Clinical Trials on Sterol Carrier Protein 2 Deficiency in Poland

Total 42 results

Oregon Health and Science University

Completed

Oral Ketones and Exercise Among Patients With Long-chain Fatty Acid Oxidation Disorders

Trifunctional Protein Deficiency | Very Long Chain Acyl Coa Dehydrogenase Deficiency | Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency | Carnitine Palmitoyltransferase Deficiency 2

United States
Oregon Health and Science University
University of Pittsburgh

Completed

Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder (Triheptanoin)

Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | Carnitine Palmitoyltransferase 2 (CPT2) Deficiency | Mitochondrial Trifunctional Protein (TFP) Deficiency | Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency

United States
Oregon Health and Science University
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Oregon...

Completed

Fatty Acid Oxidation Disorders & Body Weight Regulation Grant

Trifunctional Protein Deficiency

United States
Oregon Health and Science University
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Oregon...

Terminated

Vitamin E Treatment for Long-Chain 3-Hydroxyacyl Coenzyme A (CoA) Dehydrogenase (LCHAD) Associated Neuropathy

Peripheral Neuropathy | Mitochondrial Trifunctional Protein Deficiency

United States
VU University of Amsterdam
Amsterdam UMC, location VUmc; University of Helsinki

Completed

The (Cost)Effectiveness of Increasing Protein Intake on Physical Funtioning in Older Adults

Physical Disability | Protein-Energy Malnutrition

Finland, Netherlands
Maasstad Hospital

Recruiting

Effect of Protein Supplementation on Fat Free Mass Preservation After Bariatric Surgery (PROMISE)

Bariatric Surgery Candidate | Muscle Loss | Protein Deficiency

Netherlands
Oregon Health and Science University

Completed

Fatty Acid Oxidation Defects and Insulin Sensitivity

Normal Volunteers | Trifunctional Protein Deficiency | Very Long-chain Acyl-CoA Dehydrogenase Deficiency | Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency | Medium-chain Acyl-CoA Dehydrogenase Deficiency | Carnitine Palmitoyltransferase II Deficiency, Myopathic

United States
Northwell Health

Completed

Metformin Use and Vitamin B12 Deficiency

Type 2 Diabetes Mellitus | Vitamin B12 Deficiency

United States
Glycomine, Inc.

Active, not recruiting

Natural History Study Protocol in PMM2-CDG (CDG-Ia)

Phosphomannomutase 2 Deficiency

United States, Spain, Portugal, Belgium, Czechia, France, Italy, Netherlands, 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
Baxalta now part of Shire

Completed

Retrospective Study to Capture Dosing and Treatment Outcome Data in Participants With Severe Congenital Protein C Deficiency Who Were Treated With Protein C Concentrate Under an Emergency Use IND

Protein C Deficiency

United States
Brown University
AstraZeneca

Recruiting

Adjuvant Trastuzumab Deruxtecan (Enhertu) & Nivolumab For Patients Who Are Disease Free After Completion of Trimodality Treatment For HER-2+ Cancers of Esophagus & Gastroesophageal Junction

Esophageal Cancer | Esophageal Adenocarcinoma | HER-2 Protein Overexpression | Gastroesophageal-junction Cancer

United States
Hoffmann-La Roche

Active, not recruiting

A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors (HAVEN 6)

Hemophilia A | Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor | Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor

United States, Netherlands, Belgium, France, Germany, South Africa, Spain, Canada, United Kingdom, Poland
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
Alexion

Recruiting

Lysosomal Acid Lipase (LAL) Deficiency Registry (ALX-LALD-501)

Wolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-Deficiency

France, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia
CSL Behring

Completed

Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy

Common Variable Immunodeficiency | X-linked Agammaglobulinemia | Autosomal Recessive Agammaglobulinemia

Germany, United Kingdom, France, Italy, Poland, Romania, Spain, Sweden, Switzerland
Pharvaris Netherlands B.V.

Active, not recruiting

Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II (HAE CHAPTER-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

United States, Poland, Germany, Austria, Bulgaria, Canada, Ireland, Italy, United Kingdom
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
Spark Therapeutics

Completed

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

Hematologic Diseases | Blood Coagulation Disorders, Inherited | Coagulation Protein Disorders | Hemorrhagic Disorders | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Gene Therapy | Blood Coagulation Disorder | Gene Transfer | Adeno-Associated Virus (AAV) | Factor VIII (FVIII) | Factor VIII (FVIII)... and other conditions

United States
Takeda

Recruiting

Real-world Usage of HyQvia in Multiple Myeloma Adults With Secondary Immunodeficiency

Multiple Myeloma | Secondary Immunodeficiency (SID)

Greece, Spain, Italy, Romania, Argentina, Czechia, France, Germany, Poland, Sweden, Turkey
Michigan Technological University

Withdrawn

B7 Coreceptor Molecules in Hyper IgD Syndrome Form of Mevalonate Kinase Deficiency (HIDS-MKD)

Hyper IgD Syndrome | Mevalonate Kinase Deficiency

United States, Netherlands
Oregon Health and Science University

Terminated

Dietary Cholesterol and Defects in Cholesterol Synthesis in Mevalonate Kinase Deficiency

Immune System Diseases | Lipid Metabolism, Inborn Errors | Mevalonic Aciduria | Mevalonate Kinase Deficiency | Periodic Fever Syndromes, Hereditary

United States
dr. Laura C. G. de Graaff-Herder

Not yet recruiting

Growing up With the Young Endocrine Support System (YESS!) (YESS)

Congenital Adrenal Hyperplasia | Hypogonadotropic Hypogonadism | Growth Hormone Deficiency | Turner Syndrome | Addison's Disease | Klinefelter Syndrome | Combined Pituitary Hormone Deficiency | Androgen Insensitivity Syndrome | Thyroid Dysgenesis

Spain, United Kingdom, Belgium, Netherlands
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
Octapharma

Recruiting

Double-blind, Randomized, Placebo-controlled, Prospective Phase III Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients With Chronic Lymphocytic Leukemia ("PRO-SID" Study)

Chronic Lymphocytic Leukemia | Hypogammaglobulinemia

United States, Denmark, Germany, Hungary, Italy, Poland, Russian Federation, Spain, Czechia, Greece, Israel, Turkey
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
OHSU Knight Cancer Institute
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Research Study in Healthy Volunteers of Patients With Fanconi Anemia, Myeloproliferative Disorders, or Myeloma

Chronic Myeloproliferative Disorders | Fanconi Anemia | Multiple Myeloma and Plasma Cell Neoplasm | Myelodysplastic/Myeloproliferative Neoplasms

United States
Alexey Danilov, MD
Gilead Sciences; Oregon Health and Science University

Completed

Entospletinib and Obinutuzumab in Treating Patients With Relapsed Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, or Non-Hodgkin Lymphoma

Anemia | Mantle Cell Lymphoma | Marginal Zone Lymphoma | Lymphoplasmacytic Lymphoma | Recurrent Small Lymphocytic Lymphoma | Refractory Chronic Lymphocytic Leukemia | Hairy Cell Leukemia | Richter Syndrome | Recurrent Chronic Lymphocytic Leukemia | Refractory Small Lymphocytic Lymphoma | Grade 1 Follicular... and other conditions

United States
QuantumLeap Healthcare Collaborative

Recruiting

PRE-I-SPY Phase I/Ib Oncology Platform Program (PRE-I-SPY-PI)

Solid Tumor | Metastatic Cancer | Metastatic Breast Cancer | Triple Negative Breast Cancer | HER2-positive Breast Cancer | Solid Tumor, Adult | Solid Carcinoma | HER2-positive Metastatic Breast Cancer | Progesterone Receptor-positive Breast Cancer | HER2-negative Breast Cancer | Estrogen Receptor Positive... and other conditions

United States
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

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
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
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
American Thrombosis and Hemostasis Network
Sanofi; Pfizer; Genentech, Inc.; CSL Behring; Hemophilia of Georgia, Inc.; Hemab Therapeutics

Recruiting

ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

Hemophilia | Thrombosis | Thrombophilia | Sickle Cell Disease | Thalassemia | Bleeding Disorder | Von Willebrand Diseases | Factor IX Deficiency | Connective Tissue Disorder | Factor VIII Deficiency | Hematologic Disorder | Rare Bleeding Disorder | Platelet Disorder

United States
Carisma Therapeutics Inc

Active, not recruiting

CAR-macrophages for the Treatment of HER2 Overexpressing Solid Tumors

Adenocarcinoma | Stomach Neoplasms | Cancer | Breast Cancer | Head and Neck Cancer | Carcinoma, Hepatocellular | Colorectal Cancer | Pancreatic Cancer | Ovarian Neoplasms | Carcinoma, Ovarian Epithelial | Carcinoma, Transitional Cell | Breast Neoplasm | Prostate Cancer | Bile Duct Cancer | Bladder Cancer | Endometrial Cancer and other conditions

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute...

Completed

Total-Body Irradiation and Fludarabine Phosphate Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies or Kidney Cancer

Clear Cell Renal Cell Carcinoma | Chronic Myelomonocytic Leukemia | Previously Treated Myelodysplastic Syndrome | Recurrent Adult Acute Myeloid Leukemia | Recurrent Adult Lymphoblastic Lymphoma | Untreated Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia in Remission | Childhood Acute... and other conditions

United States, Germany, Italy
Daniel Benjamin
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Completed

Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care (PTN_POPS)

Heart Failure | Epilepsy | Pain | Pneumonia | Sepsis | Inflammation | Hypertension | Chronic Kidney Diseases | Hemophilia | Schizophrenia | Central Nervous System Infections | Nosocomial Pneumonia | Insomnia | Anxiety | Bipolar Disorder | Neutropenia | Staphylococcal Infections | Bradycardia | Seizures | Urinary Tract Infections | Pulmonary... and other conditions

United States, Canada, United Kingdom, Israel, Singapore
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute...

Completed

Mycophenolate Mofetil and Cyclosporine in Reducing Graft-Versus-Host Disease in Patients With Hematologic Malignancies or Metastatic Kidney Cancer Undergoing Donor Stem Cell Transplant

Clear Cell Renal Cell Carcinoma | 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, Germany, Italy
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute...

Completed

Fludarabine Phosphate and Total-Body Radiation Followed by Donor Peripheral Blood Stem Cell Transplant and Immunosuppression in Treating Patients With Hematologic Malignancies

Stage II Multiple Myeloma | Stage III Multiple Myeloma | 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... and other conditions

United States, Germany, Italy
Morphotek
National Cancer Institute (NCI); Children's Oncology Group

Completed

MORAb-004 in Treating Young Patients With Recurrent or Refractory Solid Tumors or Lymphoma

Unspecified Childhood Solid Tumor, Protocol Specific | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent Adult Diffuse Large Cell Lymphoma | Recurrent Adult Diffuse Mixed Cell Lymphoma and other conditions

United States
University of Nebraska

Recruiting

Integrated Cancer Repository for Cancer Research (iCaRe2)

Melanoma | Sarcoma | Kidney Cancer | Leukemia | Breast Cancer | Laryngeal Cancer | Hypopharyngeal Cancer | Nasopharyngeal Cancer | Multiple Myeloma | Gastric Cancer | Pancreatic Cancer | Esophageal Cancer | Small Intestine Cancer | Ovarian Cancer | Lynch Syndrome | Lung Cancer | Rectal Cancer | Prostate Cancer | Gastrointestinal... and other conditions

United States
National Cancer Institute (NCI)

Completed

MK2206 in Treating Younger Patients With Recurrent or Refractory Solid Tumors or Leukemia

Unspecified Childhood Solid Tumor, Protocol Specific | Chronic Myelomonocytic Leukemia | Juvenile Myelomonocytic Leukemia | Recurrent Grade 1 Follicular Lymphoma | Recurrent Grade 2 Follicular Lymphoma | Recurrent Grade 3 Follicular Lymphoma | Recurrent Mantle Cell Lymphoma | Recurrent Marginal Zone... and other conditions

United States, Canada

Clinical Trials on Sterol Carrier Protein 2 Deficiency in Poland

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Bangladesh

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

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