Clinical Trials on Molybdenum Cofactor Deficiency Type A in Poland

Total 63 results

Origin Biosciences

Completed

A Natural History Study of Molybdenum Cofactor and Isolated Sulfite Oxidase Deficiencies

Molybdenum Cofactor Deficiency | Isolated Sulfite Oxidase Deficiency

Spain, Israel, United States, United Kingdom, Canada, Netherlands, Germany, Saudi Arabia, Tunisia, Japan, Italy, Malaysia, Poland, Turkey
CSL Behring

Completed

Prothrombin Complex Concentrate for Anticoagulant Reversal

Acquired Coagulation Factor Deficiency

Austria, Germany, Hungary, Israel, Lithuania, Netherlands, Poland, Switzerland
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
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
Northwell Health

Completed

Metformin Use and Vitamin B12 Deficiency

Type 2 Diabetes Mellitus | Vitamin B12 Deficiency

United States
CENTOGENE GmbH Rostock

Completed

Biomarker for Hereditary AngioEdema Disease (BioHAE)

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | C1 Esterase Inhibitor Deficiency | HAE | Angio Edema | C4 Deficiency | Hereditary Angioedema Type III

Turkey, Armenia, Georgia, India, Peru, Poland, Romania
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
Oregon Health and Science University
Alaska Department of Health and Social Services

Completed

Metabolic Consequences of CPT-1 Deficiency

Carnitine Palmitoyl Transferase Type 1A (CPT1A) Deficiency

United States
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
Eline C. B. Eskes

Recruiting

Magnetic Resonance Spectroscopy in Acid Sphingomyelinase Deficiency (MONACO)

Asmd, Visceral Type

Netherlands
Children's Memorial Health Institute, Poland
Department of Internal Medicine, Hypertension and Vascular Diseases, The...

Not yet recruiting

Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia (EMPAtia)

Glucose 6 Phosphatase Deficiency

Poland
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
Genzyme, a Sanofi Company

Completed

Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

Gaucher Disease, Type 1 | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease | Gaucher Disease, Non-Neuronopathic Form

United States, United Kingdom, Spain, Italy, Brazil, Canada, Poland
American Thrombosis and Hemostasis Network
Novo Nordisk A/S; Sanofi; Pfizer; Genentech, Inc.; CSL Behring; Hemophilia of Georgia... and other collaborators

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
CSL Behring

Completed

Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert

Hereditary Angioedema Types I and II

Bulgaria, Hungary, Poland, Romania
Medical University of Warsaw

Recruiting

Possible Differences in HCC Course Depending on DAA Treatment

Liver Cirrhosis | Carcinoma, Hepatocellular | Hepatitis C, Chronic | Drug Effect | Hepatitis C Virus Infection, Response to Therapy of | Drug Side Effect

Poland
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
KalVista Pharmaceuticals, Ltd.

Completed

A Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900 in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II

Hereditary Angioedema

United States, Austria, Czechia, Germany, Hungary, Italy, Netherlands, North Macedonia, Poland, United Kingdom
Janssen Sciences Ireland UC

Completed

A Study to Evaluate Efficacy and Safety of Darunavir/Cobicistat/Emtricitabine/Tenofovir Alafenamide (D/C/F/TAF) Fixed Dose Combination (FDC) Versus a Regimen Consisting of Darunavir/Cobicistat FDC With Emtricitabine/Tenofovir Disoproxil Fumarate FDC in Treatment-naive HIV Type 1 Infected Subjects

Immunodeficiency Virus Type 1, Human

United States, France, Poland, United Kingdom, Belgium, Germany, Spain, Canada, Russian Federation, Puerto Rico
Erasmus Medical Center

Completed

Dutch Acute HCV in HIV Study (DAHHS-2): Grazoprevir/Elbasvir for Acute HCV (DAHHS-2)

Hepatitis C | Human Immunodeficiency Virus | Acute Hepatitis C

Netherlands, Belgium
ViiV Healthcare
Pfizer

Completed

A Study Of Maraviroc In HIV Co-Infected Subjects With Hepatitis C And/Or Hepatitis B

HIV Coinfection

United States, Germany, Poland, Spain, Czechia, United Kingdom, Hungary, Puerto Rico, France
GlaxoSmithKline

Completed

Immunogenicity and Safety Study of GlaxoSmithKline Biologicals' GSK2202083A Vaccine Administered as a Booster Dose

Hepatitis B | Tetanus | Diphtheria | Acellular Pertussis | Poliomyelitis | Haemophilus Influenzae Type b | Diphtheria-Tetanus-aPertussis-Hepatitis B-Poliomyelitis-Haemophilus Influenzae Type b-Neisseria Meni

Poland
GlaxoSmithKline

Completed

Immunogenicity and Safety Study of GSK Biologicals' GSK2202083A Vaccine in Healthy Infants at 2, 3 and 4 Months of Age

Hepatitis B | Tetanus | Diphtheria | Acellular Pertussis | Poliomyelitis | Haemophilus Influenzae Type b | Diphtheria-Tetanus-aPertussis-Hepatitis B-Poliomyelitis-Haemophilus Influenzae Type b-Neisseria Meni

Poland
ViiV Healthcare
Pfizer

Withdrawn

Maraviroc Versus Etravirine In Combination With Antiretroviral Therapy In Drug Experienced HIV And Hepatitis Co-Infected Patients

Hepatitis B | Hepatitis C, Chronic | Human Immunodeficiency Virus

United States, Canada, Poland
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
BioMarin Pharmaceutical
ICON plc

Completed

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) (MARS)

Mucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVA

United States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
NiKang Therapeutics, Inc.

Recruiting

A Study of NKT2152, a HIF2α Inhibitor, in Patients With Advanced Clear Cell Renal Cell Carcinoma

Kidney Cancer | Kidney Neoplasms | Renal Cell Carcinoma | Clear Cell Renal Cell Carcinoma | Hypoxia | Metastatic Renal Cell Carcinoma | Recurrent Renal Cell Carcinoma | Renal Cancer | Advanced Renal Cell Carcinoma | Renal Neoplasms | ccRCC | Refractory Renal Cell Carcinoma | Hypoxia Inducible Factor (HIF) | HIF2α... and other conditions

United States
Bellicum Pharmaceuticals

Active, not recruiting

Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant

Lymphoma, Non-Hodgkin | Myelodysplastic Syndromes | Acute Lymphoblastic Leukemia | Anemia, Aplastic | Primary Immune Deficiency Disorder | Cytopenia | Osteopetrosis | Leukemia, Acute Myeloid (AML), Child | Hemoglobinopathy in Children

United States
Sirtex Medical
Bright Research Partners

Recruiting

Selective Internal Radiation Therapy (SIRT) Using SIR-Spheres® Y-90 Resin Microspheres on DoR & ORR in Unresectable Hepatocellular Carcinoma Patients (DOORwaY90)

Unresectable Hepatocellular Carcinoma | BCLC Stage B Hepatocellular Carcinoma | BCLC Stage C Hepatocellular Carcinoma | BCLC Stage A Hepatocellular Carcinoma

United States
Janssen-Cilag International NV

Completed

An Efficacy and Safety Study of Telaprevir in Patients Infected With Both Chronic Hepatitis C Virus (HCV-1) and Human Immunodeficiency Virus (HIV-1) (INSIGHT)

Chronic Hepatitis C

Australia, France, United Kingdom, Spain, Russian Federation, Brazil, Poland, Sweden
Gilead Sciences

Completed

Efficacy and Safety of Sofosbuvir Plus Ribavirin in Chronic Genotype 1, 2 and 3 Hepatitis C Virus (HCV) and Human Immunodeficiency Virus (HIV) Co-infected Adults

Hepatitis C | Human Immunodeficiency Virus

United States, Puerto Rico
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
Mandos LLC

Terminated

Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

Niemann-Pick Type C Disease

United States
KemPharm Denmark A/S

Completed

A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C

Niemann-Pick Disease, Type C

Spain, France, United Kingdom, Switzerland, Denmark, Poland, Italy, Germany
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
ZevraDenmark

Active, not recruiting

Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

Niemann-Pick Disease, Type C

United States, Spain, United Kingdom, Denmark, France, Germany, Italy, Poland, Switzerland
Brown University
Taiho Oncology, Inc.

Recruiting

TAS-116 Plus Palbociclib in Breast and Rb-null Cancer

Soft Tissue Sarcoma | Advanced Breast Cancer | SCLC | Bladder Cancer | Endometrial Cancer | Treatment-Refractory Solid Tumors | Retinoblastoma Deficiency

United States
CSL Behring

Completed

A Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema

Hereditary Angioedema Types I and II

United States, Spain, Germany, Australia, Canada, Czechia, Hungary, Israel, Italy, Romania, United Kingdom
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

Late-Onset Treatment Study Extension Protocol

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

United States, France, Canada, Netherlands, Australia
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
Janssen R&D Ireland

Completed

Study to Evaluate Efficacy and Safety of Darunavir/Cobicistat/Emtricitabine/Tenofovir Alafenamide (D/C/F/TAF) Regimen Versus Boosted Protease Inhibitor (bPI) Along With Emtricitabine/Tenofovir Disoproxil Fumarate (FTC/TDF) Regimen in Virologically-Suppressed, HIV-1 Infected Participants

Human Immunodeficiency Virus Type 1

United States, France, United Kingdom, Belgium, Spain, Switzerland, Sweden, Canada, Puerto Rico, Poland
Cyclo Therapeutics, Inc.

Active, not recruiting

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 (TransportNPC)

Niemann-Pick Disease, Type C1

United States, Taiwan, United Kingdom, Israel, Spain, Brazil, Italy, Australia, Turkey, Saudi Arabia, Argentina, Poland, Germany
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
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
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
Erasmus Medical Center
Leiden University Medical Center; Medical Center Haaglanden; Rijnstate Hospital; Maasstadziekenhu... and other collaborators

Recruiting

HIV Indicator Diseases in Hospital and Primary Care (#AwareHIV)

HIV Infections | Acquired Immunodeficiency Syndrome | Sexually Transmitted Diseases, Viral | Pneumonia | Psoriasis | Weight Loss | Thrombocytopenia | Leukopenia | Dermatitis, Seborrheic | Herpes Zoster | Lymphadenopathy | Uterine Cervical Dysplasia | Primary Health Care | Diarrhea Chronic | Candida Infection | Infectious... and other conditions

Netherlands
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
GlaxoSmithKline

Terminated

Eltrombopag To Reduce The Need For Platelet Transfusion In Subjects With Chronic Liver Disease And Thrombocytopenia Undergoing Elective Invasive Procedures (ELEVATE)

Liver Diseases | HIV Infection | Thrombocytopenia | Hepatitis C Virus | HBV | NASH - Nonalcoholic Steatohepatitis | Human Immunodeficiency Virus | Chronic Liver Disease | HCV | Non-alcoholic Steatohepatitis | Hepatitis B Virus

United States, Spain, Taiwan, Italy, Belgium, France, Korea, Republic of, Canada, Russian Federation, Pakistan, Poland, India, Argentina
Replimune Inc.

Recruiting

Study of RP1 Monotherapy and RP1 in Combination With Nivolumab (IGNYTE)

Cancer | NSCLC | Melanoma (Skin) | Microsatellite Instability | Cutaneous Melanoma | Mismatch Repair Deficiency | Non-melanoma Skin Cancer

United States, Spain, France, United Kingdom, Germany

Clinical Trials on Molybdenum Cofactor Deficiency Type A in Poland

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CROs in Dominican Republic

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