Clinical Trials on Medication Errors and Other Product Use Errors and Issues in Portugal

Total 70 results

Inventiva Pharma

Completed

A Study in MPS VI to Assess Safety and Efficacy of Odiparcil (iMProveS)

Mucopolysaccharidosis VI

United Kingdom, France, Germany, Portugal
Swiss Federal Institute of Technology
Instituto de Investigação em Imunologia

Completed

Inhibiting Dietary Iron Absorption in Subjects With Hereditary Hemochromatosis by a Natural Polyphenol Supplement

Iron Metabolism Disorders | Iron Overload | Polyphenols

Portugal, Switzerland
Universidade do Porto
BioMarin Pharmaceutical; Centro Hospitalar do Porto

Completed

Nutritional Status in Phenylketonuria (TNSPKU)

Phenylketonurias

Portugal
Coopervision, Inc.
Visioncare Research Ltd.

Completed

An Evaluation of the Effectiveness of Dual Focus Soft Contact Lenses in Slowing Mypoia Progression

Myopia

Canada, Portugal, Singapore, United Kingdom
Hospital dos Lusíadas

Active, not recruiting

Ocular Biomarkers for Successful Premium Trifocal Intra-ocular Lenses Implantation

Presbyopia | Low Vision

Portugal
BioMarin Pharmaceutical

Completed

A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

Mucopolysaccharidosis VI | Maroteaux-Lamy Syndrome

United States, Portugal, France
Dopavision GmbH

Active, not recruiting

MyopiaX Treatment for the Reduction of Myopia Progression in Children and Adolescents: Safety and Efficacy Investigation (MyopiaX-1)

Myopia

Spain, Netherlands, Germany, Portugal, United Kingdom
BioMarin Pharmaceutical

Completed

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

MPS VI

United States, Australia, France, Brazil, Germany, Portugal, United Kingdom
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
Ultragenyx Pharmaceutical Inc

Completed

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Mucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VII

United States, Brazil, Mexico, Portugal
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
University of Ulm
University of Pisa; IRCCS Azienda Ospedaliero-Universitaria di Bologna; Medical... and other collaborators

Recruiting

Registry for Patients With Lipodystrophy (ECLip Registry)

Lipodystrophy Acquired | Lipodystrophy Congenital

Austria, France, Germany, Hungary, Italy, Portugal, Spain, Netherlands, Russian Federation, Slovenia, Switzerland, Turkey, United Kingdom
Aeglea Biotherapeutics

Completed

A Study of AEB1102 (Pegzilarginase) in Patients With Arginase I Deficiency (AEB1102)

Arginase I Deficiency | Hyperargininemia

United States, Canada, United Kingdom, Portugal
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
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
Aeglea Biotherapeutics

Completed

A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency

Arginase I Deficiency | Hyperargininemia

United States, Canada, United Kingdom, Portugal
Agios Pharmaceuticals, Inc.

Recruiting

Pyruvate Kinase Deficiency Global Longitudinal Registry (PEAK Registry)

Pyruvate Kinase Deficiency

United States, Germany, Spain, Italy, United Kingdom, Czechia, France, Canada, Korea, Republic of, Netherlands, Turkey, Portugal, Denmark, Ireland, Switzerland, Thailand
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
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
Implantology Institute

Completed

Facial Scanning for Lip Support Assessment in Full Arch Implant Rehabilitations

Prosthesis User | Lip | Soft Tissue

Portugal
BioMarin Pharmaceutical

Completed

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)

United States, France, Australia, Germany, Ireland, Austria, Belgium, Italy, Lithuania, Netherlands, Portugal, Sweden, United Kingdom
Amgen

Completed

Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia) (HAUSER-OLE)

Familial Hypercholesterolemia

Canada, Italy, Norway, Belgium, United States, Turkey, Australia, Austria, South Africa, Poland, Switzerland, Spain, Portugal, Slovenia, Brazil, Russian Federation, Hungary, United Kingdom, Colombia, Czechia, Netherlands, Greece, Ma...
Universidade do Porto
Centro Hospitalar Universitário São João, E.P.E. (CHUSJ); Centro de Investigação... and other collaborators

Recruiting

Systemic Transthyretin Amyloidosis: Carpal Tunnel Syndrome in a Portuguese Population (CarPoS)

Carpal Tunnel Syndrome | Transthyretin Amyloidosis

Portugal
Genzyme, a Sanofi Company

Recruiting

Mucopolysaccharidosis I (MPS I) Registry

Mucopolysaccharidosis I (MPS I)

Croatia, United States, Argentina, Australia, Belgium, Brazil, Canada, Denmark, France, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Lebanon, Malaysia, Netherlands, Pakistan, Philippines, Poland, Saudi Arabia, Singapore, Slovaki... and more
Implantology Institute

Not yet recruiting

3D Evaluation of the Lip Support in a Full-arch Implant-supported Rehabilitations

Prosthesis User | Occlusion | Lip | Digital Technology | Oral Soft Tissue Conditions

Portugal
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
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
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
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
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
PTC Therapeutics

Recruiting

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

Phenylketonuria

United States, Germany, Spain, Australia, Brazil, United Kingdom, Denmark, Mexico, Portugal, Turkey, Canada, Georgia, Italy, Netherlands, Japan
Universidade do Porto
Hospital da Luz, Lisboa; Hospital Pedro Hispano; Centro Hospitalar de Leiria; Centro... and other collaborators

Recruiting

Screening of Fabry Disease in Portuguese Patients With Idiopathic Cardiomyopathies (F-CHECK)

Fabry Disease

Portugal
Ultragenyx Pharmaceutical Inc

Recruiting

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

OTC Deficiency

United States, Portugal, Australia, Italy, Spain, United Kingdom, Japan, Canada, Germany, France, Argentina, Netherlands, Brazil
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
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
Alnylam Pharmaceuticals

Completed

Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant

Amyloidosis, Familial | Transthyretin Amyloidosis

France, Germany, Italy, Portugal, Spain, Sweden, United Kingdom
BioMarin Pharmaceutical
ICON plc

Completed

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) (MARS)

Mucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVA

United States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
BioMarin Pharmaceutical

Terminated

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Late-onset Pompe Disease

United States, United Kingdom, Germany, France, Italy, Portugal, Belgium, Netherlands
CENTOGENE GmbH Rostock
Shire Human Genetic Therapies, Inc.

Completed

Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation (sifap2)

Fabry Disease | Cerebrovascular Accident

Austria, Croatia, France, Germany, Poland, Portugal, Georgia
Kyowa Kirin Pharmaceutical Development Ltd

Active, not recruiting

Registry for Patients With X-Linked Hypophosphatemia (XLH Registry)

X-Linked Hypophosphatemia

Spain, Italy, United Kingdom, France, Belgium, Denmark, Germany, Hungary, Portugal, Norway, Switzerland, Israel, Netherlands, Czechia, Bulgaria, Sweden, Slovenia, Ireland, Latvia, Slovakia
Shire

Terminated

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

Fabry Disease

Spain, Greece, Germany, Portugal, Canada, Finland, Poland, Sweden
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
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
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
Amicus Therapeutics

Recruiting

A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

Fabry Disease

Spain, United States, Japan, Australia, France, Portugal
Alexion

Recruiting

Lysosomal Acid Lipase (LAL) Deficiency Registry (ALX-LALD-501)

Wolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-Deficiency

France, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia
Alnylam Pharmaceuticals

Completed

The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant

ATTR Amyloidosis | Transthyretin (TTR)-Mediated Amyloidosis | Familial Amyloidotic Polyneuropathy (FAP) | Familial Amyloid Neuropathies

France, Germany, Portugal, United Kingdom, Sweden, Spain
Idorsia Pharmaceuticals Ltd.

Completed

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2 (RETRIEVE)

GM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Gaucher Disease, Type 2 | AB Variant Gangliosidosis GM2

United States, Spain, Germany, Italy, Belgium, Brazil, France, Portugal, Switzerland, United Kingdom
CENTOGENE GmbH Rostock
Shire Human Genetic Therapies, Inc.

Completed

Stroke in Young Fabry Patients (sifap1): Frequency of Fabry Disease in Young Stroke Patients (sifap1)

Cerebrovascular Accident

Austria, Belgium, Croatia, Finland, France, Georgia, Germany, Ireland, Italy, Malta, Poland, Portugal, Spain, 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

Clinical Trials on Medication Errors and Other Product Use Errors and Issues in Portugal

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Uzbekistan

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations