Clinical Trials on Metabolism, Inborn Errors in Spain

Total 2823 results

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
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
Sanofi

Recruiting

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

Glycogen Storage Disease Type II

Belgium, United States, Italy, Spain, Taiwan, United Kingdom, Netherlands, France, Germany
Freeline Therapeutics

Recruiting

A Gene Therapy Study in Patients With Gaucher Disease Type 1 (GALILEO-1)

Gaucher Disease, Type 1

Spain, Israel, United States, United Kingdom, Brazil, Germany, Paraguay
Ardea Biosciences, Inc.

Completed

Allopurinol Combination Study (RDEA594-203)

Gout

Poland, United States, Spain, Canada, Ukraine, United Kingdom
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
Sanofi

Recruiting

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa (Baby-COMET)

Glycogen Storage Disease Type II

Belgium, United States, China, Germany, Italy, Netherlands, Spain, Taiwan, United Kingdom
Ultragenyx Pharmaceutical Inc

Completed

Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

United States, Spain, Australia, France, Israel, Italy, United Kingdom
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
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
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, Turkey, Spain, United States, France, Norway, Russian Federation, South Africa, United Kingdom, Canada, Switzerland, Brazil, Lebanon, Slovenia, Poland, Taiwan, Malaysia
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
Emalex Biosciences Inc.
Psyadon Pharma

Terminated

Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease

Lesch-Nyhan Disease | Self-injurious Behavior

Spain, United States
Astellas Gene Therapies

Recruiting

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

Pompe Disease (Late-onset)

United States, Spain, France, Taiwan, Italy
Allievex Corporation

Active, not recruiting

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Mucopolysaccharidosis Type IIIB

United States, Germany, Spain, Argentina, Australia, Brazil, Colombia, Taiwan, Turkey
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
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
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
Ultragenyx Pharmaceutical Inc

Active, not recruiting

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

Glycogen Storage Disease Type IA | Von Gierke's Disease (GSD Type Ia)

Netherlands, United States, Canada, Spain
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
Protalix
Chiesi Farmaceutici S.p.A.

Completed

Extension Study of PRX-102 for up to 60 Months

Fabry Disease

United States, Spain, United Kingdom, Paraguay
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
Sanofi

Recruiting

Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia (upreACH-2)

Osteochondrodysplasia

Australia, Spain, Korea, Republic of, China, Italy
Ultragenyx Pharmaceutical Inc

Terminated

Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

United States, Spain, France, Germany, Italy, United Kingdom
Takeda

Withdrawn

A Study to Learn About Metachromatic Leukodystrophy (MLD) in Children in Spain (mECHromatic)

Metachromatic Leukodystrophy (MLD)

Spain
Ardea Biosciences, Inc.

Completed

Combining Lesinurad With Allopurinol in Inadequate Responders (CLEAR 2)

Gout

United States, Germany, New Zealand, South Africa, Spain, Canada, Switzerland, Australia, Belgium, Poland, Ukraine
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
Hospital Universitari Vall d'Hebron Research Institute
Universidad de Murcia

Unknown

"Incidence and Consequences of Disorders of Glycosylation in Patients With Conotruncal and Septal Heart Defects" ((CARDIoG))

Congenital Heart Diseases | Antithrombin III Deficiency | Congenital Disorder of Glycosylation | Conotruncal Defects

Spain
Shire

Completed

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

Sanfilippo Syndrome

United States, Spain, United Kingdom, Argentina, France, Germany, Italy, Netherlands
Alexion Pharmaceuticals

Terminated

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB

MPS IIIB (Sanfilippo B Syndrome)

Spain, United States, Brazil, United Kingdom, Portugal, Italy
Ardea Biosciences, Inc.

Completed

Lesinurad and Allopurinol Combination Extension Study in Gout

Gout

United States, South Africa, Spain, Belgium, Australia, Germany, Canada, New Zealand, Poland, Ukraine, Switzerland
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
BioMarin Pharmaceutical

Completed

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency (KAMPER)

Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency

Sweden, France, Italy, Spain, Germany, Netherlands, Austria, Slovakia, Portugal
Ultragenyx Pharmaceutical Inc

Active, not recruiting

A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Glycogen Storage Disease Type IA

United States, Brazil, Canada, Denmark, Germany, Italy, Netherlands, Spain
Alexion Pharmaceuticals, Inc.

Completed

Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840

Wilson Disease

United States, Spain, United Kingdom, New Zealand, Denmark, Russian Federation, Canada, Singapore
Allievex Corporation

Completed

A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Mucopolysaccharidosis Type IIIB | Mucopolysaccharidosis Type 3 B | MPS III B | MPS 3 B

United States, Spain, Turkey, Taiwan, Australia, Colombia, Germany, United Kingdom
Pfizer's Upjohn has merged with Mylan to form Viatris...

Completed

Celebrex In Acute Gouty Arthritis Study

Arthritis, Gouty

Canada, United States, Spain, Russian Federation, Colombia, Costa Rica, Peru, Taiwan, Korea, Republic of, Mexico, Philippines, Thailand
Novartis Pharmaceuticals

Completed

Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C (ORION-8)

Elevated Cholesterol | Homozygous Familial Hypercholesterolemia | Heterozygous Familial Hypercholesterolemia | ASCVD

United States, Canada, Czechia, Denmark, Germany, Hungary, Netherlands, Poland, South Africa, Spain, Sweden, Ukraine, United Kingdom
Ultragenyx Pharmaceutical Inc
PRA Health Sciences

Recruiting

Mucopolysaccharidosis VII Disease Monitoring Program

Sly Syndrome | Mucopolysaccharidosis VII | MPS VII | MPS 7

United States, Germany, Brazil, Argentina, France, Netherlands, Portugal, Spain, Turkey
Orphalan
Ergomed

Recruiting

International Wilson's Disease Patient Registry (iWilson Registry)

Wilson's Disease

Spain, United Kingdom, Belgium, Germany, Poland, France, Saudi Arabia
Alexion Pharmaceuticals, Inc.

Terminated

Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease

Wilson Disease

Spain, Korea, Republic of, France, Germany, Japan, Australia, Poland, United Kingdom
Shire

Terminated

A Study of Replagal in Treatment-naïve Adults With Fabry Disease

Fabry Disease

Spain, Greece, Germany, Portugal, Canada, Finland, Poland, Sweden
Protalix
Chiesi Farmaceutici S.p.A.

Completed

Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

Fabry Disease

United States, Serbia, Spain, United Kingdom, Australia, Paraguay
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
Akcea Therapeutics
Ionis Pharmaceuticals, Inc.

Completed

The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome

Familial Chylomicronemia Syndrome | Lipoprotein Lipase Deficiency | Hyperlipoproteinemia Type 1

United States, Canada, Germany, Israel, Italy, South Africa, Spain, United Kingdom, Brazil, France, Netherlands
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
Ultragenyx Pharmaceutical Inc

Active, not recruiting

Study of UX701 Gene Transfer for the Treatment of Wilson Disease

Wilson Disease

United States, Canada, Spain, Portugal, United Kingdom
BioMarin Pharmaceutical

Terminated

A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

Late-onset Pompe Patients Untreated or Treated With rhGAA

Spain, Slovenia, Serbia, Korea, Republic of, Greece, Australia, Singapore, Brazil, Canada, Taiwan, Ireland, Poland, Romania
Rocket Pharmaceuticals Inc.

Completed

Natural History of Danon Disease

Danon Disease

Ireland, Czechia, France, Italy, Spain

Clinical Trials on Metabolism, Inborn Errors in Spain

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