Clinical Trials on Hunter Syndrome (MPS II)

Total 532 results

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Withdrawn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
Benjamin Tomlinson

Recruiting

5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies

Myelodysplastic Syndromes | MDS/MPN Crossover Syndromes

United States
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
Michael Savona
Incyte Corporation; Astex Pharmaceuticals, Inc.; Theradex

Recruiting

Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes Overlap Syndromes (ABNL-MARRO) (ABNL-MARRO)

MDS/MPN

United States
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
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
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
Duke University

Recruiting

MPS II Immunophenotyping

Mucopolysaccharidosis II | Hunter Syndrome | MPS II

United States
CENTOGENE GmbH Rostock

Terminated

Biomarkers for Hunter Syndrome (BioHunter)

Mucopolysaccharidosis II | Hunter Syndrome | Hunter's Syndrome, Mild Form | Hunter's Canal Syndrome

Mexico
Stanford University
National Institutes of Health (NIH)

Completed

Non-Myeloablative Allogeneic Transplant for Myelodysplastic Syndromes and Myeloproliferative Disorders

Myelodysplastic Syndromes | Myeloproliferative Disorders | Myelodysplastic Syndromes (MDS) | Blood and Marrow Transplant (BMT) | Myeloproliferative Disorders (MPD)

United States
SCRI Development Innovations, LLC
Novartis Pharmaceuticals

Terminated

LBH589 in Refractory Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes (MDS)

United States
PersImmune, Inc
University of California, San Diego

Unknown

Collection of Samples From Patients With MDS

Myelodysplastic Syndromes(MDS)

United States
TJ Biopharma Co., Ltd.

Recruiting

A Phase 3 Clinical Study to Evaluate Lemzoparlimab for Injection in Combination With Azacitidine (AZA) Versus AZA Monotherapy in Treatment-naïve Patients With Higher-risk Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndromes(MDS)

China
National Heart, Lung, and Blood Institute (NHLBI)
National Cancer Institute (NCI)

Recruiting

The National Myelodysplastic Syndromes (MDS) Study (MDS)

Myelodysplastic Syndromes (MDS)

United States, Israel
AbbVie
Celgene; Genentech, Inc.

Completed

A Study Evaluating Venetoclax Alone and in Combination With Azacitidine in Participants With Relapsed/Refractory Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes (MDS)

United States, Australia, Germany
AbbVie
Genentech, Inc.

Active, not recruiting

A Study Evaluating Venetoclax in Combination With Azacitidine in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes (MDS)

United States, Australia, Canada, France, Germany, Italy, United Kingdom
The First Affiliated Hospital with Nanjing Medical...

Unknown

A Clinical Trial Evaluating the Efficacy of Ultra Low Dose of Decitabine in Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes (MDS)

China
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
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
Takeda
Takeda Development Center Americas, Inc.

Recruiting

A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)

Hunter Syndrome | Mucopolysaccharidosis (MPS)

United States
Dana-Farber Cancer Institute

Completed

The Myelodysplasia Transplantation-Associated Outcomes (MDS-TAO) Study

Myelodysplastic Syndromes (MDS)

United States
REGENXBIO Inc.

Active, not recruiting

RGX-111 Gene Therapy in Patients With MPS I

Hurler Syndrome | Hurler-Scheie Syndrome | Mucopolysaccharidosis Type I (MPS I)

United States, Brazil, Israel
Orchard Therapeutics

Recruiting

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT (HURCULES)

MPS-IH (Hurler Syndrome)

Netherlands, United States, Italy, United Kingdom
University of California, San Francisco
Duke University

Recruiting

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases (IUERT)

Wolman Disease | MPS IVA | Pompe Disease Infantile-Onset | Gaucher Disease, Type 2 | MPS VI | MPS I | Gaucher Disease, Type 3 | MPS II | Mps VII

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
Sumitomo Pharma America, Inc.

Terminated

A Phase 1b/2 Study of Alvocidib Plus Decitabine or Azacitidine in Patients With MDS

Myelodysplastic Syndromes (MDS)

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
Celgene

Recruiting

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Beta-thalassemia | Myelodysplastic Syndromes (MDS) | Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis

United States, Japan, Israel, United Kingdom, Turkey, Greece, Netherlands, France, Australia, Belgium, Bulgaria, Canada, Germany, Italy, Malaysia, Spain, Sweden, Taiwan, Thailand, Tunisia, China, Lebanon
University Hospital, Ghent

Terminated

Nitrous Oxide - Sevoflurane-Remifentanil Interaction

ASA Class I/II Patients Scheduled for Surgery Under General Anesthesia

Belgium
Institut fuer anwendungsorientierte Forschung und...
University Medical Center Goettingen

Completed

Screening and Genetic Monitoring of Patients With Myelodysplastic Syndromes (MDS) Under Different Treatment Modalities by Cytogenetic Analyses of Circulating CD34+Cells

Myelodysplastic Syndromes (MDS) | Chromosmal Aberrations | Karyotype Evolution | Rare Abnormalities | Cytogenetic Monitoring

Germany
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
Medical University of Vienna

Unknown

Surgical Treatment Of Complex Regional Pain Syndrome Type II (CRPS II)

CRPS Type II

Austria
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
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
Sidney Kimmel Cancer Center at Thomas Jefferson...
Celgene Corporation

Terminated

A Dose Range Finding Study of Lenalidomide in Non-5q Chromosome Deletion in Low and Intermediate Risk Myelodysplastic Syndrome (MDS) Patients

Myelodysplastic Syndrome | MDS | Low to Intermediate-1 MDS | Non-deletion 5q

United States
Prof. Janet Dunn
King's College Hospital NHS Trust; University of Manchester; University of Birmingham and other collaborators

Recruiting

Repurposed Drugs to Improve Haematological Responses in Myelodysplastic Syndromes (REPAIR-MDS)

Myelodysplastic Syndromes (MDS)

United Kingdom
Radboud University Medical Center

Recruiting

A Prospective, Multicentre European Registry for Newly Diagnosed Patients With Myelodysplastic Syndromes (EUMDS)

Myelodysplastic Syndromes (MDS)

Denmark, Switzerland, Serbia, Austria, Croatia, Czechia, France, Germany, Greece, Israel, Italy, Netherlands, Poland, Portugal, Romania, Spain, Sweden, United Kingdom
Takeda

Withdrawn

A Study of Pevonedistat Combined With Decitabine and Cedazuridine in Adults With Higher-risk Myelodysplastic Syndromes (PEVOBINE)

Myelodysplastic Syndromes (MDS)
Masonic Cancer Center, University of Minnesota

Terminated

Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

Hurler Syndrome | Mucopolysaccharidosis Type IH | MPS I

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
Dilşad Sindel

Completed

Investigation of the Effectiveness of Muscle Inhibition and Space Correction Techniques of Kinesiotaping Method in Female Patients With Myofascial Pain Syndrome Related to Upper Trapezius Active Trigger Points

Female Patients With Myofascial Pain Syndrome (MPS) Related to Upper Trapezius Active Trigger Points (TP)
Sunnybrook Health Sciences Centre
Princess Margaret Hospital, Canada; Juravinski Cancer Center

Unknown

Red Blood Cell Transfusion Thresholds and QOL in MDS (EnhanceRBC)

Quality of Life | Myelodysplastic Syndromes (MDS) | Red Blood Cell (RBC) Transfusions

Canada
Novartis Pharmaceuticals

Recruiting

STIMULUS MDS-US : Sabatolimab Added to HMA in Higher Risk MDS

Myelodysplastic Syndrome (MDS)

United States
Otsuka Pharmaceutical Co., Ltd.

Recruiting

A Phase 1 Trial of ASTX030 in Patients With Myelodysplastic Syndrome

Myelodysplastic Syndrome (MDS)

Japan
Fondazione Italiana Sindromi Mielodisplastiche-ETS

Recruiting

Personalized Medicine Program on Myelodysplastic Syndromes: Characterization of the Patient's Genome for Clinical Decision Making and Systematic Collection of Real World Data to Improve Quality of Health Care (FISiM-NGS-MDS)

MDS (Myelodysplastic Syndrome)

Italy
Fujifilm Pharmaceuticals U.S.A., Inc.

Withdrawn

A Study of FF-10501-01 in Combination With Azacitidine in Patients With Myelodysplastic Syndrome

Myelodysplastic Syndrome (MDS)
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Safety of ON 01910.Na in Patients With Myelodysplasia

Myelodysplastic Syndrome (MDS)

United States
Telik

Terminated

Study of (Telintra®) in Non-Del(5q) Myelodysplastic Syndrome

Myelodysplastic Syndrome (MDS)

United States
Oncology Specialties, Alabama

Terminated

Phase II Research Study of Arsenic Trioxide (Trisenox) in Patients With Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndrome (MDS)

United States
AbbVie
Genentech, Inc.

Active, not recruiting

Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome (Verona)

Myelodysplastic Syndrome (MDS)

United States, Australia, Austria, Belgium, Brazil, Canada, China, Czechia, France, Germany, Hungary, Israel, Italy, Japan, Korea, Republic of, Netherlands, Poland, Puerto Rico, Russian Federation, Spain, Taiwan, Turkey, United Kingdom

Clinical Trials on Hunter Syndrome (MPS II)

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