Clinical Trials on Genetic Markers in Argentina

Total 194 results

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
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
Alexion Pharmaceuticals, Inc.

Recruiting

Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP (MULBERRY)

Hypophosphatasia

Israel, Korea, Republic of, Taiwan, Sweden, Germany, Italy, Turkey, China, Poland, United States, France, Australia, Thailand, United Kingdom, Mexico, Vietnam, Argentina, Belgium, Brazil, Canada, Spain
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
Pfizer

Active, not recruiting

Study to Evaluate Efficacy and Safety of PF-04965842 With or Without Topical Medications in Subjects Aged 12 Years and Older With Moderate to Severe Atopic Dermatitis (JADE EXTEND)

Dermatitis, Atopic

China, United States, Spain, Taiwan, Germany, Australia, Canada, Hungary, Korea, Republic of, Poland, Serbia, Bulgaria, Czechia, Japan, Latvia, United Kingdom, Belgium, Israel, Italy, Finland, Mexico, Romania, Slovakia, Argentina, Brazil, Chil... and more
Amgen

Recruiting

A Study to Assess Long-term Safety, Tolerability, and Efficacy of Rocatinlimab in Adult and Adolescent Participants With Moderate-to-severe Atopic Dermatitis (AD) (ROCKET-ASCEND)

Atopic Dermatitis

United States, Japan, Korea, Republic of, Australia, Taiwan, Spain, Switzerland, Germany, Croatia, Austria, Bulgaria, France, Denmark, China, Netherlands, Hungary, Czechia, Poland, Brazil, Greece, Portugal, Italy, Finland, Argentina, Sl... and more
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
BioMarin Pharmaceutical

Completed

A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

MPS IV A

United States, Canada, France, United Kingdom, Taiwan, Argentina, Colombia, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, Germany, Portugal, Italy, Qatar
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
Shire

Completed

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

Gaucher Disease, Type 1

United States, Israel, Argentina, India, Korea, Republic of, Paraguay, Poland, Russian Federation, Spain, Tunisia, United Kingdom
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
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
Ultragenyx Pharmaceutical Inc
Mereo BioPharma

Active, not recruiting

Study to Assess Dose, Efficacy and Safety of Setrusumab in Participants With Osteogenesis Imperfecta (Orbit)

Osteogenesis Imperfecta

United States, Turkey, Germany, United Kingdom, Italy, Portugal, Canada, Australia, Argentina, France, Netherlands, Poland
Sanofi
Regeneron Pharmaceuticals

Completed

An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia

Hypercholesterolaemia

Czechia, France, Denmark, Netherlands, Austria, Mexico, Spain, United States, Argentina, Brazil, Bulgaria, Canada, Finland, Hungary, Italy, Lebanon, Norway, Poland, Russian Federation, Slovenia, South Africa, Sweden, Taiwan, Turkey
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
Alexion Pharmaceuticals

Completed

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency (ARISE)

Lysosomal Acid Lipase Deficiency

France, Poland, United Kingdom, Spain, Mexico, Turkey, Japan, Australia, Russian Federation, United States, Germany, Italy, Czechia, Argentina, Croatia
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

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
Shire
Jerini AG

Completed

EASSI - Evaluation of the Safety of Self-Administration With Icatibant (EASSI)

Hereditary Angioedema

Spain, Argentina, Austria, Denmark, France, Germany, Israel, Italy, Switzerland, United Kingdom
Sanofi

Active, not recruiting

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 (ELIKIDS)

Gaucher's Disease Type III | Gaucher's Disease Type I

Canada, Argentina, France, Italy, Japan, Russian Federation, Spain, Sweden, Turkey, 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
Hoffmann-La Roche

Recruiting

A Study To Evaluate The Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Participants With Thyroid Eye Disease

Thyroid Eye Disease | TED

Japan, Australia, Singapore, United States, Argentina, Germany, Italy, Hong Kong
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
Alexion Pharmaceuticals, Inc.

Recruiting

Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa (HICKORY)

Hypophosphatasia

Italy, Spain, Israel, Korea, Republic of, Belgium, Germany, United States, China, France, Taiwan, Austria, Japan, India, Australia, Argentina, Poland, Turkey, Brazil, Canada, Thailand, United Kingdom, Slovakia, Czechia
BioMarin Pharmaceutical

Recruiting

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Hypochondroplasia

United States, Spain, Canada, France, Japan, Australia, Italy, Argentina, Brazil, Germany, United Kingdom
Novartis

Terminated

Tobramycin Inhalation Powder (TIP) in Cystic Fibrosis Subjects (EVOLVE)

Cystic Fibrosis

United States, Argentina, Brazil, Bulgaria, Canada, Chile, Lithuania, Mexico
Pharmaxis
ethica Clinical Research Inc.; Europe: KasaConsult bvba, Hoegaarden, Belgium; Argentina: Resolution Latin America; Buenos Aires, Argentina

Completed

Long Term Administration of Inhaled Mannitol in Cystic Fibrosis

Cystic Fibrosis

United States, France, Belgium, Canada, Argentina, Germany, Netherlands
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
PTC Therapeutics

Terminated

Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

Cystic Fibrosis

United States, Spain, France, Belgium, United Kingdom, Australia, Brazil, Bulgaria, Canada, Germany, Israel, Italy, Netherlands, Poland, Argentina, Greece
Genzyme, a Sanofi Company

Completed

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) (ENCORE)

Gaucher Disease, Type 1

United States, France, Germany, Italy, Brazil, Australia, Argentina, Canada, Egypt, Russian Federation, Spain, 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
Arrowhead Pharmaceuticals

Active, not recruiting

Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS) (PALISADE)

Familial Chylomicronemia

Korea, Republic of, Canada, Australia, Singapore, Belgium, Spain, Japan, United States, Austria, Germany, Ireland, Oman, Poland, Israel, New Zealand, Argentina, France, Croatia, Mexico, Serbia, Turkey
Novartis Pharmaceuticals

Completed

Long Term Study of Canakinumab (ACZ885) in Patients With Gout

Gouty Arthritis

Colombia, Spain, Taiwan, Russian Federation, Guatemala, Belgium, United States, Argentina, Czechia, Germany, Hungary, Poland, Portugal, Singapore, Slovakia, South Africa, Turkey, United Kingdom
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
Amryt Pharma

Recruiting

LOWER: Lomitapide Observational Worldwide Evaluation Registry (LOWER)

Homozygous Familial Hypercholesterolemia

Italy, United States, Argentina, Canada, France, Greece, Netherlands, United Kingdom
Sanofi

Completed

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

Sphingomyelin Lipidosis

Italy, Czechia, Belgium, France, Portugal, Turkey, United States, Argentina, Brazil, Chile, Germany, Romania, Spain
Novartis Pharmaceuticals

Active, not recruiting

Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia (ORION-16)

Familial Hypercholesterolemia - Heterozygous

United States, Germany, South Africa, Greece, Italy, Czechia, Taiwan, Slovakia, Turkey, France, Russian Federation, Canada, Spain, Jordan, Switzerland, Hungary, Poland, Netherlands, Norway, United Kingdom, Lebanon, Argentina, Brazil, ... and more
University of California, San Francisco
National Institute of Neurological Disorders and Stroke (NINDS)

Completed

Epilepsy Phenome/Genome Project (EPGP)

Epilepsy | Infantile Spasms | Lennox-Gastaut Syndrome | Polymicrogyria | Localization-related Epilepsy | Periventricular Heterotopias

United States, Argentina, Australia
SK Life Science, Inc.

Recruiting

Investigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults

Seizures | Lennox Gastaut Syndrome

United States, Korea, Republic of, Taiwan, Colombia, Australia, Poland, Serbia, Mexico, Spain, Greece, Italy, Portugal, Germany, Argentina, Israel
PTC Therapeutics
Cystic Fibrosis Foundation; ECFS-Clinical Trial Network (ECFS-CTN)

Completed

Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)

Cystic Fibrosis

United States, Spain, United Kingdom, Netherlands, Germany, France, Belgium, Italy, Australia, Argentina, Brazil, Bulgaria, Canada, Greece, Israel, Poland
Genzyme, a Sanofi Company

Active, not recruiting

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 (AMETHIST)

Tay-Sachs Disease | Sandhoff Disease

United States, Argentina, Czechia, Portugal, Russian Federation, Turkey, United Kingdom, Brazil, France, Germany, Italy, Japan, Spain
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
Novartis

Completed

Targeted Dose Finding of Canakinumab (ACZ885) for Management of Acute Flare in Refractory or Contraindicated Gout Patients

Acute Gout

United States, Russian Federation, Switzerland, Germany, Turkey, Poland, Argentina, Canada, Belgium, France, United Kingdom
Pfizer

Terminated

A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation (REALM-DCM)

Dilated Cardiomyopathy | Lamin A/C Gene Mutation

United States, Spain, Canada, Belgium, Italy, Argentina, Mexico, Netherlands, Norway, United Kingdom
CSL Behring

Completed

Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks

Hereditary Angioedema

Bulgaria, United States, Poland, Spain, Hungary, United Kingdom, Russian Federation, Macedonia, The Former Yugoslav Republic of, Argentina, Australia, Canada, Czech Republic, Israel, Romania, Sweden
Novartis Pharmaceuticals

Completed

A Study of the Efficacy of Canakinumab in Prevention of Acute Flares in Chronic Gout Patients Initiating Allopurinol Therapy (Core Study) and a Long-term Study of the Efficacy and Safety of Canakinumab in Patients With Gout (Extension Study)

Gout

United States, Colombia, Russian Federation, Taiwan, Argentina, Turkey, Germany, Singapore, Spain, Hungary, Guatemala, Poland, South Africa, Slovakia, Portugal, Belgium, Czechia, United Kingdom
BioMarin Pharmaceutical

Completed

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IV A

United States, Canada, France, Taiwan, Argentina, Colombia, Spain, Turkey, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, United Kingdom, Germany, Norway, Portugal, Italy, Qatar
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
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

Clinical Trials on Genetic Markers in Argentina

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