Clinical Trials on Pyruvate Metabolism, Inborn Errors in Canada

Total 484 results

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
REGENXBIO Inc.
National Heart, Lung, and Blood Institute (NHLBI)

Terminated

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

Homozygous Familial Hypercholesterolemia (HoFH)

United States, Canada, Italy, Netherlands
Alexion Pharmaceuticals

Completed

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

Lysosomal Acid Lipase Deficiency

Spain, Germany, Italy, United States, Croatia, Canada, Russian Federation, Denmark, United Kingdom, Belgium, Mexico, Australia, Netherlands, Brazil, Turkey
Amgen

Completed

Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders (HAUSER-RCT)

Heterozygous Familial Hypercholesterolemia

United States, Canada, Italy, Norway, Belgium, Turkey, Australia, New Zealand, Austria, Czechia, South Africa, Poland, Spain, Switzerland, Netherlands, Taiwan, Finland, Brazil, Portugal, Slovenia, Russian Federation, Hungary, United... and more
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
PTC Therapeutics

Completed

A Study of PTC923 in Participants With Phenylketonuria

Phenylketonuria

Spain, United States, Australia, Germany, United Kingdom, Brazil, Denmark, Canada, France, Georgia, Italy, Mexico, Netherlands, Portugal, Turkey
Horizon Pharma Ireland, Ltd., Dublin Ireland

Completed

Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders

Urea Cycle Disorders

United States, Canada
Amryt Pharma

Recruiting

LOWER: Lomitapide Observational Worldwide Evaluation Registry (LOWER)

Homozygous Familial Hypercholesterolemia

Italy, United States, Argentina, Canada, France, Greece, Netherlands, United Kingdom
Kastle Therapeutics, LLC
Ionis Pharmaceuticals, Inc.

Completed

Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia (RADICHOL 1)

Metabolic Diseases | Congenital Abnormalities | Infant, Newborn, Diseases | Genetic Diseases, Inborn | Dyslipidemias | Metabolism, Inborn Errors | Lipid Metabolism Disorders | Hypercholesterolemia | Hyperlipidemias | Hyperlipoproteinemias | Lipid Metabolism, Inborn Errors | Hyperlipoproteinemia Type II | Metabolic... and other conditions

United States, Taiwan, Brazil, Canada, South Africa, United Kingdom, Singapore
Aeglea Biotherapeutics

Completed

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

Arginase I Deficiency | Hyperargininemia

United States, Italy, Canada, France, United Kingdom, Austria, Germany
Alexion Pharmaceuticals

Completed

Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency

Lysosomal Acid Lipase Deficiency | LAL-Deficiency | Cholesterol Ester Storage Disease (CESD)

France, United Kingdom, United States, Canada, Czechia
Pfizer

Completed

A 52 Week Study To Assess The Use Of Bococizumab (PF-04950615; RN316) In Subjects With Heterozygous Familial Hypercholesterolemia (SPIRE-FH)

Heterozygous Familial Hypercholesterolemia

Canada, Netherlands, United Kingdom, United States, Spain, Poland, Finland, Norway, South Africa, Italy, Bulgaria
Novartis Pharmaceuticals

Recruiting

Specified Drug-use Survey of Leqvio for s.c. Injection.

Hypercholesterolaemia | Familial Hypercholesterolaemia

Japan
Horizon Therapeutics, LLC

Completed

A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders

Urea Cycle Disorder

United States, Canada
Horizon Pharma Ireland, Ltd., Dublin Ireland

Completed

Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

Urea Cycle Disorders

United States, Canada
Ultragenyx Pharmaceutical Inc

Active, not recruiting

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency (CAPtivate)

Ornithine Transcarbamylase (OTC) Deficiency

Canada, United States, France, Spain, United Kingdom
Genzyme, a Sanofi Company

Completed

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

Gaucher Disease, Type 1

United States, United Kingdom, Bulgaria, Canada, Colombia, India, Israel, Lebanon, Mexico, Russian Federation, Serbia, Tunisia
Ultragenyx Pharmaceutical Inc

Recruiting

Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency

OTC Deficiency

United States, Portugal, Italy, Spain, United Kingdom, Japan, Canada, Germany, France, Argentina, Netherlands, Brazil
Ardea Biosciences, Inc.

Completed

Allopurinol Combination Study (RDEA594-203)

Gout

Poland, United States, Spain, Canada, Ukraine, United Kingdom
Eloxx Pharmaceuticals, Inc.

Terminated

A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis

Genetic Disease | Cystinosis | Nonsense Mutation

Canada
Pfizer

Terminated

Carotid B-mode Ultrasound Study to Compare Anti-Atherosclerotic Effect of Torcetrapib/Atorvastatin to Atorvastatin. (RADIANCE 2)

Mixed Hyperlipidemia

United States, Canada, Czech Republic, Finland, France, Netherlands
Pacific Parkinson's Research Centre
University of British Columbia; Oregon Health and Science University; University... and other collaborators

Recruiting

The GBA Multimodal Study in Parkinson's Disease

Parkinson Disease | Gaucher Disease | GBA Gene Mutation

United States, Canada
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)

X-linked Hypophosphatemia

United States, Korea, Republic of, Canada, Japan, France, Denmark
Actelion

Completed

Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease

Gaucher Disease Type 1

United States, Taiwan, Canada, Spain, Australia, France, Brazil, Hungary, Czechia, Germany, Italy, Netherlands, United Kingdom
Novartis Pharmaceuticals

Recruiting

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Adolescent ORION-16 or ORION-13 Studies (V-PEDS-OLE)

Heterozygous or Homozygous Familial Hypercholesterolemia

Netherlands, Israel, Hungary, Italy, Germany, Spain, United States, France, Norway, Russian Federation, South Africa, Turkey, United Kingdom, Canada, Switzerland, Brazil, Lebanon, Slovenia, Poland, Taiwan, Malaysia
CymaBay Therapeutics, Inc.

Completed

Evaluate the Efficacy and Safety of Arhalofenate for Preventing Flares and Reducing Serum Uric Acid in Gout Patients

Gout

United States, Canada, Georgia
CymaBay Therapeutics, Inc.

Completed

Safety/Efficacy Study to Evaluate of MBX-102 in Combination With Allopurinol in Gout Patients

Gout

United States, Canada, Georgia
Novartis Pharmaceuticals

Completed

A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome

Familial Chylomicronemia Syndrome (FCS)

South Africa, Spain, France, Germany, United Kingdom, United States, Canada, Netherlands
McGill University Health Centre/Research Institute...
Children's Hospital and Medical Center, Omaha, Nebraska

Completed

Betaine and Peroxisome Biogenesis Disorders

Peroxisome Biogenesis Disorders

Canada
Ionis Pharmaceuticals, Inc.

Active, not recruiting

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)

Familial Chylomicronemia Syndrome

Spain, United Kingdom, France, Canada, United States, Italy, Netherlands, Norway, Portugal, Slovakia, Sweden
Ardea Biosciences, Inc.

Completed

Open-Label Lesinurad Monotherapy Extension Study in Gout

Gout

United States, Canada, Belgium, Australia, Germany, New Zealand, South Africa
Genzyme, a Sanofi Company

Completed

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

Gaucher Disease

United States, Sweden, India, Netherlands, Austria, Japan, China, Serbia, France, Croatia, Australia, Portugal, Canada, Brazil, Greece, Romania, Russian Federation
University of Toronto

Unknown

Umbrella Review of the DASH Dietary Pattern and Cardiometabolic Risk

Cardiovascular Diseases | Stroke | Inflammation | Diabetes | Blood Pressure | Coronary Heart Disease | Adiposity | Cardiovascular Risk Factor | Cholesterol; Lipidosis

Canada
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC

Completed

A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease

Mucopolysaccharidosis I | Scheie Syndrome | Hurler's Syndrome | Hurler-Scheie Syndrome

Brazil, Canada
Ardea Biosciences, Inc.

Completed

Lesinurad Monotherapy in Gout Subjects Intolerant to Xanthine Oxidase Inhibitors (LIGHT)

Gout

United States, Canada, South Africa, New Zealand, Belgium, Australia, Germany
Swedish Orphan Biovitrum

Completed

Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care (OPAL)

Hereditary Tyrosinemia, Type I

Belgium, Denmark, France, Germany, Austria, Czechia, Finland, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom
Ardea Biosciences, Inc.

Completed

Lesinurad and Febuxostat Combination Extension Study in Gout

Gout

United States, Canada, Australia, New Zealand, Switzerland, Poland
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
Canadian Fabry Research Consortium
Nova Scotia Health Authority

Recruiting

Canadian Fabry Disease Initiative (CFDI) National Registry (CFDI-NR)

Fabry Disease

Canada
Novartis Pharmaceuticals

Terminated

Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.

Familial Chylomicronemia Syndrome (FCS) (HLP Type I)

South Africa, Germany, United Kingdom, France, United States, Canada, Netherlands
Amicus Therapeutics

Completed

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease

Fabry Disease

United States, Australia, France, United Kingdom, Brazil, Canada
Amicus Therapeutics

Completed

Drug-drug Interaction Study

Pompe Disease

France, United States, Canada, United Kingdom
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Efficacy and Safety of Burosumab (KRN23) Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With X Linked Hypophosphatemia (XLH)

X-Linked Hypophosphatemia

Korea, Republic of, Canada, United States, Australia, Japan, United Kingdom, Sweden
University of British Columbia
Canadian Institutes of Health Research (CIHR); AllerGen NCE Inc.

Recruiting

Diesel Exhaust Induces Glucocorticoid Resistance (DIGR)

Exposure to Pollution | Glucocorticoid Resistance

Canada
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics

Completed

The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome

Familial Chylomicronemia Syndrome

United States, Brazil, Canada, France, Germany, Hungary, Israel, Italy, Netherlands, South Africa, Spain, United Kingdom
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics

Completed

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS) (BALANCE)

Familial Chylomicronemia Syndrome

United States, Israel, Spain, Italy, United Kingdom, Netherlands, Canada, Hungary, Sweden, France, Norway, Portugal, Slovakia
Passage Bio, Inc.

Suspended

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease (GALax-C)

Leukodystrophy, Globoid Cell

United States, Brazil, Netherlands, United Kingdom, Canada, Israel
Alexion Pharmaceuticals

Completed

An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype

Lysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD)

United States, United Kingdom, France, Canada, Czech Republic, Italy, Poland, Switzerland
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC

Completed

Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

Mucopolysaccharidosis I | Hurler-Scheie Syndrome | Hurlers Syndrome

United States, Canada, Germany
Novartis Pharmaceuticals

Completed

Canakinumab in the Treatment of Acute Gout Flares and Prevention of New Flares in Patients Unable to Use Non-steroidal Anti-inflammatory Drugs (NSAIDs) and/or Colchicine Including a 12 Weeks Extension and an Open-label 48 Weeks Extension Study (β-RELIEVED)

Acute Gout

Colombia, Switzerland, Germany, Russian Federation, Singapore, Australia, Belgium, Canada, Estonia, Guatemala, Latvia, Lithuania, Mexico, Norway, Poland, Sweden, Ukraine

Clinical Trials on Pyruvate Metabolism, Inborn Errors in Canada

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