Clinical Trials on X-linked Adrenoleukodystrophy in Spain

Total 178 results

Onofre, Aurora Pujol, M.D.
Instituto de Salud Carlos III; Fundacion Hesperia; ELA España Association

Completed

Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy (XAMNPIOP2011)

Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy

Spain
Minoryx Therapeutics, S.L.

Active, not recruiting

A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT) (NEXUS)

Cerebral Adrenoleukodystrophy

Spain, Germany, France, Argentina
Edimer Pharmaceuticals

Completed

Evaluation of Phenotypic and Genetic Properties in Male Subjects Affected By Hypohidrotic Ectodermal Dysplasia (ECP-012) (ECP-012)

X-linked Hypohidrotic Ectodermal Dysplasia | Hypohidrotic Ectodermal Dysplasia

Spain
MedDay Pharmaceuticals SA

Completed

MD1003-AMN MD1003 in Adrenomyeloneuropathy

Adrenoleukodystrophy | Adrenomyeloneuropathy | AMN

France, Germany, Spain
Onofre, Aurora Pujol, M.D.

Completed

Plasma Exchange With Albumin in AMN Patients

Adrenoleukodystrophy | Adrenomyeloneuropathy

Spain
Onofre, Aurora Pujol, M.D.
Ministerio de Sanidad, Servicios Sociales e Igualdad; Fundacion Hesperia

Completed

A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants

Adrenomyeloneuropathy

Spain
Parc de Salut Mar

Completed

"Using Epigallocatechin Gallate (EGCG) and Cognitive Training to Modulate Cognitive Performance in Patients With Fragile X Syndrome" (TESFX)

Fragile X Syndrome

Spain
Yolanda de Diego Otero

Completed

Safety and Efficacy Study of Antioxidants for the Treatment of the Fragile X Syndrome (SXF-TRA152)

Fragile X Syndrome

Spain
Parc de Salut Mar
Hospital Infantil Universitario Niño Jesús, Madrid, Spain; Hospital Universitario... and other collaborators

Completed

Pediatric Exploratory Research Study of EGCG Use and Safety (PERSEUS) (PERSEUS)

Down Syndrome | Fragile X Syndrome

Spain
Minoryx Therapeutics, S.L.

Active, not recruiting

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients. (Advance)

Adrenoleukodystrophy

Spain, United States, Hungary, France, Germany, Italy, Netherlands, United Kingdom
Fundación Pública Andaluza para la Investigación...
Delos Clinical

Completed

Clinical Trial for Asses the Safety and Efficacy of Vitamin C and Vitamin E in Combination Versus Placebo for Treating Cognitive and Behavior Disorder in Children With Fragile X Syndrome (SXF2-8)

X Fragile Syndrome

Spain
EspeRare Foundation
Iqvia Pty Ltd; Pierre Fabre Medicament

Recruiting

Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia (EDELIFE)

X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)

United States, Spain, France, Germany, Italy, United Kingdom
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
Mitsubishi Tanabe Pharma America Inc.

Recruiting

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP (INSPIRE)

Erythropoietic Protoporphyria (EPP) | X-Linked Protoporphyria (XLP)

United States, Spain, France, Japan, United Kingdom, Italy, Australia, Poland, Bulgaria, Czechia, Netherlands
Novartis Pharmaceuticals

Terminated

Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome

Fragile X Syndrome

Germany, United States, Switzerland, Australia, Canada, Denmark, France, Italy, United Kingdom, Spain
Novartis Pharmaceuticals

Completed

Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

Fragile X Syndrome

United States, Switzerland, Germany, Australia, Canada, Denmark, France, Italy, Spain, United Kingdom
Novartis Pharmaceuticals

Terminated

Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)

Fragile X Syndrome

United States, Belgium, Australia, Israel, Switzerland, Germany, Denmark, Italy, Netherlands, Spain, Sweden, United Kingdom, France
Hoffmann-La Roche

Completed

A Study of RO4917523 in Patients With Fragile X Syndrome

Fragile X Syndrome

France, Spain, United States, Peru, Canada, United Kingdom, Chile, Argentina, Mexico, Sweden
Novartis Pharmaceuticals

Completed

Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome

Fragile X Syndrome

United States, Belgium, Australia, Israel, Switzerland, Germany, France, Canada, Denmark, Indonesia, Italy, Netherlands, Spain, Sweden, Turkey, United Kingdom
Jazz Pharmaceuticals
GW Pharmaceuticals

Terminated

A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome

Rett Syndrome | RTT

United States, Canada, Italy, Spain, United Kingdom, Australia
Jazz Pharmaceuticals

Terminated

Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome (ARCH)

Rett Syndrome | RTT

United States, Spain, Italy, United Kingdom
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
Sarepta Therapeutics, Inc.
Hansa Biopharma AB

Active, not recruiting

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)

Duchenne Muscular Dystrophy

Spain
Rocket Pharmaceuticals Inc.

Completed

Natural History of Danon Disease

Danon Disease

Ireland, Czechia, France, Italy, Spain
University of Malaga

Completed

Effectiveness of a Multimodal Physiotherapy Program With Virtual Reality Glasses in Duchenne and Becker. (RVDuchenne)

Muscular Dystrophy, Duchenne and Becker Types

Spain
Universidad de Almeria

Completed

Physical Therapy Treatment on Children and Adolescents With Neurological Pathologies

Neurologic Disorder | Pediatric Disorder | Duchenne | Sensorimotor Disorder Nos

Spain
Baxalta now part of Shire

Terminated

A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects

Hemophilia B

Turkey, Spain
University of Malaga

Not yet recruiting

Effectiveness of 5-week Digital Respiratory Practice in a Group of Children With Duchenne Muscular Dystrophy and Becker Muscular Dystrophy. (DMDrespy2024)

Muscular Dystrophy | Duchenne Muscular Dystrophy | Becker Muscular Dystrophy

Spain
BioMarin Pharmaceutical

Recruiting

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Spain, United Kingdom
Barcelona Macula Foundation

Recruiting

Structural and Functional Characterization of Rare Ocular Diseases (RADIS)

Retinitis Pigmentosa | Choroideremia | Stargardt Disease 1 | Best Disease | Pattern Dystrophy of Macula

Spain
Hoffmann-La Roche
Sarepta Therapeutics, Inc.

Recruiting

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (ENVOL)

Duchenne Muscular Dystrophy

Spain, Germany
Shire

Completed

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

Hunter Syndrome

United States, Spain, Canada, United Kingdom, Mexico, Australia, France
Shire

Completed

Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)

Hunter Syndrome | Mucopolysaccharidosis (MPS)

United States, Argentina, Mexico, Spain, United Kingdom
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
JCR Pharmaceuticals Co., Ltd.

Recruiting

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

Mucopolysaccharidosis II

United States, Germany, Spain, France, Brazil, Argentina, Turkey, United Kingdom, Poland, Italy
EspeRare Foundation

Completed

Rimeporide in Patients With Duchenne Muscular Dystrophy (RIM4DMD)

Muscular Dystrophy, Duchenne

Italy, France, Spain, United Kingdom
Novo Nordisk A/S

Completed

Safety and Mode of Action of a Single Dose and Multiple Doses of Long Acting Activated Recombinant Human Factor VII in Patients With Haemophilia A and B

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Spain
Janssen Research & Development, LLC
Janssen Research & Development, LLC

Active, not recruiting

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

X-Linked Retinitis Pigmentosa

United States, Canada, Israel, United Kingdom, Spain, Denmark, France, Belgium, Italy, Netherlands, Switzerland
Takeda

Available

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Hunter Syndrome

United States, Australia, Mexico, Spain, United Kingdom
Novo Nordisk A/S

Completed

Project to Update the Study of Congenital Haemophilia in Spain (PUCHS)

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Spain
Janssen Research & Development, LLC
Janssen Research & Development, LLC

Active, not recruiting

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

X-Linked Retinitis Pigmentosa

Belgium, Canada, United States, Israel, United Kingdom, Spain, Denmark, France, Italy, Netherlands, Switzerland
Shire

Completed

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment (AIM-IT)

Hunter Syndrome

Spain, United States, Canada, United Kingdom, France, Australia, Mexico
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.

Completed

Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)

Duchenne Muscular Dystrophy

Spain, United States, Russian Federation, Italy, China, Turkey
Denali Therapeutics Inc.

Recruiting

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) (COMPASS)

Mucopolysaccharidosis II

United States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Colombia, Brazil
Novo Nordisk A/S

Completed

A Trial Investigating the Pharmacokinetics and Pharmacodynamics of rFVIIa in Patients With Haemophilia A or B With or Without Inhibitors

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

Spain
Great Ormond Street Hospital for Children NHS Foundation...
Catholic University of the Sacred Heart; Università degli Studi di Ferrara; Universidad... and other collaborators

Recruiting

Brain Involvement in Dystrophinopathies Part 1

Duchenne Muscular Dystrophy | Becker Muscular Dystrophy

Denmark, France, Italy, Netherlands, Spain, United Kingdom
University Hospital, Basel, Switzerland

Completed

Tamoxifen in Duchenne Muscular Dystrophy (TAMDMD)

Duchenne Muscular Dystrophy

France, Spain, Germany, Netherlands, Switzerland, United Kingdom
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
CSL Behring

Completed

Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B

Hemophilia B

Spain, Italy, France, Germany, Austria, Israel
CSL Behring

Completed

Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy

Common Variable Immunodeficiency | X-linked Agammaglobulinemia | Autosomal Recessive Agammaglobulinemia

Germany, United Kingdom, France, Italy, Poland, Romania, Spain, Sweden, Switzerland

Clinical Trials on X-linked Adrenoleukodystrophy in Spain

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