Clinical Trials on MPS-IH (Hurler Syndrome)

Total 489 results

Otsuka Australia Pharmaceutical Pty Ltd

Completed

Refine and Assess the Readability of Patient, Carer, and Clinician Treatment Preference in Myelodysplasia Questionnaire (TPMQ)

Myelodysplastic Syndrome (MDS)

Australia
Yale University

Withdrawn

CPX-351 in Patients Treated for Higher-risk Myelodysplastic Syndromes Experiencing Hypomethylating Agent Failure.

MDS

United States
Assistance Publique - Hôpitaux de Paris

Unknown

Best Promising Drug Association With Azacitidine in Higher Risk Myelodysplastic Syndromes (AZA-PLUS)

MDS

France
Groupe Francophone des Myelodysplasies
Novartis

Completed

A Study of LDE255 in Combination With Azacitidine for High Risk Myelodysplastic Syndrome Patients (LDE255)

MDS

France
Gruppo Italiano Malattie EMatologiche dell'Adulto

Terminated

Velcade in Myelodysplastic Syndrome - Pilot Study

MDS

Italy
Kyowa Kirin Co., Ltd.

Completed

A Clinical Study of KRN321 in Adult Subjects With Myelodysplastic Syndrome

MDS

Japan, Korea, Republic of
Amgen

Completed

Romiplostim Treatment of Thrombocytopenia in Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndromes | Thrombocytopenia | MDS
Amgen

Completed

Determination of Safe and Effective Dose of Romiplostim (AMG 531) in Subjects With Myelodysplastic Syndrome (MDS)Receiving Hypomethylating Agents

Myelodysplastic Syndromes | Thrombocytopenia | MDS
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome

Myelodysplastic Syndrome (MDS) | Severe Aplastic Anemia | Hypo-Plastic MDS

United States
Novartis Pharmaceuticals

Completed

A Phase Ib Study of Panobinostat (LBH589) in Combination With 5-Azacitidine for Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML) or Acute Myeloid Leukemia (AML) Patients

Myelodysplastic Syndromes (MDS) | Acute Myeloid Leukemia (AML) | Chronic Myelomonocytic Leukemia (CMML)

Japan
Amgen

Completed

Evaluating the Safety of Long Term Dosing of Romiplostim (Formerly AMG 531) in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes | Thrombocytopenia | MDS | Hematology
Merck Sharp & Dohme LLC

Completed

Study of Sotatercept for the Treatment of Anemia in low-or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) or Non-proliferative Chronic Myelomonocytic Leukemia (CMML)

Myelodysplastic Syndromes | Anemia | Chronic Myelomonocytic Leukemia | Myelodysplastic Syndromes (MDS) | Chronic Myelomonocytic Leukemia (CMML) | Low to Intermediate-1 MDS

United States, France
Amgen

Completed

Determination of Safe Dose of Romiplostim (AMG 531) in Patients With Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes | Thrombocytopenia | MDS | Refractory Cytopenias
Institut de Recherches Internationales Servier

Active, not recruiting

Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation (AGILE)

Leukemia, Myeloid, Acute | Untreated AML | Newly Diagnosed Acute Myeloid Leukemia (AML) | AML Arising From Myelodysplastic Syndrome (MDS)

Spain, United States, France, Korea, Republic of, Taiwan, Australia, Canada, Germany, China, Italy, Austria, Czechia, Poland, Japan, United Kingdom, Russian Federation, Netherlands, Brazil, Israel, Mexico
Novartis Pharmaceuticals

Terminated

Phase II Study to Evaluate Overall Response in Patients With Higher Risk Myelodysplastic Syndromes (MDS) Treated With Azacitidine With or Without Deferasirox.

High Risk MDS

United States
Assistance Publique - Hôpitaux de Paris

Unknown

Myelodysplasic Syndromes and Risk Factors for Infection (MYRIFIC)

Infection | Risk Factors | Myelodysplastic Syndrome (MDS) | IPSS High Risk

France
University of Florida

Withdrawn

iCare3: Monitoring Circulating Cancer DNA After Chemotherapy in MDS and AML (iCare3)

Myelodysplastic Syndromes | AML | Acute Leukemia | MDS
University of Cologne

Not yet recruiting

Immun Response in Children With MIS-C

MIS-C Multisystem Inflammatory Syndrome in Children

Germany
Carelon Research

Active, not recruiting

Long-Term Outcomes After the Multisystem Inflammatory Syndrome in Children (MUSIC)

Multisystem Inflammatory Syndrome in Children (MIS-C)

United States, Canada
M.D. Anderson Cancer Center

Recruiting

Phase I/II Study of CAR.70- Engineered IL15-transduced Cord Blood-derived NK Cells in Conjunction With Lymphodepleting Chemotherapy for the Management of Relapse/Refractory Hematological Malignances

B-Cell Lymphoma | Myelodysplastic Syndromes (MDS) | Acute Myeloid Leukemia (AML)

United States
Onconova Therapeutics, Inc.

Completed

Efficacy and Safety of Oral Rigosertib in Transfusion-dependent, Low or Int-1 or Trisomy 8 Int-2 Myelodysplastic Syndrome (ONTARGET)

Myelodysplastic Syndrome | MDS | Trisomy 8

United States
Philipps University Marburg Medical Center
Linkoeping University

Completed

Treatment of Premenstrual Syndrome - Internet-based Self-help (praemensis)

Premenstrual Syndrome (PMS)

Germany
Ultragenyx Pharmaceutical Inc
PRA Health Sciences

Recruiting

Mucopolysaccharidosis VII Disease Monitoring Program

Sly Syndrome | Mucopolysaccharidosis VII | MPS VII | MPS 7

United States, Germany, Brazil, Argentina, France, Netherlands, Portugal, Spain, Turkey
Ultragenyx Pharmaceutical Inc

Completed

A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Mucopolysaccharidosis | Sly Syndrome | MPS VII | MPS 7

United States
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
Montefiore Medical Center
M.D. Anderson Cancer Center; Flamingo Therapeutics NV

Recruiting

Danvatirsen Monotherapy Followed by Combination With Venetoclax in Relapsed/Refractory MDS & AML

Acute Myeloid Leukemia | Myelodysplastic Syndromes | AML/MDS

United States
Yale University
National Institute of Allergy and Infectious Diseases (NIAID); Hospital for...

Active, not recruiting

Pilot Study of Gut Commensals in Antiphospholipid Syndrome

Antiphospholipid Syndrome (APS)

United States
Cell Medica Ltd
University College, London; Cell Therapy Catapult

Completed

A Phase I/II Study of Gene-modified WT1 TCR Therapy in MDS & AML Patients

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

United Kingdom, Germany, Belgium
Soonchunhyang University Hospital

Completed

Horner's SD After Thoracic Epidural Block

Horner's Syndrome

Korea, Republic of
Ruijin Hospital

Recruiting

Effect of Telitacicept on Antibody Titers in Primary APS Patients

Antiphospholipid Syndrome (APS)

China
University Hospital, Rouen

Unknown

Study of the Efficiency of Hydroxychloroquine on the Endothelial Dysfunction and Its Vascular Consequences During the Antiphospholipid Syndrome (APLAQUINE)

Antiphospholipid Syndrome (APS)

France
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
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
Humanigen, Inc.

Suspended

Study of KB004 in Subjects With Hematologic Malignancies (Myelodysplastic Syndrome, MDS, Myelofibrosis, MF)

Myelodysplastic Syndrome (MDS) | Myelofibrosis (MF)

United States, Australia
Institute of Hematology & Blood Diseases Hospital...
Beijing Health Alliance Charitable Foundation

Recruiting

The Tapering Dose of Luspatercept in Patients With Lower-risk Myelodysplastic Syndromes

Lower Risk MDS Per IPSS-R

China
Terra Biological LLC

Completed

Oxaloacetate Supplementation for Emotional PMS (OAA4PMS)

Premenstrual Syndrome (PMS)
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Safety of Peptide Vaccination for Patients With Myelodysplastic Syndrome

Myelodysplastic Syndrome (MDS)

United States
Daegu Catholic University Medical Center

Completed

Clinical Efficacy of Supraclavicular Block for Arthroscopic Shoulder Surgery

Horner's Syndrome

Korea, Republic of
University of Colorado, Denver

Completed

Health, Fitness, and Quality of Life in Adolescent Girls With Turner Syndrome

Turner Syndrome | Turner Syndrome Mosaicism, 45, X/46, XX or XY | Turner Syndrome Mosaicism 46,X,I(X)(Q10)/45,X | Turner Syndrome Karyotype 46,X With Abnormal Sex Chromosome , Except I(Xq)

United States
Peking University People's Hospital

Completed

Haplo-identical/Mismatched Hematopoietic Stem Cell Transplantation for Myelodysplastic Syndrome

Advanced MDS Without Identical Sibling Donor

China
Memorial Sloan Kettering Cancer Center

Completed

Maintenance Low Dose 5'-Azacitidine Post T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Myelodysplastic Syndrome and Acute Myelogenous Leukemia With High Risk for Post-Transplant Relapse

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

United States
University of Pennsylvania
Forest Laboratories

Terminated

Escitalopram for Premenstrual Syndrome (PMS) in Teens: A Pilot Study

PMS

United States
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
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
FibroGen
AstraZeneca; Astellas Pharma Inc

Terminated

Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome With Low Red Blood Cell Transfusion Burden

Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts)

United States, Australia, Belgium, Germany, Israel, Italy, Korea, Republic of, Russian Federation, Spain, United Kingdom, France, Turkey, Canada, Denmark, India, Poland
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells

MDS (Myelodysplastic Syndrome) | Severe Aplastic Anemia

United States
Eli Lilly and Company
Loxo Oncology, Inc.

Active, not recruiting

Study of Oral LY3410738 in Patients With Advanced Hematologic Malignancies With IDH1 or IDH2 Mutations

Acute Myeloid Leukemia (AML) | Chronic Myelomonocytic Leukemia (CMML) | Myelodysplastic Syndrome (MDS) | Myeloproliferative Neoplasms (MPNs)

United States, Spain, Korea, Republic of, France, Taiwan, Australia, Belgium, Singapore, Israel, Canada, Germany, Finland
Masonic Cancer Center, University of Minnesota

Completed

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

Hurler Syndrome

United States
Denver Health and Hospital Authority
University of Colorado, Denver

Terminated

Exploration of Pupil Dilation in Horner's Patients Taking Flomax

Horner Syndrome

United States
AbbVie

Terminated

Study to Evaluate Adverse Events and Movement of Lemzoparlimab in Body When Used Intravenously (IV) With Azacitidine Subcutaneously or IV and Venetoclax Orally in Participants With Acute Myeloid Leukemia and With Azacitidine With or Without Venetoclax in Participants With Myelodysplastic Syndrome

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

United States, Australia, Germany, Israel, Italy, Japan, Spain

Clinical Trials on MPS-IH (Hurler Syndrome)

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