Clinical Trials on Low Birth Weight in Portugal

Total 297 results

Novo Nordisk A/S

Completed

Observational Study on Safety of Room Temperature Stable NovoSeven® in Patients With Haemophilia A or B

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Belgium, Germany, France, United Kingdom, Italy, Hungary, Austria, Sweden, Iran, Islamic Republic of, Argentina, Ireland, Poland, Portugal, Slovakia
Astellas Pharma Inc

Completed

Equivalence of the Response to Vaccination of Tacrolimus Ointment to a Steroid Ointment Regimen in Children With Atopic Dermatitis

Atopic Dermatitis

Australia, Belgium, Poland, Hungary, Portugal, Germany, Iceland, Malta
Bayer
Regeneron Pharmaceuticals

Active, not recruiting

Extension Study to Evaluate the Long-term Outcomes of Subjects in Study 20090 (FIREFLEYE next)

Retinopathy of Prematurity (ROP)

Spain, Korea, Republic of, Singapore, Malaysia, Japan, Taiwan, Bulgaria, Italy, Argentina, Czechia, Greece, Israel, Netherlands, Portugal, Romania, Russian Federation, Slovakia, Sweden, Turkey, United Kingdom, Ukraine, Hungary, Belgium and more
Janssen-Cilag International NV

Completed

Treatment Simplification by Darunavir/Ritonavir 800/100 mg Once a Day Versus a Triple Combination Therapy With Darunavir/Ritonavir (MONET)

HIV Infections | Human Immunodeficiency Virus | Acquired Immunodeficiency Syndrome Virus | AIDS Virus

United Kingdom, Belgium, Germany, Spain, Portugal, Israel, Denmark, Russian Federation, Austria, Hungary, Switzerland
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
Octapharma

Completed

Surveillance of Safety and Efficacy of Wilate in Patients With Von Willebrand Disease

Von Willebrand Disease

United States, Canada, Sweden, Spain, Uruguay, United Kingdom, Portugal, Argentina, Czechia, Germany
CytoSorbents, Inc

Recruiting

CytOSorb TreatMent Of Critically Ill PatientS Registry (COSMOS)

Sepsis | Burns | Septic Shock | Trauma | Infectious Disease | Pancreatitis | Acute Respiratory Distress Syndrome | Liver Transplant; Complications | Drug Overdose | Acute Liver Failure | Cardiogenic Shock | Rhabdomyolysis | Acute on Chronic Liver Failure | Hemophagocytic Lymphohistiocytoses | Extracorporeal Life Support and other conditions

Spain, Italy, Germany, Portugal
European Organisation for Research and Treatment...

Completed

Liposomal Amphotericin B in Treating Granulocytopenia and Persistent Unexplained Fever in Cancer Patients

Cancer

Belgium, Netherlands, Italy, Hungary, France, Spain, Germany, Israel, Czech Republic, United Arab Emirates, Sweden, Greece, Turkey, Portugal, Slovakia
ViiV Healthcare
GlaxoSmithKline

Completed

A Study To Assess GW433908 (Fosamprenavir) Containing Regimens In HIV-1 Infected Subjects

Infection, Human Immunodeficiency Virus

United States, Spain, France, United Kingdom, Chile, Brazil, Italy, Portugal
Seelos Therapeutics, Inc.

Terminated

STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia (STRIDES)

Spinocerebellar Ataxia Type 3

United States, Spain, Korea, Republic of, Australia, United Kingdom, Brazil, Germany, Portugal
Biogen

Terminated

A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3

Spinocerebellar Ataxia Type 3

United States, Netherlands, Israel, Portugal, United Kingdom, Germany
Baxalta now part of Shire
Baxalta Innovations GmbH, now part of Shire

Completed

Phase 3/4 Study of a Recombinant Protein-Free Factor VIII (rAHF-PFM): Comparison of Continuous Infusion Versus Intermittent Bolus Infusion in Hemophilia A Subjects Undergoing Major Orthopedic Surgery

Hemophilia A

United States, Belgium, France, Norway, Netherlands, Austria, Hungary, Italy, Poland, Portugal, Romania, Russian Federation, Spain, Sweden
Novo Nordisk A/S

Completed

Semaglutide Effects on Heart Disease and Stroke in Patients With Overweight or Obesity (SELECT)

Obesity | Overweight

United States, France, Spain, Taiwan, United Kingdom, Germany, India, Brazil, Canada, Japan, Malaysia, Russian Federation, Australia, Norway, Mexico, Netherlands, Portugal, Hungary, Italy, Austria, Greece, South Africa, Turkey, Finland, Puerto Rico and more
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
Ionis Pharmaceuticals, Inc.

Completed

Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

Amyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | Transthyretin

United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
Novo Nordisk A/S

Completed

Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B With Inhibitors (ONE)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

Saudi Arabia, Algeria, Turkey, Germany, Poland, Netherlands, Italy, Belgium, South Africa, United Kingdom, Sweden, Austria, Portugal, France, Czech Republic, Venezuela
Alnylam Pharmaceuticals

Completed

The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)

Amyloidosis

United States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Netherlands, Portugal, Spain, Sweden, Taiwan, United Kingdom, Germany, France, Korea, Republic of, Brazil, Mexico, Turkey
Boehringer Ingelheim

Recruiting

A Study to Test the Effect of Survodutide (BI 456906) on Cardiovascular Safety in People With Overweight or Obesity (SYNCHRONIZE™ - CVOT)

Obesity

United States, Korea, Republic of, Belgium, Australia, United Kingdom, Mexico, Spain, Germany, Taiwan, Netherlands, Brazil, Hungary, Denmark, Canada, Poland, Austria, China, Finland, Greece, Italy, Hong Kong, Czechia, Portugal, Saudi... and more
ViiV Healthcare
Pfizer

Active, not recruiting

An Open Label Pharmacokinetic, Safety And Efficacy Study Of Maraviroc In Combination With Background Therapy For The Treatment Of HIV-1 Infected, CCR5 -Tropic Children

Human Immunodeficiency Virus

Spain, United States, Italy, South Africa, Brazil, Mexico, Portugal, Puerto Rico, Thailand
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.

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
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
Ascendis Pharma A/S

Completed

A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia

Achondroplasia

United States, Australia, Austria, Germany, China, Canada, Denmark, France, Ireland, Italy, New Zealand, Portugal, Spain, Switzerland, United Kingdom
Octapharma

Completed

Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients

Severe Hemophilia A

Canada, United States, France, Spain, Ukraine, United Kingdom, Belarus, Georgia, Germany, India, Italy, Moldova, Republic of, Morocco, Poland, Portugal, Russian Federation, Slovenia
Santhera Pharmaceuticals

Completed

Study to Assess the Efficacy and Safety of Raxone in LHON Patients (LEROS)

Leber's Hereditary Optic Neuropathy (LHON)

United States, United Kingdom, Spain, Poland, Austria, Belgium, Bulgaria, Germany, Italy, Portugal
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
QED Therapeutics, Inc.

Recruiting

Prospective Clinical Assessment Study in Children With Hypochondroplasia (HCH)

Hypochondroplasia

United States, Australia, Norway, Portugal, Singapore, Spain, Canada, France, Germany, Sweden, United Kingdom
Boehringer Ingelheim

Recruiting

A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome

Netherton Syndrome

Belgium, United Kingdom, Australia, Germany, China, Malaysia, United States, Japan, France, Israel, Bulgaria, Italy, Finland, Switzerland, Netherlands, Portugal
CSL Behring

Completed

Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A

Congenital Hemophilia A

United States, Austria, Germany, Malaysia, Netherlands, Philippines, Ukraine, Romania, Spain, Australia, France, Georgia, Italy, Lebanon, Poland, Portugal, Switzerland, Thailand, Turkey
PedNet Haemophilia Research Foundation
Lund University Hospital

Recruiting

The European Paediatric Network for Haemophilia Management ( PedNet Registry) (PedNet)

Factor IX Deficiency | Factor VIII Deficiency

Norway, Austria, Belgium, Canada, Czechia, Denmark, Finland, France, Germany, Greece, Ireland, Israel, Italy, Netherlands, Portugal, Spain, Sweden, Switzerland, United Kingdom
Pfizer

Terminated

Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.

Achondroplasia

United States, Canada, China, Japan, Australia, Germany, France, United Kingdom, Denmark, Belgium, Italy, Portugal, Spain, Switzerland
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
University of Leicester

Unknown

Chemotherapy Plus Surgery in Treating Children at Risk of or With Stage I Wilms' Tumor

Kidney Cancer

France, Netherlands, Sweden, Norway, Denmark, Portugal, Belgium, Croatia, Czech Republic, Greece, Italy, Poland, Slovenia, Spain, United Arab Emirates
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
KU Leuven

Completed

Assessment of Patterns of Patient Reported Outcomes in Adults With Congenital Heart Disease - International Study II (APPROACH-IS II)

Congenital Heart Disease

Botswana, United States, Korea, Republic of, Belgium, Sweden, France, Switzerland, Denmark, Pakistan, Norway, Taiwan, Malta, Brazil, Bulgaria, India, Austria, Argentina, Australia, Cameroon, Canada, Chile, Colombia, Ethiopia, Greece, Italy and more
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...
AstraZeneca

Recruiting

PASS of Paediatric Patients Initiating Selumetinib

Neurofibromatosis Type 1

Spain, United Kingdom, Israel, France, Italy, Portugal, Switzerland, Germany, Austria
Takeda

Completed

A Study of the Burden of Illness and Treatment Patterns in Teenagers and Adults With Hereditary Angioedema (BOISTERN)

Hereditary Angioedema

Belgium, Bulgaria, Estonia, Spain, Hungary, Israel, Germany, Czechia, Portugal, Greece, Lithuania, Poland, Latvia, Croatia, Slovakia, Austria, Canada, Ireland, North Macedonia, Romania, Serbia, Slovenia
Novo Nordisk A/S

Recruiting

A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors

Haemophilia A With or Without Inhibitors

India, China, Korea, Republic of, Canada, Spain, Netherlands, South Africa, United States, Israel, Poland, United Kingdom, Italy, Japan, Lithuania, Portugal, Switzerland, Taiwan
Janssen R&D Ireland

Completed

A Study to Assess the Safety, Tolerability and Efficacy of TMC435 Along With Pegylated Interferon Alpha-2a (Pegasys) and Ribavirin (Copegus) Triple Therapy in Chronic Hepatitis C Genotype-1 Infected Patients Co-infected With Human Immunodeficiency Virus-Type 1

Hepatitis C Virus Genotype-1

United States, France, United Kingdom, Germany, Spain, Portugal, Canada, Puerto Rico
European Society of Anaesthesiology

Completed

Prospective Evaluation of a RIsk Score for Postoperative Pulmonary COmPlications in Europe (PERISCOPE)

Complication of Surgical Procedure | Pulmonary Failure | Surgical Complications From General Anesthesia | Postoperative Respiratory Complications | Acute Respiratory Failure, Adult

Belgium, Spain, Albania, Bosnia and Herzegovina, Croatia, Czechia, Estonia, France, Germany, Hungary, Italy, Latvia, Lithuania, Luxembourg, Poland, Portugal, Romania, Russian Federation, Switzerland, Turkey, Ukraine
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
Vertex Pharmaceuticals Incorporated

Active, not recruiting

Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

Cystic Fibrosis

Spain, Belgium, Netherlands, France, Canada, Germany, Sweden, Switzerland, Italy, Czechia, Portugal, Austria, Hungary, Norway, Poland
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
OHB Neonatology Ltd.

Recruiting

A Clinical Efficacy and Safety Study of OHB-607 in Preventing Bronchopulmonary Dysplasia in Extremely Premature Infants

Intraventricular Hemorrhage | Bronchopulmonary Dysplasia | Chronic Lung Disease of Prematurity | Retinopathy of Prematurity (ROP)

Finland, United States, France, Italy, United Kingdom, Germany, Canada, Ireland, Japan, Netherlands, Portugal, Spain, Sweden
Gilead Sciences

Completed

Study to Evaluate Switching From Regimens Consisting of a Nonnucleoside Reverse Transcriptase Inhibitor Plus Emtricitabine and Tenofovir DF to the Elvitegravir/Cobicistat/Emtricitabine/Tenofovir DF Single-Tablet Regimen in Virologically Suppressed, HIV-1 Infected Patients

HIV Infections | Acquired Immunodeficiency Syndrome

United States, United Kingdom, France, Spain, Germany, Belgium, Puerto Rico, Canada, Italy, Austria, Australia, Portugal
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
Clementia Pharmaceuticals Inc.

Terminated

An Efficacy and Safety Study of Palovarotene for the Treatment of MO (MO-Ped)

Exostoses, Multiple Hereditary

United States, Spain, Canada, Japan, Italy, Portugal, United Kingdom, Turkey, Australia, Belgium, France, Netherlands
PTC Therapeutics
Cooperative International Neuromuscular Research Group; The John Walton Muscular...

Active, not recruiting

Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Muscular Dystrophy, Duchenne

Germany, France, United Kingdom, Brazil, Italy, Austria, Sweden, Czechia, Greece, Hungary, Israel, Latvia, Norway, Portugal, Romania, Slovenia
Calcilytix Therapeutics, Inc., a BridgeBio company

Recruiting

ADH1 and ADH2 Disease Monitoring Study (DMS) (CLARIFY)

Autosomal Dominant Hypocalcemia

United States, France, Denmark, Australia, Japan, Belgium, Canada, Italy, Netherlands, United Kingdom, Portugal, Finland, Germany

Clinical Trials on Low Birth Weight in Portugal

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