gov)
July 20149/10/2014A Multicenter Phase I/II Clinical Trial to Evaluate Safety of Mesenchymal Stem Cell in Patients With Amyotrophic Sclerosis LateralA 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 SevereAmyotrophic Lateral SclerosisOther: 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 MSCAndalusian Initiative for Advanced Therapies - Fundación Pública Andaluza Progreso y SaludIniciativa Andaluza en Terapias AvanzadasActive, not recruiting18 YearsN/AAll52Phase 1;Phase 2Spain
20NCT01786174
(ClinicalTrials.gov)
August 20134/2/2013Gilenya 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 SclerosisDrug: Gilenya;Other: PlaceboMassachusetts General HospitalALS Therapy Development InstituteCompleted18 YearsN/AAll30Phase 2United States
No.TrialIDDate_
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21NCT01709149
(ClinicalTrials.gov)
October 201216/10/2012Study 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 SclerosisDrug: CK-2017357;Other: Placebo tablets;Drug: RiluzoleCytokineticsNULLCompleted18 YearsN/AAll711Phase 2United States;Canada;France;Germany;Ireland;Netherlands;Spain;United Kingdom
22NCT01363401
(ClinicalTrials.gov)
February 201130/5/2011Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral SclerosisAn Open-label, Phase I/II Trial for Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral SclerosisAmyotrophic Lateral Sclerosis;ALSBiological: HYNR-CS inj;Other: Control groupCorestem, Inc.NULLCompleted25 Years75 YearsAll72Phase 1;Phase 2Korea, Republic of
23NCT02193893
(ClinicalTrials.gov)
January 201016/7/2014Biological Treatment of Amyotrophic Lateral SclerosisSafety/Efficacy Study for the Biological Treatment of Amyotrophic Lateral Sclerosis With Autologous Stem/Progenitor CellsAmyotrophic Lateral SclerosisOther: Biological: Cell-based therapeutics;Other: Symptomatic treatment of ALSPomeranian Medical University SzczecinNULLEnrolling by invitation18 Years65 YearsBoth50Phase 1Poland
24NCT00696332
(ClinicalTrials.gov)
September 200810/6/2008Talampanel 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)ALSDrug: Talampanel;Other: placeboTeva Pharmaceutical IndustriesNULLCompleted18 Years80 YearsBoth559Phase 2United States;Belgium;Canada;France;Germany;Hungary;Israel;Italy;Netherlands;Spain
25NCT00349622
(ClinicalTrials.gov)
July 20065/7/2006Clinical Trial Ceftriaxone in Subjects With ALSClinical Trial Ceftriaxone in Subjects With Amyotrophic Lateral Sclerosis (ALS)Amyotrophic Lateral Sclerosis;ALSDrug: ceftriaxone;Other: placeboMassachusetts General HospitalNational Institute of Neurological Disorders and Stroke (NINDS)Completed18 YearsN/AAll513Phase 3United States;Canada;Puerto Rico

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 deficiency", "CD8 deficiency", "ZAP-70 deficiency", "MHC class I deficiency", "MHC class II deficiency", "Combined immunodeficiency", "Wiskott-Aldrich syndrome", "Telangiectasia ataxia", "Nijmegen breakage syndrome", "Bloom syndrome", "Immunodeficiency, centromere region instability, facial anomalies syndrome", "ICF syndrome", "PMS2 deficiency", "Radiosensitivity, immunodeficiency, dysmorphic features, and learning difficulties syndrome", "RIDDLE syndrome", "Schimke syndrome", "Netherton syndrome", "Thymic hypoplasia", "DiGeorge syndrome", "22q11.2 deletion syndrome", "Hyper-IgE syndrome", "Hepatic venoocclusive immunodeficiency", "Immunodeficiency with central hepatic vein atresia", "Dyskeratosis congenita", "X-linked agammaglobulinaemia", "Common variable immunodeficiency", "Hyper-IgM syndrome", "Isolated IgG subclass deficiency", "Selective IgA deficiency", "Specific antibody production deficiency", "Infant transient hypogammaglobulinemia", "Chédiak-Higashi syndrome", "Chediak-Higashi syndrome", "X-linked lymphoproliferative syndrome", "SAP deficiency", "SH2D1A/SLAM-associated protein deficiency", "XIAP deficiency", "X-linked inhibitor of apoptosis deficiency", "Autoimmune lymphoproliferative syndrome", "ALPS", "Familial hemophagocytic syndrome", "Perforin deficiency", "Munc13-4 deficiency", "Syntaxin 11 deficiency", "Munc18-2 deficiency", "Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy", "APECED", "Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome", "IPEX syndrome", "CD25 deficiency", "ITCH deficiency", "Primary phagocytic dysfunction", "Severe congenital neutropenia", "Cyclic neutropenia", "Hermanskyi-Pudlak syndrome type 2", "Hermanskyi-Pudlak syndrome 2", "Griscelli syndrome type 2", "Griscelli syndrome 2", "p14 deficiency", "Warts, hypogammaglobulinemia, infections, myelokathexis syndrome", "WHIM syndrome", "Glycogen storage disease type Ib", "Leukocyte adhesion deficiency", "Shwachman-Diamond syndrome", "Chronic granulomatous disease", "Myeloperoxidase deficiency", "Mendelian susceptibility to mycobacterial disease", "MSMD", "Anhidrotic ectodermal dysplasia with immunodeficiency", "EDA-ID", "Interleukin-1 receptor-associated kinase-4 deficiency", "IRAK4 deficiency", "IMyD88 deficiency", "Chronic mucocutaneous candidiasis", "Epidermodysplasia verruciformis", "Herpes simplex encephalitis", "Caspase recruitment domain family member 9 deficiency", "CARD9 deficiency", "Trypanosomiasis", "Congenital complement deficiency", "C1q deficiency", "CC1r deficiency", "CC1s deficiency", "CC2 deficiency", "CC3 deficiency", "CC4 deficiency", "CC5 deficiency", "CC6 deficiency", "CC7 deficiency", "CC8 deficiency", "CC9 deficiency", "Factor D deficiency", "Properdin deficiency", "Factor I deficiency", "Factor H deficiency", "MASP1 deficiency", "3MC syndrome", "Mannose-binding protein-associated serine protease 2 deficiency", "MASP2 deficiency", "FCN3", "Hereditary angioedema type 1", "Hereditary angioedema type I", "C1 inhibitor deficiency type 1", "C1 inhibitor deficiency type I", "Hereditary angioedema type 2", "Hereditary angioedema type II", "C1 inhibitor deficiency type 2", "C1 inhibitor deficiency type II", "Hereditary angioedema type 3", "Hereditary angioedema type III", "C1 inhibitor deficiency type 3", "C1 inhibitor deficiency type III"
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.
13 / 413 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT03597594
(ClinicalTrials.gov)
December 202019/6/2018Haplocompatible 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 ImmunodeficiencyDrug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: Thiotepa;Device: CliniMACS;Other: Donor Lymphocyte InfusionSt. Jude Children's Research HospitalNULLRecruiting2 MonthsN/AAll42Phase 1;Phase 2United States
2NCT04275479
(ClinicalTrials.gov)
January 10, 202010/2/2020Diabetes/ Endocrine Surveillance in SDSEndocrine Diabetes Screening in Patients With Shwachman-Diamond Syndrome DIABETES/ ENDOCRINE SURVEILLANCE IN SDSShwachman-Diamond SyndromeDiagnostic 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 QuestionnairesWashington University School of MedicineShwachman Diamond Syndrome Foundation;Barnes-Jewish HospitalRecruiting3 YearsN/AAll60United States
3NCT03333486
(ClinicalTrials.gov)
December 7, 20172/11/2017Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood CancerA Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem CellsAccelerated 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 SyndromeDrug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body IrradiationRoswell Park Cancer InstituteNational Cancer Institute (NCI)Recruiting1 Year75 YearsAll58Phase 2United States
4NCT03113760
(ClinicalTrials.gov)
July 21, 201728/2/2017Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP DeficiencyMulticenter, 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 DeficiencyNLRC4-MAS;XIAP DeficiencyDrug: Tadekinig alfa;Other: 0.9% sodium chlorideAB2 Bio Ltd.NULLRecruitingN/A17 YearsAll10Phase 3United States;Canada;Germany
5NCT02787486
(ClinicalTrials.gov)
October 201526/5/2016Expanded Noninvasive Genomic Medical Assessment: The Enigma StudyA 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 WomenDown Syndrome;Edwards Syndrome;Patau Syndrome;Klinefelter Syndrome;Turner Syndrome;DiGeorge Syndrome;Chromosome Deletion;AneuploidyOther: Blood sampling for Laboratory Developed Test (LDT) analysisProgenity, Inc.NULLCompleted18 Years54 YearsFemale760United States
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
6NCT01380990
(ClinicalTrials.gov)
November 15, 201223/6/2011Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) DeficiencyPhase 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 IndividualsAdenosine 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-AdaGreat Ormond Street Hospital for Children NHS Foundation TrustOrchard TherapeuticsCompletedN/A15 YearsAll36Phase 1;Phase 2United Kingdom
7NCT02327351
(ClinicalTrials.gov)
July 201218/12/2014TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PIDPhase 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 DiseasesPrimary Immune Deficiency Disorder;Hematopoietic Stem Cell TransplantationOther: Biological: TCR alfa beta T cell depletionFederal Research Institute of Pediatric Hematology, Oncology and ImmunologyNULLRecruiting1 Month18 YearsBoth60Phase 2;Phase 3Russian Federation
8NCT01529827
(ClinicalTrials.gov)
February 28, 20126/2/2012Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesA Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body IrradiationAccelerated 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 SyndromeDrug: 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 transplantationRoswell Park Cancer InstituteNULLCompleted3 Years75 YearsAll94Phase 2United States
9NCT03333200
(ClinicalTrials.gov)
January 11, 201224/4/2017Longitudinal Study of Neurodegenerative DisordersLongitudinal Study of Neurodegenerative DisordersMLD;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 DiseaseOther: Palliative Care;Biological: Hematopoetic Stem Cell TransplantationUniversity of PittsburghNULLRecruitingN/AN/AAll1500United States
10NCT00358657
(ClinicalTrials.gov)
May 24, 200628/7/2006Fludarabine 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 DisordersHLA-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 CyclophosphamideImmunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;DonorProcedure: 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 IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)Active, not recruitingN/A55 YearsAll14Phase 2United States
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11NCT00579527
(ClinicalTrials.gov)
December 19, 200520/12/2007Phase I/II Thymus Transplantation With Immunosuppression #950Phase I/II Trial of Thymus Transplantation With Immunosuppression, #950DiGeorge Anomaly;Complete DiGeorge Anomaly;Complete Atypical DiGeorge Anomaly;Complete DiGeorge Syndrome;Complete Atypical DiGeorge SyndromeBiological: 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 mofetilM. Louise MarkertNational 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)CompletedN/AN/AAll14Phase 1;Phase 2United States
12NCT00576836
(ClinicalTrials.gov)
September 2, 200417/12/2007Thymus Transplantation Dose in DiGeorge #932Dose Study of Thymus Transplantation in DiGeorge Anomaly, IND 9836, #932.1DiGeorge Anomaly;DiGeorge Syndrome;Complete DiGeorge Anomaly;Complete DiGeorge SyndromeBiological: Cultured Thymus Tissue Implantation (CTTI);Other: Cultured Thymus Tissue Implantation with Parathyroid TransplantationM. Louise MarkertNational 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 GmbHCompletedN/AN/AAll7Phase 2United States
13NCT00008450
(ClinicalTrials.gov)
August 11, 19976/1/2001Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow TransplantInduction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate MofetilAdenosine 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 ImmunodeficiencyProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclosporine;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)CompletedN/AN/AAll6Phase 1United States