Search clinical trials for: severe deficiency

Total 18086 results

Shanghai Vitalgen BioPharma Co., Ltd.

Not yet recruiting

A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency

Aromatic L-amino Acid Decarboxylase Deficiency
Centre Hospitalier Universitaire de Liege
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators

Recruiting

Baby Detect : Genomic Newborn Screening

Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions

Belgium
Uniwersytecki Szpital Kliniczny w Opolu

Recruiting

Vitamin D3 Supplementation in Critically Ill Patients Undergoing CRRT (NephroD)

Vitamin D3 Deficiency

Poland
King's College London
University of Sheffield; University of Manchester; National Health Service, United... and other collaborators

Recruiting

The Experience of Screening for SCID

Severe Combined Immunodeficiency

United Kingdom
Romanian Society for Enteral and Parenteral Nutrition

Not yet recruiting

ImpACt of Very High Protein Content Enteral nUtrition Formulas on Critically Ill MUltipLe trAuma paTiEnts (ACCUMULATE)

Trauma | Gastroparesis | Muscle Loss | Metabolism | Nutritional Deficiency | Protein Malnutrition | Calorie Malnutrition Protein

Romania
Grifols Therapeutics LLC

Recruiting

A Study to Evaluate Efficacy, Safety, and Pharmacokinetics of XEMBIFY® Plus Standard Medical Treatment (SMT) Compared to Placebo Plus SMT to Prevent Infections in Participants With Hypogammaglobulinemia and Recurrent or Severe Infections Associated With B-cell Chronic Lymphocytic Leukemia

Bacterial Infections | B-cell Chronic Lymphocytic Leukemia | Hypogammaglobulinemia

United States, Bulgaria, Poland, Romania, Serbia
Institute of Hematology & Blood Diseases Hospital

Recruiting

A Study of PEG-rhG-CSF and rhG-CSF Used for Aplastic Anemia Granulocyte Deficiency

Severe Aplastic Anemia

China
University of California, Los Angeles

Recruiting

Autologous Mobilized Peripheral Blood CD34+ Hematopoietic Stem and Progenitor Cells (HSPC) Transduced With the Elongation Factor Alpha Short Promoter (EFS) - Adenosine Deaminase (ADA) Gene (EFS-ADA) Lentiviral Vector for Adenosine Deaminase Severe Combined Immune Deficiency (ADA SCID)

Adenosine Deaminase Severe Combined Immune Deficiency

United States
Peking University People's Hospital
Peking University First Hospital; Jiangsu Province Geriatric Institute

Recruiting

The Effect of Glimepiride Compared With Sitagliptin as an add-on Therapy to Metformin in Severe Insulin Deficiency Diabetes

Diabetes Mellitus, Type 2 | Severe Insulin Deficient Diabetes

China
Centre Leon Berard

Recruiting

Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation (EVAADE)

Myelodysplastic Syndromes | Severe Combined Immunodeficiency | Acute Leukemia | Haematological Malignancy | Hemoglobinopathy in Children | Bone Marrow Aplasia

France
Baylor College of Medicine
The Methodist Hospital Research Institute; Center for Cell and Gene Therapy...

Recruiting

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion (HAPLOTAB)

Myelodysplastic Syndromes | Primary Immunodeficiency Diseases | Hemoglobinopathies | Chronic Myeloid Leukemia | Cytopenia | Severe Aplastic Anemia | Bone Marrow Failure Syndrome | Acute Myeloid Leukemia in Remission | Hemophagocytic Lymphohistiocytoses | Acute Lymphoblastic Leukemia in Remission | Severe...

United States
Children's Memorial Health Institute, Poland
Siemens Healthcare Sp. z o.o.

Recruiting

Pediatric Patient Experience on a Diagnostic Path

Demyelinating Diseases | Urinary Incontinence | Brain Tumor | Headache | Visual Impairment | Growth Deficiency | Psychomotor Retardation, Mild to Severe

Poland
Pomeranian Medical University Szczecin

Not yet recruiting

Early-initiated High Flow Oxygen Therapy vs Conventional Oxygen Therapy Among Patients With ARDS in the Course of SARS-CoV2 Pneumonia.

Oxygen Deficiency | Corona Virus Infection | ARDS Due to Disease Caused by Severe Acute Respiratory Syndrome Coronavirus 2

Poland
National Institute of Allergy and Infectious Diseases...

Recruiting

Empagliflozin as a Treatment for Severe Congenital Neutropenia Due to G6PC3 Deficiency

Crohn's Disease | Glycogen Metabolism | Inflammatory Bowel Disease (IBD)

United States
Assistance Publique - Hôpitaux de Paris

Recruiting

Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE) (ARTEGENE)

Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency

France
University Hospital, Montpellier

Recruiting

Systematic Screening for Primary Immunodeficiencies in Patients Admitted for Severe Infection in Pediatric Intensive Care Unit (SPYSI)

Severe Infection | Severe Immune Deficiency

France
United States Army Research Institute of Environmental...

Recruiting

Effects of Varying Energy Deficits on Protein Turnover at Rest and Carbohydrate Oxidation During Steady-state Exercise

Stress, Physiological | Energy Supply; Deficiency | Energy Supply; Deficiency, Severe

United States
Federal Research Institute of Pediatric Hematology...

Not yet recruiting

Total Lymphoid Irradiation Pre-HSCT in Severe Congenital Neutropenia

Severe Congenital Neutropenia | GATA2 Deficiency

Russian Federation
Leiden University Medical Center
ZonMw: The Netherlands Organisation for Health Research and Development; Horizon...

Recruiting

Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID (RAG1-SCID)

Severe Combined Immunodeficiency Due to RAG1 Deficiency

Spain, Netherlands, Italy, Poland, Australia, Turkey, United Kingdom
ANRS, Emerging Infectious Diseases
European and Developing Countries Clinical Trials Partnership (EDCTP); European...

Recruiting

Determination of Adequate Tuberculosis Regimen in Patients Hospitalized With HIV-associated Severe Immune Suppression (DATURA)

Tuberculosis | HIV-1-infection | Immuno-Deficiency

Uganda, Cambodia, Cameroon, Guinea, Vietnam, Zambia
Limacorporate S.p.a
NAMSA

Recruiting

SMR Stemless Reverse vs SMR Reverse Shoulder System

Arthroplasty, Replacement

United States
Paul Szabolcs

Recruiting

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC (PRO-RIC)

Congenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias

United States
University Hospital, Montpellier

Recruiting

Systematic Screening for Primary Immunodeficiencies in Patients Hospitalized for Severe Infections in Intensive Care. (DIPREA)

Primary Immunodeficiency

France
Children's Hospital of Chongqing Medical University

Recruiting

Gene Therapy for X Linked Severe Combined Immunodeficiency

Gene Therapy

China
HEIDEGGER CP
Aalborg University

Recruiting

GLUCOSAFE 2 - A New Tool for Nutritional Management and Insulin-therapy in the Intensive Care Unit (ICU)

Hyperglycemia | Hypoglycemia | Critical Illness | Protein Deficiency | Energy Supply; Deficiency, Severe

Switzerland
Assistance Publique - Hôpitaux de Paris

Recruiting

Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients (HTLP Necker)

Pediatric Patients | Any Type of Severe Combined Immunodeficiency (SCID) | Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

France
University College, London
Medical Research Council

Recruiting

Transfer of Effector Memory T Cells (Tem) Following Allogeneic Stem Cell Transplantation (ToTem)

Lymphoma | Myelodysplastic Syndromes | Leukemia | Myeloma | Graft Vs Host Disease | Severe Aplastic Anemia | Primary Immune Deficiency

United Kingdom
Great Ormond Street Hospital for Children NHS Foundation...

Recruiting

Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency

Severe Combined Immunodeficiency, X-Linked

United Kingdom
Masonic Cancer Center, University of Minnesota

Recruiting

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

Myelodysplastic Syndromes | Fanconi Anemia | Dyskeratosis Congenita | Severe Aplastic Anemia | Bone Marrow Failure | T Cell Receptor Alpha/Beta Depletion | Telomere Biology Disorder

United States
University Of Perugia

Recruiting

Metabolic Effects of Very Low Carbohydrate Ketogenic Diet in Subjects With Severe Obesity

Obesity | Ketosis | Lysosomal Acid Lipase Deficiency | Ketogenic Dieting

Italy
University of California, San Francisco

Recruiting

Autologous Gene Therapy for Artemis-Deficient SCID

Severe Combined Immunodeficiency

United States
Jasper Therapeutics, Inc.

Recruiting

JSP191 Antibody Targeting Conditioning in SCID Patients

SCID

United States
Taiga Biotechnologies, Inc.

Not yet recruiting

Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency

Severe Combined Immunodeficiency

Israel
St. Jude Children's Research Hospital
Baylor College of Medicine; Children's Hospital of Philadelphia; Dana-Farber... and other collaborators

Recruiting

Investigation of the Genetics of Hematologic Diseases

Myelodysplastic Syndromes | Sickle Cell Disease | Fanconi Anemia | Severe Congenital Neutropenia | Bone Marrow Failure Syndromes | Blood Coagulation Disorder | Erythrocyte Disorder | Leukocyte Disorder | Hemostasis | Dyskeratosis Congenita | Diamond-Blackfan Anemia | Congenital Thrombocytopenia | Myeloproliferative...

United States
Masonic Cancer Center, University of Minnesota

Recruiting

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditions

United States
Children's Hospital Medical Center, Cincinnati
Memorial Sloan Kettering Cancer Center; Fred Hutchinson Cancer Center

Recruiting

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (RAFA)

Fanconi Anemia | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML) | Severe Marrow Failure

United States
Paul Szabolcs

Recruiting

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT (HSCT+RIC)

Congenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias | Systemic Juvenile Idiopathic Arthritis (sJIA)

United States
Washington University School of Medicine

Recruiting

Immune Disorder HSCT Protocol

Severe Combined Immunodeficiency | Chronic Granulomatous Disease | DiGeorge Syndrome | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | X-linked Lymphoproliferative Syndrome | X-linked Agammaglobulinemia | Chediak-Higashi Syndrome | IPEX | Autoimmune Lymphoproliferative Syndrome | Common Variable... and other conditions

United States
Masonic Cancer Center, University of Minnesota

Recruiting

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Common Variable Immunodeficiency | Chronic Granulomatous Disease | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | X-linked Lymphoproliferative Disease | Langerhan's Cell Histiocytosis | Chediak-Higashi Syndrome | Griscelli Syndrome | Bare Lymphocyte Syndrome | SCID | Omenn's Syndrome | Reticular... and other conditions

United States
Cure CMD

Recruiting

Congenital Muscle Disease Study of Patient and Family Reported Medical Information (CMDPROS)

Emery-Dreifuss Muscular Dystrophy | Congenital Myasthenic Syndrome | Limb-Girdle Muscular Dystrophy | Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency | Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy) and other conditions

United States
National Institute of Allergy and Infectious Diseases...

Recruiting

Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)

X-linked Severe Combined Immunodeficiency (XSCID)

United States
Ipsen

Recruiting

Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).

IGF1 Deficiency

Spain, United States, Italy, France, United Kingdom, Germany, Austria, French Guiana, Poland, Sweden, Martinique
St. Jude Children's Research Hospital

Recruiting

Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products

Mucopolysaccharidosis I | Severe Combined Immunodeficiency | Neuroblastoma | Neoplasm | Malignancy, Hematologic

United States
National Institute of Allergy and Infectious Diseases...

Recruiting

Evaluation of Patients With Immune Function Abnormalities

Chronic Granulomatous Disease (CGD) | X-Linked Severe Combined Immune Deficiency (XSCID) | Leukocyte Adhesion Deficiency 1 (LAD) | Graft Versus Host Disease (cGvHD)

United States
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Genetic Basis of Immunodeficiency

Severe Combined Immunodeficiency

United States
National Institute of Allergy and Infectious Diseases...

Enrolling by invitation

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 Immune Deficiency (SCID)

United States
National Institute of Allergy and Infectious Diseases...

Enrolling by invitation

Establishing Fibroblast-Derived Cell Lines From Skin Biopsies of Patients With Immunodeficiency or Immunodysregulation Disorders

Primary Immunodeficiency | DOCK8 | Virus Susceptibility

United States
National Institute of Allergy and Infectious Diseases...

Active, not recruiting

Natural History of Severe Allergic Inflammation and Reactions

PGM3 Deficiency | Eosinophilic and/or Atopic Dermatitis | OSMR Deficiency | Primary Localized Cutaneous Amyloidosis | Hereditary Alpha-tryptasemia

United States
Diane George

Active, not recruiting

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

Sickle Cell Disease | Hemoglobinopathies | Severe Congenital Neutropenia | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Macrophage Activation Syndrome | Bone Marrow Failure Syndrome | Amegakaryocytic Thrombocytopenia | Schwachman Diamond Syndrome | Primary Immunodeficiency Syndromes | Acquired Immunodeficiency... and other conditions

United States
David Williams

Active, not recruiting

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

Gene Therapy | Severe Combined Immunodeficiency, X Linked

United States, United Kingdom

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