Clinical Trials on MPS IIIB (Sanfilippo Syndrome)

Total 498 results

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Withdrawn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)
Mayo Clinic
National Cancer Institute (NCI)

Completed

Platelet Rich Plasma for the Treatment of Genitourinary Syndrome of Menopause in Patients With Stage 0-III Breast Cancer

Anatomic Stage I Breast Cancer AJCC v8 | Anatomic Stage IA Breast Cancer AJCC v8 | Anatomic Stage IB Breast Cancer AJCC v8 | Anatomic Stage II Breast Cancer AJCC v8 | Anatomic Stage IIA Breast Cancer AJCC v8 | Anatomic Stage IIB Breast Cancer AJCC v8 | Anatomic Stage III Breast Cancer AJCC v8 | Anatomic... and other conditions

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
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
Novartis Pharmaceuticals

Active, not recruiting

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

Myelodysplastic Syndrome (MDS)

United States
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, Israel, Italy, Japan, Korea, Republic of, Netherlands, Poland, Puerto Rico, Russian Federation, Spain, Taiwan, Turkey, United Kingdom, Hungary
Amphivena Therapeutics, Inc.

Completed

A Phase 1 Study of AMV564 in Patients With Intermediate or High-Risk Myelodysplastic Syndromes

Myelodysplastic Syndrome (MDS)

United States
Celgene

Completed

Study of Azacitidine to Evaluate Safety and Effectiveness for Chinese Patients With Higher Risk Myelodysplastic Syndrome

Myelodysplastic Syndrome (MDS)

China
Amgen

Completed

Darbepoetin Alfa MDS Companion Protocol

Myelodysplastic Syndrome (MDS)

Belgium
Novartis Pharmaceuticals

Terminated

LBH589 Alone or in Combination With Erythropoietin Stimulating Agents (ESA) in Patients With Low or Int-1 Risk Myelodysplastic Syndromes (MDS) (GEPARD)

Myelodysplastic Syndrome (MDS)

Germany
Telik

Completed

Phase 2 Study Comparing Two Dose Schedules of Telintra™ in Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndrome (MDS)

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
Case Comprehensive Cancer Center

Recruiting

CPX-351 Therapy for MDS After Hypomethylating Agent Failure

Myelodysplastic Syndrome (MDS)

United States
Novartis Pharmaceuticals

Terminated

A Study of Sabatolimab in Combination With Azacitidine and Venetoclax in High or Very High Risk MDS Participants (STIMULUS-MDS3)

Myelodysplastic Syndrome (MDS)

France, Spain, Belgium, Italy, Greece, Hungary, Germany
Weill Medical College of Cornell University
Novartis Pharmaceuticals

Completed

MRI Screening for Patients With Myelodysplastic Syndrome (MDS), Who Have Received Multiple Red Blood Cell Transfusions (T2*MRI)

Myelodysplastic Syndrome (MDS)

United States
Incyte Corporation

Terminated

Randomized Open-Label Study of INCB047986 in Subjects With Primary Myelodysplastic Syndrome (MDS)

MDS (Myelodysplastic Syndrome)

United States
Chinese Society of Hematology
Harbin Institute of Hematology & Oncology

Unknown

Myelodysplastic Syndrome--CDA-2 Hematological Improvement National Affirmation Study (MD-CHINA)

Myelodysplastic Syndrome (MDS)
Asociación Andaluza de Hematología y Hemoterapia

Completed

Study of Vidaza (Azacitidine) Versus Support Treatment in Patients With Low Risk Myelodysplastic Syndrome (Low and Intermediate-1 IPSS) Without the 5q Deletion and Transfusion Dependent Anaemia (ABRAZA)

Myelodysplastic Syndrome (MDS)

Spain
Genzyme, a Sanofi Company

Terminated

Rabbit Anti-thymocyte Globulin in the Treatment of Patients With Low to Intermediate-1 Risk Myelodysplastic Syndrome (RISE)

Myelodysplastic Syndrome (MDS)

Germany, United Kingdom, France, Netherlands
Heinrich-Heine University, Duesseldorf

Terminated

Efficacy and Tolerability of the Combination of Valproic Acid and Lenalidomide in the Treatment of Patients With Myelodysplastic Syndrome (VALENA)

Myelodysplastic Syndrome MDS

Germany
Telik

Terminated

Phase 2 Study of Telintra® in Deletion 5q Myelodysplastic Syndrome

Myelodysplastic Syndrome (MDS)

United States
Telik

Completed

Study of TLK199 Tablets in Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndrome (MDS)

United States
Seagen Inc.

Completed

A Phase I Study of Lintuzumab Combined With Lenalidomide in Patients With Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndrome (MDS)

United States
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
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
Helsinn Healthcare SA

Completed

Phase 2 Study Adding Pracinostat to a Hypomethylating Agent (HMA) in Patients With MDS Who Failed to Respond to Single Agent HMA (MEI-005)

Myelodysplastic Syndrome | MDS

United States
Sunnybrook Health Sciences Centre

Not yet recruiting

OURA Ring Wearable Testing in MDS Patients: a Feasibility and Discovery Pilot Study (OURA MDS)

Myelodysplastic Syndromes | MDS

Canada
Aprea Therapeutics

Terminated

APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes | MDS

United States
M.D. Anderson Cancer Center
Novartis

Completed

Phase I Study of Gimatecan in Patients With Myelodysplastic Syndromes

Myelodysplastic Syndromes | MDS

United States
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
Medical College of Wisconsin
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaborators

Completed

Allo vs Hypomethylating/Best Supportive Care in MDS (BMTCTN1102)

MDS

United States
Astex Pharmaceuticals, Inc.

Completed

Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral Cytidine Deaminase Inhibitor (CDAi) in Patients With Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndrome | MDS

United States, Canada
H. Lee Moffitt Cancer Center and Research Institute
Celgene Corporation

Completed

Lenalidomide and Prednisone in Low and Int-1 Myelodysplastic Syndrome (MDS) Non 5q MDS

Myelodysplastic Syndrome | MDS

United States
Groupe Francophone des Myelodysplasies
Celgene

Recruiting

Study Evaluating Combination of Luspatercept in LR-MDS Without RS Having Failed or Being Ineligible to ESA

Myelodysplastic Syndromes | MDS

France
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
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
Astex Pharmaceuticals, Inc.

Completed

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

AML | MDS | CMML

United States, Canada
Peter L Greenberg
Amgen

Completed

A Study of Darbepoetin Alfa in Patients With Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndromes | Myelodysplastic Syndromes (MDS) | Blood Cancer

United States
H. Lee Moffitt Cancer Center and Research Institute
GlaxoSmithKline

Terminated

Eltrombopag in Myelodysplastic Syndrome (MDS) Patients With Thrombocytopenia

Thrombocytopenia | Myelodysplastic Syndrome (MDS)

United States
Tarix Pharmaceuticals

Terminated

Phase I Study Evaluating TXA127 in Low/Intermediate-1 Risk Myelodysplastic Syndrome and Thrombocytopenia

Myelodysplastic Syndrome (MDS)

United States

Clinical Trials on MPS IIIB (Sanfilippo Syndrome)

Sponsors and Collaborators

Medical Conditions

Drug Interventions

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CROs in Burkina Faso

Conditions

Rare Diseases

Drug Interventions

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