Clinical Trials on Sanfilippo Syndrome Type A (MPS IIIA)

Total 350 results

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
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
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
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

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
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
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
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
Shire

Completed

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

Sanfilippo Syndrome Type A

United States
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
Samsung Medical Center

Not yet recruiting

Natural History Observational Study of MPS IIIa in SMC

Sanfilippo Syndrome A
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
LYSOGENE

Completed

Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

Mucopolysaccharidosis Type III A | Sanfilippo Disease Type A

France
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
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
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
Hospital de Clinicas de Porto Alegre
The Isaac Foundation

Active, not recruiting

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

Mucopolysaccharidoses | Mucopolysaccharidosis VI | Morquio A Syndrome | Mucopolysaccharidosis IV A | MPS IV A | MPS VI | MPS - Mucopolysaccharidosis | Morquio Syndrome A | Morquio Syndrome

Brazil
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
Denali Therapeutics Inc.

Recruiting

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

Mucopolysaccharidosis Type IIIA

United States
UniQure Biopharma B.V.
Institut Pasteur; Venn Life Sciences

Completed

Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

Sanfilippo Syndrome B

France
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
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
Gwynn Long, M.D.
ADC Therapeutics S.A.

Terminated

ADCT-301 in Patients With R/R AML, MDS, or MDS/MPN

Acute Myeloid Leukemia (AML) | Myelodysplastic Syndrome (MDS) | Myeloproliferative Neoplasm (MDS/MPN)

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
Roswell Park Cancer Institute
Merck Sharp & Dohme LLC

Active, not recruiting

Sorafenib Tosylate and Pembrolizumab in Treating Patients With Advanced or Metastatic Liver Cancer

Advanced Adult Hepatocellular Carcinoma | Child-Pugh Class A | Stage III Hepatocellular Carcinoma | Stage IIIA Hepatocellular Carcinoma | Stage IIIB Hepatocellular Carcinoma | Stage IIIC Hepatocellular Carcinoma | Stage IV Hepatocellular Carcinoma | Stage IVA Hepatocellular Carcinoma | Stage IVB Hepatocellular...

United States
Cyclacel Pharmaceuticals, Inc.

Suspended

A Study to Investigate Fadraciclib (CYC065), in Subjects With Leukemia or Myelodysplastic Syndrome (MDS)

Leukemia | Myelodysplastic Syndrome(MDS)

United States
Jacqueline Garcia, MD
National Institutes of Health (NIH)

Recruiting

Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN

Acute Myeloid Leukemia (AML) | Chronic Myelomonocytic Leukemia (CMML) | Myelodysplastic Syndrome (MDS) | Hematopoietic Stem Cell Transplant | MDS/Myeloproliferative Neoplasm-unclassifiable (MDS/MPN-unclassifiable)

United States
Greenwood Genetic Center
Shriners Hospitals for Children; BioMarin Pharmaceutical

Completed

Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome

MPS IVA | Maroteaux Lamy Syndrome | MPS VI | Morquio Syndrome A

United States
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

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
Ohio State University Comprehensive Cancer Center
Varian Medical Systems

Completed

Stereotactic Body Radiation Therapy and Transarterial Chemoembolization in Treating Patients With Liver Cancer That Cannot Be Removed by Surgery

Child-Pugh Class A | Stage IIIA Hepatocellular Carcinoma | Stage IIIB Hepatocellular Carcinoma | Stage IIIC Hepatocellular Carcinoma | Stage IVA Hepatocellular Carcinoma | Stage IVB Hepatocellular Carcinoma | Child-Pugh Class B

United States
TJ Biopharma Co., Ltd.

Completed

A Study to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of TJ011133 as Monotherapy and in Combination With Azacitidine (AZA) in Patients With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

Acute Myeloid Leukemia (AML) | Myelodysplastic Syndromes(MDS)

China
Masonic Cancer Center, University of Minnesota

Withdrawn

MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT

Mucopolysaccharidosis Type IH | Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome) | MPS IH, Hurler Syndrome
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
Stanford University
Celgene Corporation

Terminated

Azacitidine + Lenalidomide Combo in the Elderly With Previously Treated AML & High-Risk MDS

Leukemia | Myelodysplastic Syndromes (MDS) | Acute Myeloid Leukemia (AML)

United States
Alliance for Clinical Trials in Oncology
National Cancer Institute (NCI)

Completed

Glutathione in Preventing Peripheral Neuropathy Caused by Paclitaxel and Carboplatin in Patients With Ovarian Cancer, Fallopian Tube Cancer, and/or Primary Peritoneal Cancer

Pain | Neuropathy | Stage IIIA Fallopian Tube Cancer | Stage IIIA Ovarian Cancer | Stage IIIA Primary Peritoneal Cancer | Stage IIIB Fallopian Tube Cancer | Stage IIIB Ovarian Cancer | Stage IIIB Primary Peritoneal Cancer | Stage IIIC Fallopian Tube Cancer | Stage IIIC Ovarian Cancer | Stage IIIC Primary... and other conditions

United States
JCR Pharmaceuticals Co., Ltd.

Recruiting

Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

Mucopolysaccharidosis III-A

Germany
Lundquist Institute for Biomedical Innovation at...

Not yet recruiting

Study of Cannabidiol in Sanfilippo Syndrome

Mucopolysaccharidosis III | Sanfilippo Syndrome

United States
Peter MacCallum Cancer Centre, Australia
GlaxoSmithKline; Celgene Corporation

Completed

A Single Arm Pilot Study of Azacitidine in Myelodysplastic Syndromes (MDS) / Acute Myeloid Leukaemia (AML), With Eltrombopag Support for Thrombocytopenia (Aza-E)

Myelodysplastic Syndromes (MDS) | Acute Myeloid Leukaemia (AML)

Australia
Kiadis Pharma
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaborators

Withdrawn

Haplo-identical Natural Killer (NK) Cells to Prevent Post-Transplant Relapse in AML and MDS (NK-REALM)

Myelodysplastic Syndromes (MDS) | Acute Myeloid Leukemia (AML)

United States
BioMarin Pharmaceutical

Terminated

A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Morquio A Syndrome | MPS IV A | Mucopolysaccharidosis IVA

United Kingdom
Ascentage Pharma Group Inc.
Suzhou Yasheng Pharmaceutical Co., Ltd.

Recruiting

A Phase Ib Study of APG-115 Single Agent or in Combination With Azacitidine or Cytarabine in Patients With AML and MDS.

Myelodysplastic Syndromes (MDS) | Acute Myeloid Leukemia (AML)

China
BioMarin Pharmaceutical
ICON plc

Completed

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) (MARS)

Mucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVA

United States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
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
Novartis Pharmaceuticals

Active, not recruiting

HDM201 in Combination With MBG453 or Venetoclax in Patients With Acute Myeloid Leukemia (AML) or High-risk Myelodysplastic Syndrome (MDS)

Acute Myeloid Leukemia (AML) | High-risk Myelodysplastic Syndrome (MDS)

Spain, Singapore, Australia, Germany, Italy, Finland, United States
University of Ulm

Terminated

Trial of Intensive Chemotherapy With or Without Volasertib in Patients With Newly Diagnosed High-Risk Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML)

Acute Myeloid Leukemia (AML) | High-risk Myelodysplastic Syndrome (MDS)

Germany
Astex Pharmaceuticals, Inc.

Completed

SGI-110 in Patients With Myelodysplastic Syndromes (MDS) or Acute Myelogenous Leukemia (AML)

AML | MDS | CMML

United States, Canada
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
BioMarin Pharmaceutical

Terminated

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

Morquio A Syndrome | MPS IV A | Mucopolysaccharidosis IVA

France, United Kingdom, Taiwan, United States, Argentina, Netherlands, Canada, Brazil, Germany, Italy

Clinical Trials on Sanfilippo Syndrome Type A (MPS IIIA)

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