Clinical Trials on Trifunctional Protein (TFP) Deficiency in South Africa

Total 11 results

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
rEVO Biologics

Completed

Assess Incidence of Deep Vein Thrombosis(DVT)Following Administration of Recombinant Human Antithrombin (rhAT) to Hereditary Antithrombin(AT) Deficient Patients in High Risk Situations. (rhAT)

Antithrombin Deficiency, Congenital

France, United Kingdom, Germany, United States, Sweden, Italy
Monash University

Recruiting

Role of Antibiotic Therapy or Immunoglobulin On iNfections in hAematoLogy: Immunoglobulin Stopping or Extension (RATIONALISE)

Hypogammaglobulinemia | Haematological Malignancy

Australia
CSL Behring

Completed

An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Factor XIII Deficiency

United States
CSL Behring

Completed

A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Factor XIII Deficiency

United States, Spain
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
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
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
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
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

Clinical Trials on Trifunctional Protein (TFP) Deficiency in South Africa

Sponsors and Collaborators

Medical Conditions

Drug Interventions

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CROs in Georgia

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Rare Diseases

Drug Interventions

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