Clinical Trials on Error Management Training in Spain

Total 351 results

Prevail Therapeutics
Eli Lilly and Company

Recruiting

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

Gaucher Disease | Gaucher Disease, Type 1

United States, Spain, Germany
Alexion Pharmaceuticals

Completed

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

MPS IIIB (Sanfilippo Syndrome)

Spain, Netherlands, United Kingdom, United States, Brazil
Novartis Pharmaceuticals

Terminated

Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis (HH)

Hereditary Hemochromatosis

Belgium, France, Russian Federation, Romania, Slovakia, Spain, Switzerland
Origin Biosciences

Completed

Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A

Molybdenum Cofactor Deficiency, Type A

United States, Spain, Israel, Norway, Turkey, United Kingdom
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc

Active, not recruiting

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Mucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo A

United States, Australia, Spain
Ultragenyx Pharmaceutical Inc

Terminated

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

Wilson Disease | Ornithine Transcarbamylase Deficiency | Glycogen Storage Disease Type IA

Spain, United Kingdom, Canada, United States, Brazil
Amryt Pharma

Active, not recruiting

Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)

Homozygous Familial Hypercholesterolaemia (HoFH)

Germany, Israel, Italy, Saudi Arabia, Spain, Tunisia
Kyowa Kirin Pharmaceutical Development Ltd

Completed

Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age

X-linked Hypophosphatemia (XLH)

Sweden, Spain, United Kingdom, Italy, Austria, France
SOM Innovation Biotech SA
Hospital Vall d'Hebron

Completed

Study of SOM0226 in Familial Amyloid Polyneuropathy

Familial Amyloid Polyneuropathy (FAP)

Spain
Ultragenyx Pharmaceutical Inc

Completed

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

Mucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VII

Spain, United States, Portugal
Instituto Universitario de Oftalmobiología Aplicada...
Fondo de Investigacion Sanitaria

Completed

Efficacy and Safety of Intravitreal Bevacizumab in the Treatment of Choroidal Neovascular Membranes Associated to High Myopia

Choroidal Neovascularization | Myopia

Spain
Ultragenyx Pharmaceutical Inc

Terminated

Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)

Glucose Transporter Type 1 Deficiency Syndrome

United States, Spain, Denmark, United Kingdom, Australia
Arcturus Therapeutics, Inc.

Recruiting

Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810

OTC Deficiency | Ornithine Transcarbamylase Deficiency | OTCD

Spain, Belgium, United Kingdom, Sweden
CENTOGENE GmbH Rostock

Completed

Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease (LYSO-PROOF)

Lysosomal Storage Diseases | Sphingolipidoses | Gaucher Disease

Albania, Greece, India, Israel, Morocco, Pakistan, Spain
Hospital Universitario 12 de Octubre

Active, not recruiting

Clinical Long Term Evaluation of Glutamine Supplement in MELAS Syndrome

MELAS Syndrome

Spain
Rocket Pharmaceuticals Inc.

Enrolling by invitation

Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy

Fanconi Anemia | Fanconi Anemia Complementation Group A

Spain
Vyluma, Inc.
Syneos Health

Active, not recruiting

CHAMP: Study of NVK-002 in Children With Myopia

Myopia

United States, Hungary, Ireland, Netherlands, Spain, United Kingdom
Allievex Corporation

Completed

A Treatment Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Mucopolysaccharidosis Type IIIB | MPS III B

United States, Taiwan, Spain, Colombia, Germany, United Kingdom, Turkey
Shire

Terminated

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

Sanfilippo Syndrome | Mucopolysaccharidosis (MPS)

United States, United Kingdom, Spain, Germany, France, Italy, Netherlands
Jesús González de la Aleja Tejera

Completed

Glutamine Supplement in MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like Episodes) Syndrome

MELAS Syndrome

Spain
Allievex Corporation

Active, not recruiting

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

Mucopolysaccharidosis Type IIIB | MPS III B

Germany, United States, Turkey, United Kingdom, Colombia, Spain, Taiwan
Hospital Universitari Vall d'Hebron Research Institute
CIEMAT; CIBERER

Completed

Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1

Fanconi Anemia

Spain
Julian Sevilla

Completed

Eltrombopag for Treatment of Fanconi Anemia

Fanconi Anemia

Spain
AstraZeneca

Completed

PLUTO: Pediatric Lipid-redUction Trial of rOsuvastatin

Familial Hypercholesterolemia

United States, Spain, Netherlands, Norway, Canada
Pfizer

Completed

Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045. (TRAMA)

Polyneuropathy | Hereditary Transthyretin Amyloidosis (ATTRv)

Spain
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
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
Recordati Rare Diseases

Recruiting

Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach (PROTECT)

Propionic Acidemia | Methylmalonic Acidemia

France, Spain, Italy, United Kingdom, Norway, Germany, Sweden
Instituto Universitario de Oftalmobiología Aplicada...

Completed

Initial Treatment With Bevacizumab in Choroidal Neovascularization Associated to High Myopia (BENEMCOR)

Choroidal Subfoveal/Juxtafoveal Neovascularization in High Myopia

Spain
Alexion Pharmaceuticals

Completed

Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)

Hypophosphatasia

United States, Spain, Australia, United Kingdom, Italy, France, Canada, Germany, Japan, Russian Federation, Saudi Arabia, Turkey
ModernaTX, Inc.

Recruiting

A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia

Methylmalonic Acidemia

United States, Spain, Canada, Netherlands, France, Australia, United Kingdom
Hospital Infantil Universitario Niño Jesús, Madrid...
Universitat Autonoma de Barcelona; Instituto de Investigación Sanitaria de... and other collaborators

Completed

Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A (FANCOLEN-1)

Fanconi Anemia

Spain
Sanofi
Regeneron Pharmaceuticals

Completed

An 8-Week Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (ODYSSEY KIDS)

Hypercholesterolaemia

Norway, Russian Federation, Sweden, South Africa, Czechia, United States, Canada, France, Netherlands, Spain
Kyowa Kirin Pharmaceutical Development Ltd

Completed

A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth

X-Linked Hypophosphatemia

France, United Kingdom, Spain, Germany, Netherlands
Rocket Pharmaceuticals Inc.

Completed

Natural History of Danon Disease

Danon Disease

Ireland, Czechia, France, Italy, Spain
Pfizer

Completed

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

Gaucher Disease

United Kingdom, Canada, Israel, United States, Australia, Spain
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
IntraBio Inc

Completed

N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

GM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease

United States, Germany, Spain, United Kingdom
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
Agios Pharmaceuticals, Inc.

Recruiting

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period (ACTIVATE-Kids)

Pediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic Anemia

United States, Spain, Germany, Netherlands, Canada, France, Israel, Switzerland, Italy
ModernaTX, Inc.

Completed

"The MaP Study": Mapping the Patient Journey in MMA and PA

Propionic Acidemia | Methylmalonic Acidemia

United States, United Kingdom, Spain, France
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
Horizon Therapeutics, LLC

Completed

Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)

Urea Cycle Disorder

United States, Spain, Italy, Switzerland
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period (ACTIVATE-KidsT)

Pediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic Anemia

United States, Spain, Denmark, Turkey, Netherlands, Czechia, United Kingdom, Germany, Canada, Switzerland
Pfizer

Completed

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Gaucher Disease

Chile, United Kingdom, Canada, Spain, Israel, United States, Australia, South Africa
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
Millendo Therapeutics US, Inc.

Terminated

Nevanimibe HCl for the Treatment of Classic CAH

Congenital Adrenal Hyperplasia

Spain, Israel, France, Brazil, Czechia
ModernaTX, Inc.

Recruiting

A Retrospective Study to Characterize Participants With Propionic Acidemia

Propionic Acidemia

United States, Spain, Netherlands, France, United Kingdom, Australia, Canada, Italy
Pfizer

Completed

A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

Gaucher Disease

United States, Chile, United Kingdom, Israel, Canada, Spain, South Africa, Italy

Clinical Trials on Error Management Training in Spain

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