Clinical Trials on Individuals at Risk of Zinc Deficiency

Total 198 results

University of California, San Francisco
Thrasher Research Fund

Completed

Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)

Sickle Cell Disease | Hemoglobinopathies | Fanconi Anemia | Thalassemia | Hematologic Malignancies | Immunodeficiencies | Nonmalignant Diseases | Genetic Inborn Errors of Metabolism

United States
Genentech, Inc.

Completed

Bone Mineral Density (BMD) in Adolescents With Growth Hormone Deficiency (GHD)

Dwarfism, Pituitary | Turner Syndrome
University of California, San Francisco

Completed

Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

Sickle Cell Disease | Hemoglobinopathies | Thalassemia | Hematologic Malignancies | Immunodeficiencies | Fanconi's Anemia | Nonmalignant Diseases | Genetic Inborn Errors of Metabolism

United States
Diurnal Limited
National Institutes of Health Clinical Center (CC)

Completed

Comparison of Two Forms of Hydrocortisone in Patients With Congenital Adrenal Hyperplasia

Adrenogenital Syndrome | Congenital Adrenal Hyperplasia | 21-Hydroxylase Deficiency

United States
Joanne Kurtzberg, MD
Aldagen

Completed

Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells

Anemia, Aplastic | MDS | Inborn Errors of Metabolism | Congenital Marrow Failure | Congenital Immunodeficiency Syndrome

United States
Clionsky Neuro Systems Inc.

Completed

COMPARATIVE EFFECTIVENESS OF MCI and DEMENTIA TREATMENTS IN A COMMUNITY-BASED DEMENTIA PRACTICE

Brain Injuries | Hypoxia | Parkinson's Disease | Anemia | TDP-43 Proteinopathies | Dementia | Tauopathies | Mild Cognitive Impairment | Iron Deficiency | Vitamin B 12 Deficiency | Frontotemporal Dementia | Vascular Dementia | Alzheimer's Disease | Neurodegenerative Disorders | TBI | Lewy Body Dementia | Hyperhomocysteinemia
Stanford University

Withdrawn

Pilot Study of alpha1-antitrypsin to Treat Neuromyelitis Optica Relapses (A1AT for NMO)

Neuromyelitis Optica

United States
Medialis Ltd.
Metabolic Support UK

Completed

Retrospective and Prospective Disease Progression and Quality of Life in XLH

X-linked Hypophosphatemia (XLH)

United Kingdom
Spruce Biosciences

Completed

Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia

Congenital Adrenal Hyperplasia | CAH - Congenital Adrenal Hyperplasia | CAH - 21-Hydroxylase Deficiency

United States
Massachusetts General Hospital

Recruiting

Kisspeptin Administration in the Adult

PCOS | Kallmann Syndrome | Hypogonadotropic Hypogonadism | Polycystic Ovarian Syndrome | Hyperprolactinemia | GnRH Deficiency

United States
LG Chem

Recruiting

Long-term Safety and Effectiveness of Growth Hormone With GHD, TS, CRF, SGA , ISS and PWS in Children (LGS)

Growth Hormone Deficiency | Chronic Renal Failure | Turner Syndrome | Small for Gestational Age | Idiopathic Short Stature

Korea, Republic of
Merck KGaA, Darmstadt, Germany

Completed

Predictive Markers in Growth Hormone Deficiency (GHD) and Turner Syndrome (TS) Children Treated With SAIZEN®

Growth Hormone Deficiency | Turner Syndrome

Argentina, Australia, Austria, Canada, France, Germany, Italy, Norway, Russian Federation, Singapore, Spain, Sweden, United Kingdom
LG Life Sciences

Completed

Evaluation of the Ease of Use, Preference, and Safety of EutropinPen Inj.

Growth Hormone Deficiency | Chronic Renal Failure | Turner Syndrome | Infant, Small for Gestational Age | Short Stature, Idiopathic

Korea, Republic of
National Institute of Diabetes and Digestive and...

Recruiting

Natural History of Noncirrhotic Portal Hypertension

Cystic Fibrosis | Turner Syndrome | Congenital Hepatic Fibrosis | Immunologic Deficiency Syndrome | Idiopathic Non-Cirrhotic Portal Hypertension

United States
NYU Langone Health
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Completed

Health-related Quality of Life in Rare Kidney Stone

Cystinuria | Primary Hyperoxaluria | Dent Disease | Adenine Phosphoribosyl Transferase Deficiency

United States, Iceland
University Hospital Schleswig-Holstein

Withdrawn

Gene Polymorphisms Influencing Steroid Synthesis and Action

Disorders of Sex Development | Congenital Adrenal Hyperplasia | Adrenal Insufficiency | Congenital Adrenal Hypoplasia | Mineralocorticoid Deficiency | Intersex
Fundación Española de Hematología y Hemoterapía

Recruiting

PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD" (PREDIGA-2)

Niemann-Pick Diseases | Gaucher Disease | Splenomegaly | Acid SphingoMyelinase Deficiency | ASMD

Spain
Apollo Therapeutics Ltd

Not yet recruiting

A Study to Investigate APL-4098 Alone and/or in Combination With Azacitidine in R/R AML and High-Risk MDS

Myelodysplastic Syndrome With Excess Blasts | Acute Myeloid Leukemia, in Relapse | Acute Myeloid Leukemia Refractory | Myelodysplastic Syndrome Acute Myeloid Leukemia
Genzyme, a Sanofi Company

Terminated

Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)

Niemann-Pick Disease | Acid Sphingomyelinase Deficiency

United States
Dana-Farber Cancer Institute

Recruiting

InAdvance: Surveillance, Prevention, and Interception in a Population at Risk for Cancer

Lung Cancer | Cirrhosis | Barrett Esophagus | Hematologic Malignancy | Non-Alcoholic Fatty Liver Disease | Cancer Risk | Vulvar Intraepithelial Neoplasia | Childhood Cancer Survivors | Lung; Node | Non Alcoholic Steatohepatitis | Osteochondroma | Cancer Predisposition Syndrome | Hereditary Cancer Prediction | Adult... and other conditions

United States
Massachusetts General Hospital
Agios Pharmaceuticals, Inc.

Recruiting

Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Myeloproliferative Neoplasm | Myelodysplastic/Myeloproliferative Neoplasm | Clonal Cytopenia of Undetermined Significance | Pyruvate Kinase Deficiency | Pyruvate Kinase Deficiency Anemia | Hereditary Hemolytic Anemia | Clonal Myeloid Neoplasm | Other... and other conditions

United States
Rush University Medical Center
Emory University

Enrolling by invitation

Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities

Tuberous Sclerosis | Fragile X Syndrome | Rett Syndrome | Hamartoma Syndrome, Multiple | Angelman Syndrome | Creatine Deficiency, X-linked | Telomeric 22Q13 Monosomy Syndrome | Chromosome 15Q, Partial Deletion

United States
William Gahl, M.D.
National Human Genome Research Institute (NHGRI)

Completed

Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

Pulmonary Fibrosis | Inborn Errors of Metabolism | Albinism | Oculocutaneous Albinism | Platelet Storage Pool Deficiency

United States
Haukeland University Hospital
Karolinska Institutet; University Hospitals Bristol and Weston NHS Foundation... and other collaborators

Completed

Dynamic Hormone Diagnostics in Endocrine Disease (ultradian)

Congenital Adrenal Hyperplasia | Acromegaly | Growth Hormone Deficiency | Cushing Syndrome | Primary Hyperaldosteronism | Adrenal Insufficiency

Norway, Sweden, Greece, United Kingdom
Augusta University

Completed

Contribution of Dolutegravir to Obesity and Cardiovascular Disease

Vascular Diseases | Body Weight Changes | Cardiovascular Abnormalities | HIV-1-infection | Body Fat Disorder | HIV-Associated Lipodystrophy Syndrome | Antiviral Drug Adverse Reaction | Abnormality of Adipose Tissue

United States
Maastricht University Medical Center

Enrolling by invitation

Reproductive Options in Inherited Skin Diseases (REPRO-ISD)

Tuberous Sclerosis | Basal Cell Nevus Syndrome | Cutis Laxa | Epidermolysis Bullosa | Ichthyosis | Ectodermal Dysplasia | Albinism | Xeroderma Pigmentosum | Birt-Hogg-Dube Syndrome | Palmoplantar Keratoses

Netherlands
Albert Einstein College of Medicine
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program (ScreenPlus)

Mucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditions

United States
Faculty Hospital Kralovske Vinohrady

Completed

The Epidemiology, Management and Comorbidities in Alopecia Areata in Czech Republic

Infections | Multiple Sclerosis | Autoimmune Diseases | Diabetes Mellitus, Type 1 | Rheumatoid Arthritis | Vaccine Reaction | Inflammatory Bowel Diseases | Psoriasis | Crohn Disease | Ulcerative Colitis | Down Syndrome | Atopic Dermatitis | Vitiligo | Alopecia Areata | COVID-19 Pandemic | Ankylosing Spondylitis | Lupus Erythematosus and other conditions

Czechia
Sanford Health
National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators

Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)

Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions

United States, Australia
Eunice Kennedy Shriver National Institute of Child...

Recruiting

Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls

Niemann-Pick Disease Type C1 | Juvenile Neuronal Ceroid Lipofuscinosis | Smith-Lemli-Opitz Syndrome | Creatine Transporter Deficiency

United States
National Center for Research Resources (NCRR)
University of California, Los Angeles

Completed

Stem Cell Transplantation (SCT) for Genetic Diseases

Thrombocytopenia | Inborn Errors of Metabolism | Metachromatic Leukodystrophy | Fanconi's Anemia | Thalassemia Major | Pure Red-Cell Aplasia

United States
Masonic Cancer Center, University of Minnesota

Terminated

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Sphingolipidoses | Niemann-Pick Disease, Type C | Krabbe Disease | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Hurler's Syndrome | Maroteaux-Lamy Syndrome | Wolman's Disease | Niemann-Pick Disease Type B

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
UK Kidney Association

Recruiting

National Registry of Rare Kidney Diseases (RaDaR)

Vasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditions

United Kingdom
Children's Hospital of Philadelphia
Illumina, Inc.

Active, not recruiting

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Mucopolysaccharidoses | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2 Gangliosidosis | Zellweger... and other conditions

United States
Children's Hospital of Philadelphia
Eli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaborators

Recruiting

The Myelin Disorders Biorepository Project (MDBP)

Mucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditions

United States
Momentum Data
Pfizer; University of Oxford; University of Surrey

Completed

The Epidemiology, Management, and the Associated Burden of Related Conditions in Alopecia Areata

Multiple Sclerosis | Pneumonia | Rheumatoid Arthritis | Systemic Lupus Erythematosus | Anxiety Disorders | Psoriasis | Asthma | Type 1 Diabetes | Graves Disease | Celiac Disease | Allergic Rhinitis | Urinary Tract Infections | Crohn Disease | Influenza | Ulcerative Colitis | Herpes Zoster | Herpes Simplex | Atopic Dermatitis | V... and other conditions

United Kingdom
Mayo Clinic

Completed

Spontaneous Coronary Artery Dissection (SCAD) and Autoimmunity

Multiple Sclerosis | Rheumatoid Arthritis | Systemic Lupus Erythematosus | Uveitis | Vasculitis | Psoriasis | Graves Disease | Systemic Sclerosis | Celiac Disease | Crohn Disease | Type 1 Diabetes Mellitus | Myasthenia Gravis | Ulcerative Colitis | Dermatomyositis | Polymyositis | Vitiligo | Hepatitis, Autoimmune | Polymyalgia... and other conditions

United States
Joanne Kurtzberg, MD
The Marcus Foundation

Recruiting

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (DUOC-01)

Mucopolysaccharidosis II | Brain Diseases, Metabolic, Inborn | Adrenoleukodystrophy | Batten Disease | Leukodystrophy, Metachromatic | Leukodystrophy, Globoid Cell | Alpha-Mannosidosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | Neimann Pick Disease | Sanfilippo Mucopolysaccharidoses

United States
Children's Hospital Los Angeles

Completed

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Granuloma | Anemia | Thrombocytopenia | Neutropenia | Sickle Cell Disease | Thalassemia | Niemann-Pick Disease | Wiskott-Aldrich Syndrome | Osteopetrosis | Fucosidosis | Chediak Higashi Syndrome | Hurler Disease

United States
Fairview University Medical Center

Unknown

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Mucopolysaccharidosis I | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Pulmonary Complications | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Mannosidosis | I Cell Disease

United States
Talaris Therapeutics Inc.
Duke University

Terminated

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Niemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...

United States
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia

Chronic Myelomonocytic Leukemia | Fanconi Anemia | Childhood Acute Erythroleukemia (M6) | Childhood Acute Megakaryocytic Leukemia (M7) | Childhood Acute Monoblastic Leukemia (M5a) | Childhood Acute Monocytic Leukemia (M5b) | Childhood Acute Myeloblastic Leukemia With Maturation (M2) | Childhood Acute... and other conditions

United States
St. Jude Children's Research Hospital

Recruiting

Familial Investigations of Childhood Cancer Predisposition (SJFAMILY)

Pancreatic Cancer | Hodgkin Lymphoma | Lynch Syndrome | Tuberous Sclerosis | Fanconi Anemia | AML | Non Hodgkin Lymphoma | Familial Adenomatous Polyposis | Acute Leukemia | Nevoid Basal Cell Carcinoma Syndrome | Neurofibromatosis Type 1 | Neuroblastoma | Retinoblastoma | MDS | Rhabdomyosarcoma | Von Hippel-Lindau Disease | Adrenocortical Carcinoma and other conditions

United States
Sanguine Biosciences

Completed

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

Depression | Melanoma | Epilepsy | Lymphoma | Multiple Sclerosis | Kidney Cancer | Cervical Cancer | Diabetes Mellitus | Leukemia | Breast Cancer | Chronic Kidney Diseases | Chronic Obstructive Pulmonary Disease | Parkinson's Disease | Systemic Lupus Erythematosus | Multiple Myeloma | Hepatitis B | Insomnia | Colorectal Cancer | HIV/AID... and other conditions

United States
National Cancer Institute (NCI)

Completed

Sunitinib Malate in Treating HIV-Positive Patients With Cancer Receiving Antiretroviral Therapy

HIV Infection | Clear Cell Renal Cell Carcinoma | Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell... and other conditions

United States
Roswell Park Cancer Institute

Completed

Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Primary Myelofibrosis | Polycythemia Vera | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma and other conditions

United States
Roswell Park Cancer Institute
National Cancer Institute (NCI)

Completed

Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders

Primary Myelofibrosis | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent... and other conditions

United States

Clinical Trials on Individuals at Risk of Zinc Deficiency

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