Clinical Trials on MDS

Total 724 results

Radboud University Medical Center

Completed

Sodium Lactate Infusion in GLUT1DS Patients

Epilepsy | Ketogenic Dieting | Lactic Acid Blood Increased | GLUT1DS1 | GLUT1 Deficiency Syndrome | GLUT1DS2

Netherlands
Erimos Pharmaceuticals

Terminated

Phase 1 Study of Terameprocol (EM-1421) in Patients With Leukemia

Chronic Lymphocytic Leukemia (CLL) | Acute Myeloid Leukemia (AML) | Chronic Myelomonocytic Leukemia (CMML) | Myelodysplastic Syndrome (MDS) | Acute Lymphocytic Leukemia (ALL) | Leukemias | Adult T Cell Leukemia (ATL) | Chronic Myeloid Leukemia (CML-BP)

United States
St. Jude Children's Research Hospital

Completed

Haploidentical Stem Cell Transplantation for Patients With Hematologic Malignancies

Myelodysplastic Syndrome (MDS) | Leukemia, Acute Lymphocytic (ALL) | Leukemia, Myeloid, Acute(AML) | Leukemia, Myeloid, Chronic(CML) | Hemoglobinuria, Paroxysmal Nocturnal (PNH) | Lymphoma, Non-Hodgkin (NHL) | Juvenile Myelomonocytic Leukemia(JMML)

United States
Jiulongpo No.1 People's Hospital

Recruiting

NHFOV as Primary Support in Very Preterm Infants With RDS

to Test the Hypothesis That NHFOV is More Effective Than nCPAP in the Treatment of Respiratory Distress Syndrome (RDS) in Verypreterm Neonates

China
Sangamo Therapeutics

Terminated

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

Mucopolysaccharidosis II | MPS II

United States
St. Jude Children's Research Hospital

Recruiting

Provision of TCRγδ T Cells and Memory T Cells Plus Selected Use of Blinatumomab in Naïve T-cell Depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies Relapsed or Refractory Despite Prior Transplantation

Acute Myeloid Leukemia (AML) | Acute Lymphoblastic Leukemia (ALL) | Myelodysplastic Syndrome (MDS) | Myeloid Sarcoma | Chronic Myeloid Leukemia (CML) | Non-Hodgkin Lymphoma (NHL) | Juvenile Myelomonocytic Leukemia (JMML)

United States
Celldex Therapeutics

Terminated

A Safety and Tolerability Study of CDX-301 With or Without Plerixafor for Stem Cell Mobilization in Matched Related Allogeneic Donor/Recipient Sibling Transplant Pairs

Chronic Lymphocytic Leukemia (CLL) | Acute Lymphoblastic Leukemia (ALL) | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML) | Chronic Myelogenous Leukemia (CML) | Non-Hodgkins Lymphoma (NHL) | For Donors | Related Donors Giving Peripheral Blood Stem Cells (PBSC) to a Sibling | For Recipients and other conditions

United States
Masonic Cancer Center, University of Minnesota

Completed

Myeloablative Umbilical Cord Blood Transplantation in Hematological Diseases

Lymphoma | Acute Myeloid Leukemia | Myelodysplastic Syndromes | Leukemia | Multiple Myeloma | Non-Hodgkin's Lymphoma | Myelofibrosis | Chronic Lymphocytic Leukemia | Acute Lymphocytic Leukemia | MDS | Chronic Myelogenous Leukemia | Prolymphocytic Leukemia | Refractory Anemia

United States
National Institute of Allergy and Infectious Diseases...
Autoimmunity Centers of Excellence; Clinical Trials in Organ Transplantation...

Completed

COVID-19: Pediatric Research Immune Network on SARS-CoV-2 and MIS-C (PRISM)

Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) | Coronavirus Disease 2019 (COVID-19) | Multisystem Inflammatory Syndrome in Children (MIS-C)

United States
Johns Hopkins University
National Institute of Allergy and Infectious Diseases (NIAID); National Institutes... and other collaborators

Active, not recruiting

A Nurse Case Management Intervention to Improve MDR-TB/HIV Co-Infection Outcomes

HIV | MDR-TB

South Africa
University of California, San Francisco
United States Department of Defense

Recruiting

A Closed Loop, Doctor to Patient, Mobile Application for Depression in People With Multiple Sclerosis (MS-CATCH)

Depression | Multiple Sclerosis | MS

United States
US Department of Veterans Affairs

Completed

Effect of Robot-assisted Training on Foot Drop in Multiple Sclerosis

MS (Multiple Sclerosis)

United States
Parc de Salut Mar

Completed

Normalization of dyrk1A and APP Function as an Approach to Improve Cognitive Performance and Decelerate AD Progression in DS Subjects: Epigallocatechin Gallate as Therapeutic Tool

Down Syndrome (DS)

Spain
Takeda

Completed

A Study to Improve the Awareness of Mucopolysaccharidosis Type II in Brazil

Mucopolysaccharidosis (MPS)

Brazil
Jazz Pharmaceuticals

Terminated

Safety and Effectiveness of Nabiximols Oromucosal Spray as Add-on Therapy in Participants With Spasticity Due to Multiple Sclerosis (RELEASE MSS3)

Multiple Sclerosis (MS)

United States, Poland, Czechia, United Kingdom, Romania
Duke University

Recruiting

MPS II Immunophenotyping

Mucopolysaccharidosis II | Hunter Syndrome | MPS II

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
Ultragenyx Pharmaceutical Inc

Available

Expanded Access to Triheptanoin

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Institute of Hematology & Blood Diseases Hospital

Recruiting

National Longitudinal Cohort of Hematological Diseases (NICHE)

Lymphoma | Acute Myeloid Leukemia | Leukemia | Hemophilia A | Hemophilia B | Hemophilia | Multiple Myeloma | Myelodysplastic Syndrome | Bone Marrow Transplantation | MDS | Aplastic Anemia | Bleeding Disorder | Myeloma, Multiple | Myelomas, Multiple | Leukemias, Acute Myeloid | Myeloid Leukemias, Acute | Hemophilia As | Hemophilia... and other conditions

China
Ultragenyx Pharmaceutical Inc

Completed

Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

United States, Spain, Australia, France, Israel, Italy, United Kingdom
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
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
Cure CMD
Congenital Muscle Disease International Registr

Withdrawn

LAMA2-related Muscular Dystrophy Brain Study

LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)

United States
REGENXBIO Inc.

Active, not recruiting

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Mucopolysaccharidosis Type II (MPS II)

United States, Canada
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
Sanofi

Completed

A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting (HomERT)

Pompe Disease | Mucopolysaccharidosis Type I (MPS I)

Italy
Regeneron Pharmaceuticals

Completed

Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)

Genetic Diseases, Inborn | Familial Cold Autoinflammatory Syndrome (FCAS) | Familial Cold Urticaria | Muckle-Wells Syndrome (MWS)

United States
Sheikh Shakhbout Medical City

Recruiting

EXOPULSE Mollii Suit, Motor Function & Multiple Sclerosis (EXOSEP 2)

Spasticity, Muscle | Sclerosis, Multiple | Spastic | MS (Multiple Sclerosis)

United Arab Emirates
Stony Brook University
Novartis

Completed

Computerized Exercise Training for Cognitive Remediation in Adults With Multiple Sclerosis Treated With Gilenya

Multiple Sclerosis | Cognitive Deficits | Gilenya Modifying Therapy for MS

United States
Takeda

Not yet recruiting

A Study on Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS) in Children, Teenagers and Adults in Portugal (DRALEGA-PT)

Dravet Syndrome (DS) | Lennox-Gastaut Syndrome (LGS)
Takeda

Recruiting

A Study to Gather Information About Overall Occurrence and New Cases of Dravet and Lennox-Gastaut Syndromes in Children, Teenagers and Adults in Spain (DRALEGA)

Dravet Syndrome (DS) | Lennox-Gastaut Syndrome (LGS)

Spain
Adlai Nortye Biopharma Co., Ltd.
Merck Sharp & Dohme LLC

Active, not recruiting

AN0025 and Pembrolizumab Combination in Advanced Solid Tumors

Cervical Cancer | Triple-negative Breast Cancer | NSCLC, Squamous or Non-Squamous | Urothelial Carcinoma of the Bladder | Microsatellite Stable (MSS) Colorectal Cancer (CRC)

United States, France
Takeda

Recruiting

A Study of Soticlestat as an Add-on Therapy in Children and Adults With Dravet Syndrome or Lennox-Gastaut Syndrome

Lennox Gastaut Syndrome (LGS) | Dravet Syndrome (DS)

United States, China, Spain, France, Belgium, Australia, Brazil, Canada, Germany, Greece, Hungary, Italy, Japan, Latvia, Netherlands, Poland, Serbia, Mexico, Russian Federation, Ukraine
Adrian Lacy
Ultragenyx Pharmaceutical Inc

Completed

An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome (GLUT1DS)

Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)

United States
Takeda

Active, not recruiting

A Study of Soticlestat in Adults and Children With Rare Epilepsies (Endymion 1)

Epilepsy | Dravet Syndrome (DS) | Lennox-Gastaut Syndrome (LGS)

United States, Canada, Australia, China, Israel, Poland, Spain, Portugal
Qilu Hospital of Shandong University

Unknown

Imaging Evaluation of Central Nervous Autoimmune Diseases

MS (Multiple Sclerosis) | NMO Spectrum Disorder
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
NICHD Neonatal Research Network
National Center for Research Resources (NCRR)

Terminated

Inhaled Nitric Oxide for Preterm Infants With Severe Respiratory Failure (Preemie iNO)

Pneumonia | Sepsis | Hypertension, Pulmonary | Infant, Small for Gestational Age | Infant, Premature | Infant, Low Birth Weight | Infant, Newborn | Respiratory Distress Syndrome (RDS)

United States
Sheikh Shakhbout Medical City

Recruiting

EXOPULSE Mollii Suit, Spasticity, Muscular Oxygenation & Multiple Sclerosis (ENNOX 2)

Spasticity, Muscle | Oxygen Consumption | Sclerosis, Multiple | Spastic | MS (Multiple Sclerosis)

United Arab Emirates
Brown, Theodore R., M.D., MPH
Evergreen Healthcare

Completed

A Pilot Study of Warm and Cold Compress to Reduce Injection Site Erythema Due to Peginterferon-beta-1a in MS (PEG-Thermal)

Erythema | MS (Multiple Sclerosis)

United States
Polyphor Ltd.

Completed

Safety and Efficacy of POL6326 for Mobilization/Transplant of Sibling Donor in Patients With Hematologic Malignancies

Myeloproliferative Disorders | Adult Acute Lymphoblastic Leukemia in Remission | Chronic Lymphocytic Leukemia (CLL) | Myelodysplastic Syndrome (MDS) | Acute Myeloid Leukemia in Remission | Chronic Myelogenous Leukemia (CML) | Multiple Myeloma (MM) | Non-Hodgkin's Lymphoma (NHL) or Hodgkin's Disease...

United States
University Health Network, Toronto

Recruiting

Dyspnea in Hypermobile Ehlers-Danlos Syndrome and Hypermobility Spectrum Disorder

Hypermobile EDS (hEDS) | Hypermobility Syndrome

Canada
Ultragenyx Pharmaceutical Inc

Terminated

Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

United States, Spain, France, Germany, Italy, United Kingdom
University at Buffalo
University of Pavia

Completed

Centralized Reading Assessment of Chronic CerebroSpinal Venous Insufficiency (CCSVI) in Patients With Multiple Sclerosis and Other Neurological Diseases

Multiple Sclerosis (MS) | Clinically Isolated Syndrome (CIS) | Other Neurological Disorders (OND) | Healthy (HC)
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
Tel-Aviv Sourasky Medical Center

Unknown

Intrathecal Methotrexate for Progressive Multiple Sclerosis: An Open Label Single Arm Study (ITMTXPMS)

To Evaluate the Effect of Therapy With IT MTX on the Disease Course of Patients With Progressive MS
Daniel Bernstein

Completed

B-Receptor Signaling in Cardiomyopathy

Soft Tissue Sarcoma | Lymphoma, Non-Hodgkin | Leukemia | Breast Cancer | Myeloproliferative Disorders | Multiple Myeloma | Anemia | Pancreatic Cancer | Hodgkin Disease | Esophageal Cancer | Amyloidosis | Lung Cancer | Bone Cancer | Prostate Cancer | Neuroendocrine Tumors | Mesothelioma | Skin Cancer | Thyroid Cancer | Anal Cancer | Hepatobiliary Neoplasm and other conditions

United States
Columbia University

Completed

SUNLIGHT Study: Online Support Groups for MS to Address COVID-19

COVID-19 | MS (Multiple Sclerosis) | Support Groups

United States
Center for International Blood and Marrow Transplant...
National Marrow Donor Program

Recruiting

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

Autoimmune Diseases | Hodgkin Lymphoma | Hematologic Malignancies | Acute Lymphoblastic Leukemia (ALL) | Non-hodgkin Lymphoma | Severe Aplastic Anemia | Inherited Disorders of Metabolism | Inherited Abnormalities of Platelets | Histiocytic Disorders | Acute Myelogenous Leukemia (AML or ANLL) | Other Acute... and other conditions

United States
LMU Klinikum
University of Pisa; German Federal Ministry of Education and Research; European...

Recruiting

Global Registry and Natural History Study for Mitochondrial Disorders

Mitochondrial Diseases | MDS | Mitochondrial Myopathies | MELAS Syndrome | MIDD | Kearns-Sayre Syndrome | MERRF Syndrome | Barth Syndrome | Leigh Syndrome | MNGIE | LHON | Pearson Syndrome | NARP Syndrome | Coenzyme Q10 Deficiency | SANDO | SCAE | MIRAS | CPEO

Austria, Germany, Italy

Clinical Trials on MDS

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Burundi

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations