Search clinical trials for: Sanfilippo A

Total 495103 results

Lundquist Institute for Biomedical Innovation at...

Not yet recruiting

Study of Cannabidiol in Sanfilippo Syndrome

Mucopolysaccharidosis III | Sanfilippo Syndrome

United States
Denali Therapeutics Inc.

Recruiting

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Mucopolysaccharidosis Type IIIA

United States
Phoenix Nest
Aparito Ltd.; Vaincres Les Maladies Lysosomales; Association Sanfilippo Sud

Not yet recruiting

Natural History Study of Participants With Sanfilippo Syndrome Type IIIC (MPSIIIC)

Sanfilippo Syndrome Type C

United States, France
Lysosomal and Rare Disorders Research and Treatment...
Team Sanfilippo

Recruiting

The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)

Sanfilippo Syndrome | MPS3

United States
Sanguine Biosciences
Denali Therapeutics Inc.

Recruiting

A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)

Mucopolysaccharidosis III-A

United States
Samsung Medical Center

Not yet recruiting

Natural History Observational Study of MPS IIIa in SMC

Sanfilippo Syndrome A
Mario Negri Institute for Pharmacological Research
Fondazione IRCCS Istituto Nazionale dei Tumori, Milano; Humanitas Hospital,...

Recruiting

Study on Trabectedin in Combination With Pioglitazone in Patients Myxoid Liposarcomas With Stable Disease After T Alone. (TRABEPIO)

Liposarcoma, Myxoid | Liposarcoma, Dedifferentiated | Liposarcoma, Round Cell

Italy
Humanitas Clinical and Research Center

Recruiting

Diastolic dYsfunctioN AssessMent in critICally Ill Patients (DYNAMIC)

Critical Illness | Mechanical Ventilation Complication | Weaning Failure | Diastolic Heart Failure | Diastolic Dysfunction

Italy
Italian Sarcoma Group
PharmaMar

Recruiting

Trial in Patients With Metastatic or Locally Advanced Leiomyosarcoma (ISG-ARTICLE)

Soft Tissue Sarcoma | Leiomyosarcoma of Ovary

Italy
Washington University School of Medicine
Massachusetts General Hospital; Ontario Clinical Oncology Group (OCOG); St. Luke...

Recruiting

Chronic Venous Thrombosis: Relief With Adjunctive Catheter-Directed Therapy (The C-TRACT Trial) (C-TRACT)

Deep Vein Thrombosis | Post Thrombotic Syndrome | Venous Insufficiency | Venous Leg Ulcer | Venous Stasis | Venous Reflux

United States
Joanne Kurtzberg, MD
The Marcus Foundation

Recruiting

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (DUOC-01)

Mucopolysaccharidosis II | Brain Diseases, Metabolic, Inborn | Adrenoleukodystrophy | Batten Disease | Leukodystrophy, Metachromatic | Leukodystrophy, Globoid Cell | Alpha-Mannosidosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | Neimann Pick Disease | Sanfilippo Mucopolysaccharidoses

United States
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc

Enrolling by invitation

A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102

Mucopolysaccharidosis III-A

United States, Australia, Spain
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc

Active, not recruiting

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Mucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo A

United States, Australia, Spain
Phoenix Nest
National Institute of Neurological Disorders and Stroke (NINDS)

Enrolling by invitation

A Natural History Study of Sanfilippo Syndrome Type D (MPSIIID)

Sanfilippo Syndrome Type D

United States
Lundquist Institute for Biomedical Innovation at...
Swedish Orphan Biovitrum; Cure Sanfilippo Foundation

Completed

Open-label Study of Anakinra in MPS III

Mucopolysaccharidosis III

United States
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc

Terminated

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

Mucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo A

United States, Spain, Australia
Albert Einstein College of Medicine
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program (ScreenPlus)

Mucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditions

United States
Talaris Therapeutics Inc.
Duke University

Terminated

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Niemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...

United States
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Sanfilippo Type A-B-C-D Disease (BioSanfilippo) (BioSanfilippo)

Developmental Delay | Mental Retardation

Germany, India, Sri Lanka, Egypt
Allievex Corporation

Active, not recruiting

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

Mucopolysaccharidosis Type IIIB | MPS III B

Germany, United States, Turkey, United Kingdom, Colombia, Spain, Taiwan
Allievex Corporation

Active, not recruiting

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Mucopolysaccharidosis Type IIIB

United States, Germany, Spain, Argentina, Australia, Brazil, Colombia, Taiwan, Turkey
Abeona Therapeutics, Inc

Terminated

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

Mucopolysaccharidosis III-B

United States, Germany, France
Abeona Therapeutics, Inc

Terminated

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB (MPSIIIB)

Mucopolysaccharidosis Type 3 B

United States, Germany, France, Spain
Swedish Orphan Biovitrum

Completed

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

Sanfilippo Syndrome Type A (MPS IIIA)

United States, Turkey
Swedish Orphan Biovitrum

Completed

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

Sanfilippo Syndrome Type A (MPS IIIA)

United States, Germany, Turkey
LYSOGENE

Active, not recruiting

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (AAVance)

Mucopolysaccharidosis Type IIIA

Germany, United States, United Kingdom, France, Netherlands
Shire

Terminated

Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Sanfilippo Syndrome

Netherlands, United Kingdom
Shire

Completed

Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients

Mucopolysaccharidosis (MPS)

United Kingdom, Netherlands
Shire

Completed

A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Sanfilippo Syndrome Type A

United States
Shire

Terminated

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

Sanfilippo Syndrome | Mucopolysaccharidosis (MPS)

United States, United Kingdom, Spain, Germany, France, Italy, Netherlands
Shire

Completed

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

Sanfilippo Syndrome

United States, Spain, United Kingdom, Argentina, France, Germany, Italy, Netherlands
Shire

Completed

Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

Sanfilippo Syndrome Type B

United States, United Kingdom, Germany
Allievex Corporation

Completed

A Treatment Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Mucopolysaccharidosis Type IIIB | MPS III B

United States, Taiwan, Spain, Colombia, Germany, United Kingdom, Turkey
Allievex Corporation

Completed

A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Mucopolysaccharidosis Type IIIB | Mucopolysaccharidosis Type 3 B | MPS III B | MPS 3 B

United States, Spain, Turkey, Taiwan, Australia, Colombia, Germany, United Kingdom
UniQure Biopharma B.V.
Institut Pasteur; Venn Life Sciences

Completed

Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

Sanfilippo Syndrome B

France
University of Minnesota
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

Neurobehavioral Phenotypes in MPS III

Hurler Syndrome | Sanfilippo Syndrome Type A | Sanfilippo Syndrome Type B

United States
University of Chicago
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Krabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)

United States
Masonic Cancer Center, University of Minnesota

Completed

HSCT for High Risk Inherited Inborn Errors

Wolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Sanfilippo Syndrome | GM1 Gangliosidosis | Globoid Cell Leukodystrophy | Tay Sachs Disease | Sandhoffs Disease | I-Cell Disease

United States
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
Alexion Pharmaceuticals

Completed

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

Mucopolysaccharidosis IIIB

United States, United Kingdom, Spain
Alexion Pharmaceuticals

Terminated

A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously (CL01-T)

Mucopolysaccharidosis III, Type B (MPS IIIB) | Sanfilippo B

United Kingdom
Masonic Cancer Center, University of Minnesota

Completed

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

Lysosomal Storage Disease | Peroxisomal Disorder

United States
LYSOGENE

Completed

Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

Mucopolysaccharidosis Type III A | Sanfilippo Disease Type A

France
Alexion Pharmaceuticals

Completed

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

MPS IIIB (Sanfilippo Syndrome)

Spain, Netherlands, United Kingdom, United States, Brazil
Alexion Pharmaceuticals

Completed

Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging

MPS IIIB (Sanfilippo B Syndrome)

United Kingdom
Nationwide Children's Hospital
Sanfilippo Children's Research Foundation; The Sanfilippo Research Foundation; The Children's Medical Research Foundation

Completed

Natural History Studies of Mucopolysaccharidosis III

Mucopolysaccharidosis Type IIIA | Mucopolysaccharidosis Type IIIB

United States
Duke University

Completed

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

Hemoglobinopathies | Thalassemia | Lysosomal Storage Disease | Immunodeficiencies | Inborn Errors of Metabolism | Sickle Cell | Non Malignant Disorders | Congenital Marrow Failures

United States
Aldagen

Terminated

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases (UCBT-002)

Mucopolysaccharidosis | Inborn Errors of Metabolism | Lysosomal Storage Disorders | Inherited Metabolic Diseases | Peroxisomal Storage Diseases

United States
LYSOGENE

Completed

Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

Mucopolysaccharidosis Type III A | Sanfilippo Disease Type A

France
Tanta University

Not yet recruiting

External Oblique Intercostal Plane Block Versus Thoracic Paravertebral Block for Post Thoracotomy Pain

Post Thoracotomy Pain | Thoracic Paravertebral Block | External Oblique Intercostal Plane Block

Egypt

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