Clinical Trials on Urea Cycle Disorders, Inborn in Argentina

Total 110 results

Shire
Takeda Development Center Americas, Inc.

Active, not recruiting

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy (EMBOLDEN)

Metachromatic Leukodystrophy (MLD)

United States, Canada, Belgium, Israel, Netherlands, United Kingdom, Germany, Spain, Japan, Brazil, Argentina, France, Greece, Italy
Octapharma

Completed

Surveillance of Safety and Efficacy of Wilate in Patients With Von Willebrand Disease

Von Willebrand Disease

United States, Canada, Sweden, Spain, Uruguay, United Kingdom, Portugal, Argentina, Czechia, Germany
Octapharma

Completed

Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ

Hemophilia A

Norway, United States, France, United Kingdom, Italy, Czechia, Argentina, Belarus, Ecuador, Guatemala, Lithuania, Portugal, Slovakia
Crinetics Pharmaceuticals Inc.

Recruiting

Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia (TouCAHn)

Congenital Adrenal Hyperplasia | Classic Congenital Adrenal Hyperplasia

Brazil, India, United States, Argentina, Italy, United Kingdom
Ionis Pharmaceuticals, Inc.

Completed

Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

Amyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | Transthyretin

United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
Denali Therapeutics Inc.

Recruiting

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) (COMPASS)

Mucopolysaccharidosis II

United States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Brazil, Colombia
Pfizer

Completed

Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII

Hemophilia A

United States, Argentina, Austria, Colombia, Croatia, Jordan, Mexico, New Zealand, Oman, Peru, Poland, Romania, Turkey
Ionis Pharmaceuticals, Inc.

Completed

Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

Amyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | Transthyretin

New Zealand, United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
Kastle Therapeutics, LLC

Completed

A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol (FOCUS FH)

Hypercholesterolemia | Heterozygous Familial

United States, Italy, Poland, Ukraine, United Kingdom, Germany, Hong Kong, Taiwan, Spain, Argentina, Belgium, Israel, Malaysia, South Africa, Turkey, Netherlands, Korea, Republic of, Canada, New Zealand, Russian Federation, Brazil, Austral... and more
Bayer

Active, not recruiting

A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood (Alfa-PROTECT)

Hemophilia A | Children | Prophylaxis of Bleeding | Treatment of Bleeding

Canada, United States, Brazil, Italy, Turkey, Argentina, Norway
Merck Sharp & Dohme LLC

Active, not recruiting

A Study of MK-0616 (Oral PCSK9 Inhibitor) in Adults With Hypercholesterolemia (MK-0616-013) CORALreef Lipids

Hypercholesterolemia | Familial Hypercholesterolemia

Colombia, Israel, Taiwan, United States, Argentina, China, Germany, Italy, Japan, Korea, Republic of, Mexico, South Africa, Spain, Turkey
Novo Nordisk A/S

Completed

Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B (paradigm™6)

Congenital Bleeding Disorder | Haemophilia B

Spain, United Kingdom, Australia, Taiwan, France, Malaysia, United States, Austria, Canada, Israel, Japan, Thailand, Algeria, Argentina
Baxalta now part of Shire

Completed

BAX 326 (rFIX) Continuation Study

Hemophilia B

Taiwan, Colombia, Argentina, Brazil, Bulgaria, Chile, Czechia, India, Ireland, Italy, Japan, Poland, Romania, Russian Federation, Sweden, Ukraine, United Kingdom
Novo Nordisk A/S

Completed

Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A (pathfinder™6)

Congenital Bleeding Disorder | Haemophilia A

United States, Spain, Australia, France, Thailand, Italy, Malaysia, Austria, Greece, Japan, Taiwan, Canada, Israel, Algeria, Argentina, Bulgaria, Germany, Romania
Hoffmann-La Roche

Completed

A Screening Protocol to Assess Adults and Adolescents With Down Syndrome for Eligibility For Upcoming Study of RG1662 (Study BP27832)

Down Syndrome

United States, Spain, Canada, New Zealand, Argentina, Mexico, Singapore
Sanofi

Recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease (PERIDOT)

Fabry Disease

China, Finland, Germany, Norway, United Kingdom, United States, Denmark, Austria, Greece, Romania, Argentina, Brazil, Canada, Japan, Mexico, Poland, Italy, France, Turkey, Australia, Switzerland
Novo Nordisk A/S

Completed

Haemophilia Patients With Inhibitors Being Treated for Acute Joint Bleeds

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

United States, Spain, Taiwan, Turkey, Poland, Croatia, Italy, Malaysia, Brazil, United Kingdom, Hungary, Israel, Thailand, Japan, South Africa, Canada, France, Argentina
Novo Nordisk A/S

Completed

Efficacy of NovoSeven® in Bleeding Prophylaxis in Hemophilia

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

France, United States, Spain, Brazil, Philippines, South Africa, Poland, Turkey, Romania, Bulgaria, Russian Federation, Argentina
Fondazione Angelo Bianchi Bonomi
Sintesi Research Srl

Completed

Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET)

Hemophilia A

United States, India, Spain, Argentina, Austria, Brazil, Chile, Egypt, Iran, Islamic Republic of, Italy, Mexico, Saudi Arabia, South Africa, Turkey
Bayer

Completed

BAY81-8973 Pediatric Safety and Efficacy Trial

Haemophilia A

Spain, Russian Federation, United States, Hungary, Italy, Mexico, Norway, Romania, Bulgaria, Canada, Argentina, Denmark, Israel, Poland, Ireland, Latvia, Lithuania
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
Bayer

Completed

Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A

Hemophilia A

Belgium, New Zealand, Netherlands, Greece, United Kingdom, Austria, United States, Italy, Romania, Bulgaria, Canada, Israel, Poland, Lithuania, Norway, Argentina, Spain
Bioverativ, a Sanofi company

Completed

A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A (XTEND-1)

Factor VIII Deficiency

United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, France, Germany, Greece, Hungary, Italy, Japan, Korea, Republic of, Mexico, Netherlands, Spain, Taiwan, United Kingdom
Bayer

Completed

A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A (Leopold II)

Hemophilia A

China, United States, Ukraine, Taiwan, Mexico, Thailand, Romania, Colombia, Turkey, Serbia, Russian Federation, South Africa, Japan, Indonesia, Argentina, Czech Republic, India, Slovakia
Bayer

Completed

Trial to Evaluate the Effect of Secondary Prophylaxis With rFVIII Therapy in Severe Hemophilia A Adult and/or Adolescent Subjects Compared to That of Episodic Treatment (SPINART)

Hemophilia A

United States, Bulgaria, Romania, Argentina
Cooperative International Neuromuscular Research...
United States Department of Defense; National Institutes of Health (NIH); U.S... and other collaborators

Unknown

Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

United States, Canada, Australia, India, Israel, Argentina, Italy, Sweden
Merck KGaA, Darmstadt, Germany
Merck Serono S.A., Geneva

Completed

First Year Growth Response Associated Genetic Markers Validation Phase IV Open-label Study in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children: the PREDICT Pharmacogenetics Validation Study

Turner Syndrome | Idiopathic Growth Hormone Deficiency

Spain, France, Canada, United Kingdom, Czech Republic, Argentina, Germany, Italy, Sweden
Hoffmann-La Roche

Completed

A Study of RG1662 in Adults and Adolescents With Down Syndrome (CLEMATIS)

Down Syndrome

United States, Italy, Argentina, France, Spain, Mexico, Canada, New Zealand, Singapore, United Kingdom
Teva Branded Pharmaceutical Products R&D, Inc.
Nuvelution TS Pharma, Inc.

Completed

A Study to Test if TEV-50717 is Effective in Relieving Tics Associated With Tourette Syndrome (TS) (ARTISTS2)

Tourette Syndrome

United States, Argentina, Australia, Colombia, Hungary, Italy, Korea, Republic of, Mexico, Poland, Ukraine
Genzyme, a Sanofi Company

Recruiting

Fabry Disease Registry & Pregnancy Sub-registry

Fabry Disease

Croatia, United States, Argentina, Australia, Belgium, Bulgaria, Canada, Germany, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Malaysia, Philippines, Poland, Romania, Singapore, Slovakia, Taiwan, Thailand, United Kingdom, Vietn... and more
Alnylam Pharmaceuticals

Active, not recruiting

HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

Transthyretin Amyloidosis | Amyloidosis, Hereditary

United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Cyprus, France, Germany, Greece, Italy, Japan, Korea, Republic of, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, United Kingdom
Genzyme, a Sanofi Company

Recruiting

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

Gaucher Disease | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease

Hungary, Croatia, United States, Australia, Belgium, Brazil, Bulgaria, Canada, Denmark, Germany, Greece, Hong Kong, India, Indonesia, Italy, Jordan, Korea, Republic of, Kuwait, Lebanon, Malaysia, Pakistan, Philippines, Poland, Saudi Arabia and more
Bayer

Completed

Trial to Evaluate the Efficacy and Safety of a New Full Length Recombinant Human FVIII for Hemophilia A (Leopold I)

Hemophilia A | Blood Coagulation Disorders

Spain, Taiwan, Austria, United States, Germany, Thailand, United Kingdom, Italy, Turkey, Croatia, Serbia, Poland, Israel, Sweden, Indonesia, Argentina, Norway, South Africa, India, Hong Kong, Denmark, Pakistan
QED Therapeutics, Inc.

Recruiting

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Achondroplasia

United States, Spain, Singapore, Canada, United Kingdom, France, Australia, Norway, Argentina, Germany, Italy
Women and Infants Hospital of Rhode Island
Sequenom, Inc.

Completed

A New Prenatal Blood Test for Down Syndrome (RNA)

Down Syndrome | Trisomy 21

United States, Canada, Israel, Hungary, Ireland, Argentina, Australia, Czech Republic, Italy, Spain
QED Therapeutics, Inc.

Enrolling by invitation

A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia (PROPEL3)

Achondroplasia

Australia, United States, Argentina, Canada, France, Germany, Italy, Norway, Singapore, Spain, United Kingdom
Bioverativ, a Sanofi company

Active, not recruiting

Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A (XTEND-ed)

Hemophilia A

United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, China, France, Germany, Greece, Hungary, Ireland, Italy, Japan, Korea, Republic of, Netherlands, Spain, Sweden, Switzerland, Taiwan, Turkey, United Kingdom
Bayer

Terminated

BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A

Hemophilia A

Belgium, Italy, Spain, United States, Canada, Switzerland, Estonia, Taiwan, France, Chile, Netherlands, United Kingdom, Austria, New Zealand, South Africa, Romania, Croatia, Israel, Germany, Poland, Australia, Denmark, Turkey, Argentina, Lithuan... and more
Hoffmann-La Roche

Completed

A Study of RO4917523 in Patients With Fragile X Syndrome

Fragile X Syndrome

France, Spain, United States, Peru, Canada, United Kingdom, Chile, Argentina, Mexico, Sweden
Teva Branded Pharmaceutical Products R&D, Inc.
Nuvelution TS Pharma, Inc.

Terminated

Evaluation of Safety and Tolerability of Long-term TEV-50717 (Deutetrabenazine) for Treatment of Tourette Syndrome in Children and Adolescents (ARTISTS)

Tourette Syndrome

United States, Argentina, Australia, Canada, Colombia, Denmark, Hungary, Italy, Korea, Republic of, Mexico, Poland, Russian Federation, Serbia, Spain, Ukraine
Pfizer

Recruiting

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B (BASIS KIDS)

Hemophilia A | Hemophilia B

Denmark, Taiwan, Canada, Turkey, Spain, China, United Kingdom, South Africa, Italy, Saudi Arabia, Israel, Korea, Republic of, Austria, Germany, Japan, Australia, India, Slovakia, France, United States, Brazil, Argentina
Alnylam Pharmaceuticals

Completed

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

Amyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies

United States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
Alnylam Pharmaceuticals

Active, not recruiting

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Australia, Brazil, Bulgaria, France, Italy, Japan, Mexico, Netherlands, Taiwan, United Kingdom, Belgium, Czechia, Denmark, New Zealand, Sweden, Korea, Republic of, Argentina, Chile, Hong Kong, Portugal
BioMarin Pharmaceutical

No longer available

Extension Study of Drisapersen in DMD Subjects

Duchenne Muscular Dystrophy

United States, Germany, United Kingdom, Japan, Netherlands, France, Spain, Korea, Republic of, Italy, Belgium, Taiwan, Argentina, Australia, Bulgaria, Czechia, Israel, Norway, Poland, Russian Federation, Turkey
Pfizer

Completed

Transthyretin Amyloidosis Outcome Survey (THAOS) (THAOS)

Transthyretin Amyloidosis | Transthyretin Gene Mutations

United States, France, Spain, Taiwan, Israel, Korea, Republic of, Denmark, Canada, Netherlands, Japan, Sweden, Brazil, Mexico, Argentina, Portugal, Belgium, Bulgaria, Cyprus, Germany, Italy, Malaysia, Romania, Saudi Arabia, Turkey, United...
Alnylam Pharmaceuticals

Active, not recruiting

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Poland, Argentina, Australia, Belgium, France, Germany, Japan, Netherlands, Portugal, Spain, Sweden, United Kingdom, Canada, Czechia, Denmark, Hungary, Ireland, Norway, Korea, Republic of, Austria, Croatia, Israel, Latvia, Lithuan... and more
Hoffmann-La Roche

Recruiting

GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease.

Huntington Disease

United States, Australia, Argentina, Canada, Germany, Italy, France, United Kingdom, Spain, Portugal, New Zealand, Austria, Poland, Switzerland, Denmark
Novo Nordisk A/S

Completed

Observational Prospective Study on Patients Treated With Norditropin®

Chronic Kidney Disease | Turner Syndrome | Noonan Syndrome | Chronic Renal Insufficiency | Growth Hormone Disorder | Growth Hormone Deficiency in Children | Adult Growth Hormone Deficiency | Genetic Disorder | Foetal Growth Problem | Small for Gestational Age

Israel, Saudi Arabia, Spain, Argentina, Russian Federation, Germany, Netherlands, Italy, Belgium, United Kingdom, Slovenia, Ireland, Sweden, Denmark, Hungary, Finland, Norway, France, Czechia, Lithuania, Luxembourg, Serbia, Switzerland
Hoffmann-La Roche

Completed

Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

United States, Belgium, Canada, Australia, Japan, France, Netherlands, Spain, Argentina, Germany, Italy, United Kingdom, Sweden
Novartis Pharmaceuticals

Completed

An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload

Transfusional Iron Overload in β-thalassemia

United States, Greece, Argentina, Germany, Belgium, Italy, United Kingdom, France, Tunisia, Canada, Turkey, Brazil

Clinical Trials on Urea Cycle Disorders, Inborn in Argentina

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Algeria

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations