Clinical Trials on Congenital Deformity in Spain

Total 340 results

Valerion Therapeutics, LLC
Institut de Myologie, France; Genethon

Completed

Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM) (MTM)

Myotubular Myopathy

France, Belgium, United States, Canada, Germany, Italy, Spain
Novo Nordisk A/S

Completed

Investigating Safety and Pharmacokinetics of 2 Different Single Doses of NNC128-0000-2011 in Haemophilia A or B Patients

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Spain, Germany, Japan
Glycomine, Inc.

Recruiting

24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG

Pmm2-CDG

Spain, Australia, United States
Radboud University Medical Center
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Completed

Ursodeoxycholic Acid as Treatment for Polycystic Liver Disease (CURSOR)

Polycystic Kidney, Autosomal Dominant | Polycystic Liver Disease

Netherlands, Spain
Spanish Society of Cardiology

Unknown

Spanish Registry on Spontaneous Coronary Artery Dissection (SR-SCAD)

Spontaneous Coronary Artery Dissection

Spain
Mirum Pharmaceuticals, Inc.

Completed

Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS) (ICONIC)

Alagille Syndrome

France, Belgium, Poland, Australia, Spain, United Kingdom
AstraZeneca

Completed

PK Study of Ticagrelor in Children Aged Less Than 24 Months, With Sickle Cell Disease (HESTIA4) (HESTIA4)

Sickle Cell Disease

Belgium, United Kingdom, Italy, Spain, Kenya, Lebanon
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
Fundacion para la Investigacion Biomedica del Hospital...
University of Alcala; Hospital Universitario Ramon y Cajal; Instituto de Salud... and other collaborators

Recruiting

Prevalence of Hyperandrogenism in Type 1 Diabetes

Polycystic Ovary Syndrome | Hyperandrogenism | Type 1 Diabetes | Hirsutism | Oligomenorrhea | Ovulation Disorder

Spain
University of Valencia
Hospital Universitario La Fe; Hospital General Universitario de Valencia; Hospital...

Completed

Intervention in Chronic Pediatric Patients and Their Families. (FACTORADAPT)

Asthma | Type 1 Diabetes | Allergic Rhinitis | Cystic Fibrosis | Pulmonary Disease | Atopic Dermatitis | Food Allergy | Allergic Asthma | Rhinoconjunctivitis | Primary Ciliary Dyskinesia | Short Stature

Spain
QED Therapeutics, Inc.

Recruiting

Prospective Clinical Assessment Study in Children With Hypochondroplasia (HCH)

Children With Hypochondroplasia

United States, Australia, Norway, Portugal, Singapore, Spain
Taewoong Medical Co., Ltd.
Avania

Active, not recruiting

PULSTA Transcatheter Pulmonary Valve Pre-Approval Study

Heart Diseases | Tetralogy of Fallot | Pulmonary Valve Stenosis | Pulmonary Valve Regurgitation

Germany, Spain, Korea, Republic of, Italy, Netherlands, Turkey
Actelion

Completed

BREATHE 5-OL: Tracleer (Bosentan) in Patients With Pulmonary Arterial Hypertension Related to Eisenmenger Physiology

Pulmonary Arterial Hypertension

Canada, United Kingdom, Italy, United States, Netherlands, Australia, Austria, Belgium, France, Germany, Spain
Mayne Pharma International Pty Ltd

Terminated

A Safety, Efficacy and Systemic Exposure Study of CD5789 Cream in Adults and Adolescents With Lamellar Ichthyosis

Lamellar Ichthyosis

United States, Spain, Canada, Germany, France, Australia, Israel, Ukraine, United Kingdom
Fondazione Telethon

Unknown

Natural History Study in Subjects With Usher Syndrome

Usher Syndrome, Type 1B

Italy, Netherlands, Spain
Novo Nordisk A/S

Completed

Survey Evaluating the Psychosocial Effects of Living With Haemophilia (HERO)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia B | Relatives to/Carers of Patients

Spain, Canada, United Kingdom
Novo Nordisk A/S

Completed

Investigation of the Pharmacokinetics of Turoctocog Alfa in Subjects With Haemophilia A

Congenital Bleeding Disorder | Haemophilia A

Spain, Germany, Malaysia, Latvia
Sanofi

Terminated

Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat (STAGED-PKD-EXT)

Congenital Cystic Kidney Disease

Germany, Spain, United States, Australia, Belgium, Japan, Korea, Republic of, Netherlands
Pierre Fabre Dermatology

Completed

Study in Infants With Infantile Hemangioma to Compare Propranolol Gel to Placebo

Infantile Hemangioma

Spain, France, Poland
Agios Pharmaceuticals, Inc.

Completed

A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease

Healthy Volunteers | Anemia, Sickle Cell

United States, Spain, Denmark
Actelion

Completed

Effects of Tracleer (Bosentan) on Pulmonary Arterial Hypertension Related to Eisenmenger Physiology

Pulmonary Arterial Hypertension Related to Eisenmenger Physiology

Canada, United Kingdom, Italy, United States, Netherlands, Australia, Austria, Belgium, France, Germany, Spain
Novartis Pharmaceuticals

Completed

Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)

Fragile X Syndrome

United States, Spain
Novo Nordisk A/S

Terminated

A Multi-centre, Open Labelled, Multiple Dosing Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of NNC 0172-2021 Administered Subcutaneously to Healthy Male Subjects and Haemophilia Subjects (explorer™2)

Healthy | Congenital Bleeding Disorder | Haemophilia A

Austria, Spain, United Kingdom, France, Germany
Seattle Children's Hospital
Children's Hospital of Philadelphia; University of North Carolina, Chapel Hill and other collaborators

Completed

Craniofacial Microsomia: Accelerating Understanding of the Significance and Etiology (CAUSE)

Craniofacial Microsomia | Goldenhar Syndrome | Microtia | Microtia-Anotia | OAVS | OAV Syndrome | Hemifacial Microsomia

United States, Colombia, Peru, Spain
Phoenicis Therapeutics

Terminated

A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in the COL7A1 Gene

Epidermolysis Bullosa Dystrophica, Recessive | Epidermolysis Bullosa Dystrophica, Dominant

United States, Spain, France
BioCeryx
University of British Columbia; University College London Hospitals; Brugmann... and other collaborators

Unknown

Automated Screen for Fetal Aneuploidy (FAST1)

Trisomy 21 and Other Fetal Aneuploidy

Australia, United Kingdom, Spain, Canada, Belgium, Portugal
Novo Nordisk A/S

Completed

Treatment of Congenital Factor VII Deficiency (F7CONDEF)

Congenital Bleeding Disorder | Congenital FVII Deficiency

Slovakia, Israel, Spain, India, Iran, Islamic Republic of, Pakistan, Turkey, Germany, Serbia, Italy, Greece, Thailand, France, United States, Hong Kong, Venezuela
Intercept Pharmaceuticals

Terminated

Obeticholic Acid in Pediatric Subjects With Biliary Atresia (CARE)

Biliary Atresia

Israel, United Kingdom, Poland, Belgium, France, Germany, Italy, Netherlands, Spain
Novartis Pharmaceuticals

Recruiting

Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation. (EPIK-L1)

Lymphatic Malformations

Spain, France, Germany, Australia, United States
Novo Nordisk A/S

Completed

Comparison of the Action of Drugs in the Body and Safety of N8 and Advate® in Haemophilia A Subjects

Congenital Bleeding Disorder | Haemophilia A

Spain, Germany, Switzerland, Italy, Israel
ArQule, Inc. (a wholly owned subsidiary of Merck...
Worldwide Clinical Trials

Terminated

Study of Miransertib (MK-7075) in Participants With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome (MOSAIC) (MK-7075-002) (MOSAIC)

PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome

United States, Australia, Italy, Spain
Hospital Universitari de Bellvitge
Institut d'Investigació Biomèdica de Bellvitge

Unknown

E6/E7 mRNA Performance to Detect HSIL and Cost-effectiveness Analysis of This Screening Strategy in HIV + MSM (ELAVI-67)

Hiv | Anal Cancer | HPV - Anogenital Human Papilloma Virus Infection | HSIL, High Grade Squamous Intraepithelial Lesions

Spain
Centre d'Etude des Cellules Souches
Genethon

Recruiting

Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome (AUDIOWOLF)

Wolfram Syndrome

France, Spain
Vaderis Therapeutics AG

Recruiting

Assess Safety and Efficacy of VAD044 in HHT Patients

Hereditary Hemorrhagic Telangiectasia (HHT)

United States, Spain, France, Belgium, Netherlands, Italy
Novo Nordisk A/S

Completed

Evaluation of Recombinant Factor XIII for Prevention of Bleeding in Patients With FXIII Inherited Deficiency

Congenital Bleeding Disorder | Congenital FXIII Deficiency

United States, Israel, Austria, Spain, Switzerland, Germany, Canada, France, United Kingdom, Italy, Finland
Hoffmann-La Roche

Completed

Non-drug Study to Evaluate the Suitability of Neurocognitive Tests and Functioning Scales for the Measurement of Cognitive and Functioning Changes in Children With Down Syndrome

Down Syndrome

United States, France, Spain
Igenomix

Recruiting

RCT Study to Validate niPGT-A Clinical Benefit. (niPGT-A_RCT)

Infertility | Aneuploidy | Chromosome Abnormality

France, Argentina, Spain, United States, Brazil, Italy
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
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
Albireo

Enrolling by invitation

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia (BOLD-EXT)

Biliary Atresia

Spain, United States, Netherlands, Malaysia, Canada, Germany, Turkey, Italy, France, Australia, Poland, New Zealand
BioMarin Pharmaceutical

Recruiting

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Hypochondroplasia

United States, Spain, Canada, France, Japan, Australia, Italy, Argentina, Brazil, Germany, United Kingdom
SECRETO Study Consortium
Sahlgrenska University Hospital, Sweden; Academy of Finland; Hospital District... and other collaborators

Recruiting

Searching for Explanations for Cryptogenic Stroke in the Young: Revealing the Etiology, Triggers, and Outcome (SECRETO)

Ischemic Stroke | Thrombosis | Brain Infarction | Foramen Ovale, Patent

Italy, Estonia, Finland, Germany, Lithuania, Netherlands, Norway, Sweden, Turkey, United Kingdom, Greece, Portugal, Spain
Novo Nordisk A/S

Completed

Induction of Puberty With 17-beta Estradiol in Girls With Turner Syndrome

Turner Syndrome | Genetic Disorder

Spain
Novo Nordisk A/S

Completed

Pharmacokinetics of Single Bolus Dose of NovoSeven® in Paediatric and Adult Patients With Haemophilia A or B in a Non- Bleeding State

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Spain, Italy, Greece, Germany, United Kingdom
Natera, Inc.

Completed

Development of Non-invasive Prenatal Screening Test for Microdeletions Based on Fetal DNA Isolated From Maternal Blood (MAPS)

Trisomy 21 | Trisomy 18 | Trisomy 13 | Sex Chromosome Abnormalities | Microdeletion Syndromes

United States, Spain, Taiwan
Regeneron Pharmaceuticals

Recruiting

A Study of DB-OTO, an Adeno-associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations (CHORD)

Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)

Spain, United Kingdom, United States
Abbott Medical Devices

Recruiting

Trevisio Post-Approval Study (TrevisioPAS)

PFO - Patent Foramen Ovale | VSD - Muscular Ventricular Septal Defect | PIVSD - Post Infarct Muscular Ventricular Septal Defect | ASD - Atrial Septal Defect

Spain, Germany, Poland, France, Italy, Netherlands, Switzerland
Novo Nordisk A/S

Completed

Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725 (mentor™2)

Congenital Bleeding Disorder | Congenital FXIII Deficiency

France, United States, Israel, Spain, Switzerland, Germany, Canada, Japan, United Kingdom, Italy, Finland, Austria
Agios Pharmaceuticals, Inc.

Active, not recruiting

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

Pyruvate Kinase Deficiency

United States, Spain, Japan, Canada, United Kingdom, Korea, Republic of, Ireland, Thailand, Italy, Turkey, Denmark, Brazil, France, Germany, Netherlands, Switzerland
Hoffmann-La Roche

Active, not recruiting

A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome

Angelman Syndrome

United States, Spain, Netherlands, Italy

Clinical Trials on Congenital Deformity in Spain

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in United Kingdom

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations