Clinical Trials on Aldehyde Dehydrogenase-2 (ALDH2) Deficiency in United Kingdom

Total 93 results

Memorial Sloan Kettering Cancer Center

Completed

Talazoparib and Avelumab in Participants With Metastatic Renal Cell Carcinoma

Metastatic Renal Cell Carcinoma | Fumarate Hydratase Deficient Renal Cell Carcinoma | Succinate Dehydrogenase Deficient Renal Cell Carcinoma

United States
The University of Texas Health Science Center at...

Completed

Effect of Ketogenic Diet on Glucose Metabolism and Energy Expenditure in Type 2 Diabetes

Type 2 Diabetes | Energy Supply; Deficiency | Glucose Metabolism Disorders (Including Diabetes Mellitus)

United States
Washington University School of Medicine

Recruiting

Vitamin D and Immune Mechanisms of Hypertension in Type 2 Diabetics (VDIM)

Hypertension | Diabetes Mellitus, Type 2 | Vitamin D Deficiency

United States
University of Washington

Completed

T-IR- Study to Understand the Effects of Testosterone and Estrogen on the Body's Response to the Hormone Insulin (T-IR)

Obesity | Type 2 Diabetes Mellitus | Insulin Resistance | Metabolic Disease | Androgen Deficiency

United States
Pharvaris Netherlands B.V.

Completed

Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema (RAPIDe-1)

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditions

Bulgaria, United States, Spain, Israel, Germany, Canada, Czechia, France, Hungary, Italy, Netherlands, Poland, United Kingdom
Eunice Kennedy Shriver National Institute of Child...
Rockefeller University

Completed

Phase II Randomized Study of Tin Mesoporphyrin for Neonatal Hyperbilirubinemia

Hyperbilirubinemia | Glucosephosphate Dehydrogenase Deficiency | Hemolytic Disease of Newborn

United States
University of Texas Southwestern Medical Center
National Cancer Institute (NCI); Howard Hughes Medical Institute

Recruiting

An Investigation of Kidney and Urothelial Tumor Metabolism in Patients Undergoing Surgical Resection and/or Biopsy

Kidney Cancer | Renal Cell Carcinoma | Urothelial Carcinoma | Hereditary Leiomyomatosis and Renal Cell Cancer | Metastatic Urothelial Carcinoma | Chromophobe Renal Cell Carcinoma | Papillary Renal Cell Carcinoma | HLRCC | Clear Cell Carcinoma | Metastatic Kidney Cancer | Fumarate Hydratase Deficiency | Succinate...

United States
Genzyme, a Sanofi Company

Completed

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

Glycogen Storage Disease Type II | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe Disease

United States
Memorial Sloan Kettering Cancer Center

Active, not recruiting

A Study of Nivolumab In Combination With Cabozantinib in Patients With Non-Clear Cell Renal Cell Carcinoma

Chromophobe Renal Cell Carcinoma | Papillary Renal Cell Carcinoma | Unclassified Renal Cell Carcinoma | Advanced or Metastatic Non-clear Cell Renal Cell Carcinoma | Fumarate Hydratase Deficient Renal Cell Carcinoma | Succinate Dehydrogenase Deficient Renal Cell Carcinoma | Collecting Duct Renal...

United States
State University of New York at Buffalo

Completed

Oxygenation Changes After 2-month Exercise in Sedentary Older Adults With Diabetes

Diabetes Mellitus, Type 2 | Oxygen Deficiency

United States
Pharvaris Netherlands B.V.

Recruiting

Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema (RAPIDe-3)

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditions

United States, Puerto Rico
Genzyme, a Sanofi Company

Completed

A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

Glycogen Storage Disease Type II | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe Disease

United States
Pharvaris Netherlands B.V.

Recruiting

Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema (RAPIDe-2)

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditions

United States, Bulgaria, Czechia, Hungary, Spain, France, Germany, Poland, Canada, Israel
Passage Bio, Inc.

Active, not recruiting

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis (Imagine-1)

GM1 Gangliosidosis | GM1 Gangliosidosis, Type I | GM1 Gangliosidosis, Type 2 | Beta-Galactosidase-1 (GLB1) Deficiency

United States, Canada, United Kingdom, Turkey, Brazil
National Cancer Institute (NCI)

Terminated

A Phase II Trial of the DNA Methyl Transferase Inhibitor, Guadecitabine (SGI-110), in Children and Adults With Wild Type GIST,Pheochromocytoma and Paraganglioma Associated With Succinate Dehydrogenase Deficiency and HLRCC-associated Kidney Cancer

Carcinoma, Renal Cell | Gastrointestinal Stromal Tumors | Pheochromocytoma | Paraganglioma | Renal Neoplasms

United States
Genzyme, a Sanofi Company

Completed

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

Glycogenesis 2 Acid Maltase Deficiency | Pompe Disease (Late-Onset) | Glycogen Storage Disease Type II (GSD II)

United States, Netherlands, United Kingdom, Germany
Spark Therapeutics

Completed

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Lysosomal Storage Diseases | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase Deficiency

United States, Italy, United Kingdom, Netherlands, France, Germany
Genzyme, a Sanofi Company

Completed

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

Pompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2

United States, France, Netherlands
Children's Hospital Medical Center, Cincinnati
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

Wolman Disease | Cholesterol Ester Storage Disease | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2

United States
Genzyme, a Sanofi Company

Approved for marketing

Alglucosidase Alfa Temporary Access Program (ATAP)

Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe Disease (Late-Onset)

United States
Inozyme Pharma
Engage Health Inc.; GACI Global

Completed

Understanding the Spectrum of ENPP1 Deficiency and Acute ABCC6 Deficiency

Generalized Arterial Calcification in Infancy | Autosomal Recessive Hypophosphatemic Rickets Type 2

United States
Temple University
The Comprehensive NeuroAIDS Center (CNAC) at Temple University

Terminated

Liraglutide for HIV-associated Neurocognitive Disorder

HIV Infection | Obesity | Overweight | Metabolic Syndrome | Diabetes Mellitus Type 2

United States
Spark Therapeutics

Active, not recruiting

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Lysosomal Storage Diseases | Glycogen Storage Disease Type II | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase Deficiency

United States, Canada, Netherlands, France, Denmark, Germany, Italy, United Kingdom
State University of New York at Buffalo
National Center for Advancing Translational Sciences (NCATS)

Withdrawn

Pioglitazone and Insulin Resistance in ADT

Diabetes Mellitus, Type 2 | Insulin Resistance | Prostate Cancer | Androgen Deficiency

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
Genzyme, a Sanofi Company

Terminated

Immune Tolerance Induction Study

Glycogen Storage Disease Type II (GSD-II) | Pompe Disease | Glycogenesis 2 Acid Maltase Deficiency

United States, Israel
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
Genzyme, a Sanofi Company

Completed

High Dose or High Dose Frequency Study of Alglucosidase Alfa

Glycogen Storage Disease Type II (GSD-II) | Pompe Disease | Glycogenesis 2 Acid Maltase Deficiency

United States, Canada, Australia
Genzyme, a Sanofi Company

Terminated

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Glycogenosis 2 | Glycogen Storage Disease Type II (GSD II) | Acid Maltase Deficiency | Pompe Disease (Infantile-Onset)

United States, Taiwan, Germany
Genzyme, a Sanofi Company

Recruiting

Pompe Pregnancy Sub-Registry

Pompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Glycogenesis 2 Acid Maltase Deficiency

Croatia, United States, Italy, Belgium, Czechia
MP Biomedicals, LLC
MP Biomedicals Asia Pacific Pte. Ltd.

Completed

Dual Algorithm Post Market Clinical Study

HTLV-I Infections | HTLV-II Infections | Human T-lymphotropic Virus 1 | Human T-lymphotropic Virus 2 | HTLV I Associated T Cell Leukemia Lymphoma | HTLV I Associated Myelopathies

United States
MP Biomedicals, LLC
Vital Systems Inc.

Unknown

Evaluation of the MP Diagnostics HTLV Blot 2.4

HTLV-I Infections | HTLV-II Infections | Human T-lymphotropic Virus 1 | Human T-lymphotropic Virus 2 | HTLV I Associated T Cell Leukemia Lymphoma | HTLV I Associated Myelopathies

United States
MP Biomedicals, LLC
MP Biomedicals Asia Pacific Pte. Ltd.

Completed

MP Diagnostics HTLV Blot 2.4 Post-Market Clinical Study

HTLV-I Infections | HTLV-II Infections | Human T-lymphotropic Virus 1 | Human T-lymphotropic Virus 2 | HTLV I Associated T Cell Leukemia Lymphoma | HTLV I Associated Myelopathies

United States
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
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
White Plains Hospital

Active, not recruiting

Pancreatic Cancer Early Detection Program (PCEDP)

Pancreatic Cancer | Pancreatic Adenocarcinoma | Lynch Syndrome | Pancreas Cancer | HNPCC | Familial Pancreatic Cancer | Hereditary Pancreatitis | BRCA 1/2 | FAMMM | Familial Atypical Multiple Mole Melanoma | Peutz Jeghers Syndrome

United States
OHSU Knight Cancer Institute
National Cancer Institute (NCI); Oregon Health and Science University

Recruiting

Identifying and Caring for Individuals With Inherited Cancer Syndrome

Malignant Solid Neoplasm | Lynch Syndrome | Hematopoietic and Lymphoid System Neoplasm | Breast Ductal Carcinoma In Situ | Hereditary Neoplastic Syndrome | BRCA1/2-Associated Hereditary Breast and Ovarian Cancer Syndrome

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
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
National Cancer Institute (NCI)

Terminated

Ibrutinib in Treating Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma in Patients With HIV Infection

HIV Infection | 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 Lymphoblastic Lymphoma | Recurrent Grade 1 Follicular Lymphoma | Recurrent Grade 2 Follicular... 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
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

Clinical Trials on Aldehyde Dehydrogenase-2 (ALDH2) Deficiency in United Kingdom

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CROs in Panama

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