Clinical Trials on Mental Retardation, X-linked 102 in Brazil

Total 76 results

Faculdades Metropolitanas Unidas
University of Sao Paulo

Completed

Functional Abilities in Rett Syndrome

Rett Syndrome

Brazil
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-141 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II

Brazil
Takeda

Completed

A Study to Improve the Awareness of Mucopolysaccharidosis Type II in Brazil

Mucopolysaccharidosis (MPS)

Brazil
JCR Pharmaceuticals Co., Ltd.

Active, not recruiting

An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II

Brazil
bluebird bio

Active, not recruiting

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

Cerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)

United States, Argentina, Australia, Brazil, France, Germany, Netherlands, United Kingdom, Italy
REGENXBIO Inc.

Completed

Mucopolysaccharidosis Type II Natural History

Mucopolysaccharidosis II

Brazil, United States, United Kingdom
Shire
Covance; PRA Health Sciences; PharmaNet

Completed

Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

Mucopolysaccharidosis II | Hunter Syndrome | MPS II

Brazil, Poland, Taiwan
REGENXBIO Inc.

Active, not recruiting

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Mucopolysaccharidosis Type II (MPS II)

United States, Brazil
Shire

Completed

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

Hunter Syndrome

United Kingdom, United States, Brazil
Shire

Completed

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

Mucopolysaccharidosis II

United States, United Kingdom, Brazil, Germany
Minoryx Therapeutics, S.L.

Recruiting

A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy (CALYX)

Cerebral Adrenoleukodystrophy (cALD)

United States, Brazil, Argentina
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
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
Shire

Completed

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

Hunter Syndrome | Mucopolysaccharidosis II (MPS II)

United States, France, United Kingdom, Germany, Sweden, Spain, Brazil, Canada, Italy, Romania
NightstaRx Ltd, a Biogen Company

Enrolling by invitation

Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa (SOLSTICE)

Choroideremia | X-Linked Retinitis Pigmentosa

United States, United Kingdom, Netherlands, Germany, France, Brazil, Canada, Denmark, Finland
The Hospital for Sick Children

Completed

Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World

Hemophilia A | Hemophilia B

Canada, Brazil
University of Sao Paulo

Completed

Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Brazil
University of Sao Paulo

Completed

Heart Rate Variability in Duchenne Muscular Dystrophy During Computer Task

Duchenne Muscular Dystrophy

Brazil
Monica Levy Andersen

Completed

Mother-caregivers of Children With Duchenne Muscular Dystrophy (DMDK)

Other Diseases or Conditions

Brazil
University of Brasilia

Completed

Resistance Training Program, Labor Inclusion, Intellectual Disability

Muscle Weakness | Body Composition, Beneficial | Mental Retardation

Brazil
NightstaRx Ltd, a Biogen Company

Completed

Natural History of the Progression of Choroideremia Study (NIGHT)

Choroideremia

Finland, United States, Canada, Denmark, Netherlands, Germany, United Kingdom, Brazil, France
BioMarin Pharmaceutical

Terminated

A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

Duchenne Muscular Dystrophy

United States, Netherlands, Belgium, Germany, Argentina, Brazil, France, Italy, Sweden, Turkey
University of Sao Paulo General Hospital
Pfizer

Recruiting

Substudy (NCT04456582): Noninvasive Assessment of Myocardial Stiffness by 2D-SWE Ultrasound Technique (Two-dimensional Shear Wave Elastography) in Patients With Amyloidosis and Fabry Disease. (FABRY)

Amyloidosis | Fabry Disease

Brazil
Amicus Therapeutics

Completed

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

Fabry Disease

Australia, Brazil
University of Sao Paulo General Hospital

Completed

Viscosupplementation in Patients With Hemophilic Arthropathy

Hemophilia A | Hemophilia B | Osteoarthritis | Hemarthrosis

Brazil
Novo Nordisk A/S

Completed

To Quantify the Range of Main Psychosocial Factors Affecting Patients and Caregivers in Their Daily Lives (HERO)

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

Brazil
Medexus Pharma, Inc.

Completed

Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B

Hemophilia B

Turkey, Brazil, Georgia, Moldova, Republic of, South Africa, Ukraine
AVROBIO

Terminated

Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201

Fabry Disease

Australia, Brazil
University of Sao Paulo

Completed

Clinical Trial of Fat Grafts Supplemented With Adipose-derived Regenerative Cells

Craniofacial Microsomia

Brazil
Hospital Sirio-Libanes

Completed

Fat Grafts With Adipose-derived Regenerative Cells for Soft Tissue Reconstruction in Children

Craniofacial Microsomia

Brazil
Baxalta now part of Shire

Completed

IMMUNINE Pre-Treatment Study

Hemophilia B

Colombia, Bulgaria, Chile, Poland, Romania, Argentina, Brazil, Czechia, Russian Federation, Ukraine
Baxalta now part of Shire

Completed

Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients

Hemophilia B

Poland, United Kingdom, Spain, Bulgaria, Romania, Japan, Russian Federation, Brazil, Chile, Sweden, Colombia, Ukraine, Argentina, Czechia
Amicus Therapeutics

Completed

A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

Fabry Disease

Japan, Taiwan, Turkey, United States, Brazil
Amicus Therapeutics

Completed

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease

Fabry Disease

United States, Australia, France, United Kingdom, Brazil, Canada
Federal University of Rio Grande do Sul
Hospital de Clinicas de Porto Alegre; Federal University of Health Science...

Completed

Effects of Nordic Walking in Parkinson Disease Patients (Walking)

Parkinson Disease | Parkinson Disease 10

Brazil
Baxalta now part of Shire

Completed

Efficacy and Safety Study of Prophylactic Versus On-Demand Treatment With Feiba NF in Subjects With Hemophilia A or B and a High Titer Inhibitor

Hemophilia A | Hemophilia B | Hemophilia A or B With Inhibitors

Poland, United States, Bulgaria, Romania, Japan, Russian Federation, New Zealand, Croatia, Ukraine, Brazil
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
Baxalta now part of Shire

Completed

BAX 326 (rFIX) Continuation Study

Hemophilia B

Taiwan, Colombia, Argentina, Brazil, Bulgaria, Chile, Czechia, India, Ireland, Italy, Japan, Poland, Romania, Russian Federation, Sweden, Ukraine, United Kingdom
AVROBIO

Terminated

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

Fabry Disease

United States, Australia, Brazil
Amicus Therapeutics

Terminated

Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study

Fabry Disease

United Kingdom, United States, Australia, Brazil, France
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
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
GlaxoSmithKline

Completed

A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD114044)

Muscular Dystrophies

France, Denmark, Germany, Belgium, Spain, Turkey, Korea, Republic of, Brazil, Netherlands, Italy, Chile, Japan, Canada, Hungary, Russian Federation, Czechia, Poland, Norway, Argentina, Taiwan
ApcinteX Ltd
Centessa Pharmaceuticals plc

Recruiting

Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)

Hemophilia A | Hemophilia B

Spain, Taiwan, United Kingdom, Italy, Germany, United States, South Africa, Australia, Poland, Armenia, France, India, Canada, Brazil, Turkey, Egypt
Genzyme, a Sanofi Company

Completed

A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (FIELD)

Fabry Disease

United States, Poland, Netherlands, Canada, Argentina, United Kingdom, Norway, Brazil, Czech Republic
Pfizer

Active, not recruiting

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

Hemophilia A | Hemophilia B

United States, Canada, Spain, Australia, Korea, Republic of, Taiwan, Brazil, Italy, Japan, Turkey, Germany, United Kingdom, Saudi Arabia, France, Sweden, Belgium, Greece, Israel
GlaxoSmithKline

Terminated

Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

Muscular Dystrophies

France, Bulgaria, Denmark, Germany, Belgium, Canada, Spain, Turkey, United Kingdom, Korea, Republic of, Brazil, Netherlands, Italy, Japan, Czech Republic, Chile, Israel, Hungary, Russian Federation, Australia, Poland, Norway, Argentina and more
Pfizer

Active, not recruiting

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2)

Hemophilia B

Germany, United Kingdom, Canada, Australia, Taiwan, Japan, France, Turkey, Korea, Republic of, United States, Brazil, Greece, Saudi Arabia, Sweden
Pfizer

Recruiting

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B (BASIS KIDS)

Hemophilia A | Hemophilia B

Denmark, Taiwan, Canada, Turkey, Spain, China, United Kingdom, South Africa, Saudi Arabia, Israel, Korea, Republic of, Austria, Germany, Japan, Australia, Italy, India, Slovakia, France, United States, Brazil, Argentina
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B

Severe Hemophilia B

Sweden, United States, France, Italy, Russian Federation, United Kingdom, Germany, China, Poland, Japan, Australia, Brazil, Canada, India, South Africa, Hong Kong, Belgium

Clinical Trials on Mental Retardation, X-linked 102 in Brazil

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