Clinical Trials on Mucopolysaccharidosis (MPS)

Total 806 results

Masonic Cancer Center, University of Minnesota

Terminated

Stem Cell Transplant for Hematological Malignancy

Multiple Myeloma | AML | MDS | Non-hodgkin's Lymphoma | Leukemia, Lymphocytic, Acute | Hodgkin's Disease | Leukemia, Myeloid, Chronic | Leukemia, Lymphocytic, Chronic | JMML

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
Compugen Ltd

Recruiting

COM902 (A TIGIT Inhibitor) in Subjects With Advanced Malignancies

Multiple Myeloma | Ovarian Cancer | Lung Cancer | Advanced Cancer | Colon Cancer | Plasma Cell Neoplasm | HNSCC | Microsatellite Stable Colorectal Carcinoma | MSS-CRC

United States
National Human Genome Research Institute (NHGRI)

Recruiting

Analysis of Specimens From Individuals With Pulmonary Fibrosis

Pulmonary Fibrosis | Healthy Volunteers | Hermansky-Pudlak Syndrome (HPS)

United States
Boston Children's Hospital

Completed

Pilot of Abatacept-based Immunosuppression for Prevention of Acute GvHD During Unrelated Donor HCT

AML | ALL | Biphenotypic Leukemia | Refractory Anemia | Refractory Cytopenia With Multilineage Dysplasia | Refractory Anemia With Ringed Sideroblasts | Undifferentiated Leukemia | Ref. Cytopenia w Multilineage Dysplasia & Ringed Sideroblasts | Refractory Anemia With Excess Blasts-1 (5-10% Blasts) | Refractory... and other conditions

United States
Everett Meyer
Doris Duke Charitable Foundation

Terminated

Haploidentical Allogeneic Transplant With Post-transplant Infusion of Regulatory T-cells

Chronic Lymphocytic Leukemia (CLL) | Acute Lymphoblastic Leukemia (ALL) | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML) | Leukemia, Acute | Chronic Myelogenous Leukemia (CML) | Non-Hodgkin Lymphoma (NHL)

United States
National Cancer Institute (NCI)

Completed

Wilm's Tumor 1 Protein Vaccine to Treat Cancers of the Blood

Myelodysplastic Syndrome (MDS) | Non-Hodgkin's Lymphoma (NHL) | Leukemia, Acute Myelogenous (AML) | Leukemia, Acute Lymphocytic (ALL) | Leukemia, Chronic Myelogenous (CML)

United States
National Human Genome Research Institute (NHGRI)

Terminated

Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome

Pulmonary Fibrosis | Metabolic Disease | Oculocutaneous Albinism | Hermansky-Pudlak Syndrome (HPS) | Platelet Storage Pool Deficiency

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

Recruiting

Assessing Changes in Multi-parametric MRI in MS Patients Taking Clemastine Fumarate as a Myelin Repair Therapy (ReVIVE)

Multiple Sclerosis, Chronic Progressive | Multiple Sclerosis, Relapsing-Remitting | Multiple Sclerosis (MS) | Multiple Sclerosis Relapse | Multiple Sclerosis, Primary Progressive | Multiple Sclerosis Brain Lesion | Multiple Sclerosis Benign

United States
Fred Hutchinson Cancer Center

Completed

Treosulfan, Fludarabine Phosphate, and Total-Body Irradiation in Treating Patients With Hematological Cancer Who Are Undergoing Umbilical Cord Blood Transplant

Myelodysplastic Syndrome (MDS) | Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Acute Biphenotypic Leukemia | Acute Myeloid Leukemia in Remission | Acute Lymphoblastic Leukemia in Remission | Blasts Under 5 Percent of Bone Marrow...

United States
Memorial Sloan Kettering Cancer Center
United States Department of Defense

Completed

Clinical and Histopathologic Characteristics of BAP1 Mutations

Renal Cell Carcinoma | Cholangiocarcinoma | Malignant Pleural Mesothelioma (MPM) | Choroidal Nevus | Primary Uveal Melanoma (UM) | Metastatic Uveal Melanoma (UM)

United States
University Hospital, Basel, Switzerland
Swiss National Science Foundation

Recruiting

Imaging the Interplay Between Axonal Damage and Repair in Multiple Sclerosis (INsIDER)

Multiple Sclerosis (MS) | Relapsing-remitting Multiple Sclerosis (RRMS) | Secondary-progressive Multiple Sclerosis (SPMS) | Primary Progressive Multiple Sclerosis (PPMS)

Switzerland
Merck Sharp & Dohme LLC

Active, not recruiting

A Study of the Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)

PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)

United States, Australia, Brazil, Italy, United Kingdom
Incyte Corporation

Completed

Open-Label, Dose-Escalation Study of Pemigatinib in Subjects With Advanced Malignancies - (FIGHT-101)

Breast Cancer | Multiple Myeloma | Gastric Cancer | Lung Cancer | Solid Tumor | Cholangiocarcinoma | Endometrial Cancer | Urothelial Cancer | Myeloproliferative Neoplasms | UC | MPN

United States, Denmark
University of Pennsylvania
University of South Florida; University of Oxford

Completed

The ANCA Vasculitis Questionnaire (AAV-PRO©) (AAV-PRO)

Vasculitis | Churg-Strauss Syndrome (CSS) | Microscopic Polyangiitis (MPA) | Eosinophilic Granulomatosis With Polyangiitis (Churg-Strauss) (EGPA) | Granulomatosis With Polyangiitis (Wegener's) (GPA) | Wegener Granulomatosis (WG) | ANCA-Associated Vasculitis (AAV)

United States
Children's Hospital Medical Center, Cincinnati
Memorial Sloan Kettering Cancer Center; Fred Hutchinson Cancer Center

Recruiting

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (RAFA)

Fanconi Anemia | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML) | Severe Marrow Failure

United States
Novartis Pharmaceuticals

Terminated

A Phase I/Ib Study of NZV930 Alone and in Combination With PDR001 and /or NIR178 in Patients With Advanced Malignancies.

Ovarian Cancer | Non-small Cell Lung Cancer (NSCLC) | Renal Cell Carcinoma (RCC) | Triple Negative Breast Cancer (TNBC) | Pancreatic Ductal Adenocarcinoma (PDAC) | Metastatic Castration Resistant Prostate Cancer (mCRPC) | Colorectal Cancer Microsatellite Stable (MSS)

Canada, Australia, Singapore, United States, Japan, Spain, United Kingdom
Beth Israel Deaconess Medical Center
Amgen

Completed

Nonmyeloablative Allo SCT for the Treatment of Hematologic Disorders (MINI HEME)

AML | MDS | CLL | ALL | CML Chronic Phase, Accelerated Phase, or Blast Crisis | RELAPSED NON-HODGKIN'S OR HODGKIN'S LYMPHOMA | APLASTIC ANEMIA | MULTIPLE MYELOMA | MYELOPROLIFERATIVE DISORDER (P Vera, CMML, ET)
Assistance Publique - Hôpitaux de Paris

Completed

Association Corticosteroid/Azathioprine in Microscopic Polyangiitis/ Polyarteritis Nodosa or Eosinophilic Granulomatosis With Polyangiitis (Churg Strauss Syndrome) (CHUSPAN2)

MPA | PAN or EGPA With FFS=0 | At Diagnosis or Within the First 15 Days Following Initiation of Corticosteroids

France
Wills Eye
United States Department of Defense

Completed

Electric Stimulation of the Eye to Improve Vision After Trauma (TES)

Trauma | Multiple Sclerosis (MS) | Non-arteritic Anterior Ischemic Optic Neuropathy (NAION)

United States
St. Jude Children's Research Hospital

Terminated

Repeat Transplantation for Relapsed or Refractory Hematologic Malignancies Following Prior Transplantation

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

United States
East Kent Hospitals University NHS Foundation Trust

Completed

Qualitative Study Patient & Physician Experiences Botox COVID-19 (QualBotoxCov)

Cerebral Stroke | Chronic Brain Injury | MS (Multiple Sclerosis)

United Kingdom
St. Jude Children's Research Hospital

Completed

Mismatched Family Member Donor Transplantation for Children and Young Adults With High Risk Hematological Malignancies

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

United States
Children's Hospital of Philadelphia
Eli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaborators

Recruiting

The Myelin Disorders Biorepository Project (MDBP)

Mucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditions

United States
Children's Hospital of Philadelphia
Illumina, Inc.

Active, not recruiting

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Mucopolysaccharidoses | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2 Gangliosidosis | Zellweger... and other conditions

United States
Cornell University
Laidlaw Foundation; Epperson Fund; Beck Fellowship; Dan Cane Fund

Completed

Effect of 'Cramp Bites' on Period Cramps in Women Aged 18-25 (NUTRITION; GYN)

Polycystic Ovary Syndrome | Ovarian Cysts | PCOS | Dysmenorrhea | Menstrual Pain | Premenstrual Dysphoric Disorder | Premenstrual Syndrome | Period Pain | Menstrual Problem | PMS | Menstrual Discomfort | Dysmenorrhea Primary | Cramps | Menstrual Cycle Abnormal | PCOS (Polycystic Ovary Syndrome) of Bilateral Ovaries | Period... and other conditions

United States
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
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
Children's Hospital Medical Center, Cincinnati

Recruiting

Two Point Discrimination (TPD)

Low Back Pain | Chronic Widespread Pain | Fibromyalgia | Complex Regional Pain Syndrome (CRPS) | Ehlers-Danlos Syndrome (EDS) | Amplified Musculoskeletal Pain Syndrome (AMPS)

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
Diane George

Active, not recruiting

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

Acute Lymphoblastic Leukemia (ALL) | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML) | Chronic Myeloid Leukemia (CML) | Juvenile Myelomonocytic Leukemia (JMML) | Lymphoma (Hodgkin's and Non-Hodgkin's)

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
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
University of Southern Denmark
University of Aarhus; Danish Cancer Society; Odense Patient Data Explorative... and other collaborators

Active, not recruiting

Chronic Inflammatory Disease, Lifestyle and Risk of Disease (PROCID-DCH)

Autoimmune Diseases | Inflammatory Bowel Diseases | Spondylarthropathies | Multiple Sclerosis (MS) | Crohn Disease (CD) | Ulcerative Colitis (UC) | Arthritis, Rheumatoid (RA) | Arthritis, Psoriatic (PsA) | Psoriasis (PsO)

Denmark
Chugai Pharmaceutical

Not yet recruiting

Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)

Antiphospholipid Syndrome (APS) | Immune Thrombocytopenia (ITP) | Dermatomyositis (DM) | Bullous Pemphigoid (BP) | Behçet's Syndrome (BS) | Immune-mediated Necrotizing Myopathy (IMNM)

United States, Japan
Peter Merkel
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaborators

Recruiting

Clinical Transcriptomics in Systemic Vasculitis (CUTIS) (CUTIS)

Vasculitis | Eosinophilic Granulomatosis With Polyangiitis (EGPA) | Cryoglobulinemic Vasculitis (CV) | Drug-induced Vasculitis | IgA Vasculitis | Isolated Cutaneous Vasculitis | Granulomatosis With Polyangiitis (GPA) | Microscopic Polyangiitis (MPA) | Polyarteritis Nodosa (PAN) | Urticarial Vasculitis

United States, Canada
Fujian Cancer Hospital

Not yet recruiting

the Efficacy and Safety of Dalpiciclib Combined With Third-generation EGFR-TKI in Patients With EGFR Mutation and Meningial Metastasis in Non-small Cell Lung Cancer Progressing Through Third-generation TKI and Platinum-containing Two-drug Chemotherapy

Non-small Cell Lung Cancer Confirmed by Histopathology or Cytology | The TNM (8th) Phase is IV | With EGFR Mutations | Meningeal Progression Occurred After Previous Platinum-containing Two-drug Chemotherapy and Third-generation EGFR-TKI Treatment | Male or Female | The Age of 18 Years Old or... and other conditions
National Human Genome Research Institute (NHGRI)

Completed

Clinical and Pathophysiological Investigations Into Erdheim Chester Disease

Cancer | Myelofibrosis | Pulmonary Fibrosis | Gaucher Disease | Hermansky-Pudlak Syndrome (HPS)

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
Culicchia Neurological Clinic
Flowonix Medical

Recruiting

Transitioning to a Valve-Gated Intrathecal Drug Delivery System (IDDS)

Traumatic Brain Injury | Muscle Spasticity | CVA (Cerebrovascular Accident) | Cerebral Palsy, Spastic | MS (Multiple Sclerosis) | Injuries, Spinal Cord

United States
Novartis Pharmaceuticals

Terminated

A Phase 2 Study of NIR178 in Combination With PDR001 in Patients With Solid Tumors and Non-Hodgkin Lymphoma

Melanoma | Head and Neck Cancer | Pancreatic Cancer | Urothelial Cancer | NSCLC, Non Small Cell Lung Cancer | RCC, Renal Cell Cancer | DLBCL, Diffused Large B Cell Lymphoma | MSS, Microsatellite Stable Colon Cancer | TNBC, Triple Negative Breast Cancer | mCRPC, Metastatic Castration Resistant Prostate...

Belgium, Italy, Taiwan, Germany, Spain, United States, Argentina, Australia, Austria, Czechia, France, Japan, Netherlands, Switzerland, Singapore
Ashvattha Therapeutics, Inc.

Recruiting

Safety, PK and Biodistribution of 18F-OP-801 in Patients With ALS, AD, MS, PD and Healthy Volunteers

Amyotrophic Lateral Sclerosis (ALS) | Multiple Sclerosis (MS) | Parkinson Disease (PD) | Alzheimer Disease (AD)

United States
QuantalX Neuroscience

Completed

Development of Automated Analysis to Electroencephelogram (EEG) Data in Patients Treated at the Sagol Hyperbaric Medicine and Research Center at the Years 2017-2019.

Healthy | Hemorrhage | Cognitive Impairment | Dementia | Fibromyalgia | TBI (Traumatic Brain Injury) | Cognitive Decline | Concussion, Brain | MS (Multiple Sclerosis) | Adhd | CVA | ABD | Mci

Israel
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
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
Avanir Pharmaceuticals

Completed

PRISM Registry: Pseudobulbar Affect Registry Series

Stroke | Parkinson's Disease | Traumatic Brain Injury | Amyotrophic Lateral Sclerosis (ALS) | Alzheimer's Disease | Multiple Sclerosis (MS)

United States
National Institute of Allergy and Infectious Diseases...
Rho Federal Systems Division, Inc.; Autoimmunity Centers of Excellence

Completed

COVID-19 Booster Vaccine in Autoimmune Disease Non-Responders

Systemic Lupus Erythematosus (SLE) | Multiple Sclerosis (MS) | Rheumatoid Arthritis (RA) | Juvenile Dermatomyositis (JDM) | Juvenile Idiopathic Arthritis (JIA) | Pemphigus Vulgaris | Systemic Sclerosis (SSc) | Pediatric SLE | Pediatric-Onset Multiple Sclerosis (POMS)

United States
University of Pennsylvania
University of South Florida; Duke University

Recruiting

Vasculitis Pregnancy Registry (V-PREG)

Vasculitis | Wegener's Granulomatosis | Systemic Vasculitis | Eosinophilic Granulomatosis With Polyangiitis (EGPA) | Churg-Strauss Syndrome (CSS) | Cryoglobulinemic Vasculitis | Behcet's Disease | CNS Vasculitis | IgA Vasculitis | Granulomatosis With Polyangiitis (GPA) | Microscopic Polyangiitis (MPA) | Polyarteritis... and other conditions

United States
Massachusetts General Hospital

Recruiting

Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders (BMT)

Autoimmune Diseases | Multiple Myeloma | Hodgkin Disease | AL Amyloidosis | Myelofibrosis | Chronic Kidney Disease | Sickle Cell Anemia | Thalassemia | Aplastic Anemia | Chronic Lymphocytic Leukemia (CLL) | Acute Myeloid Leukemia (AML) | Acute Lymphoblastic Leukemia (ALL) | Myelodysplastic Syndrome (MDS) | Non-Hodgkin... and other conditions

United States

Clinical Trials on Mucopolysaccharidosis (MPS)

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Slovakia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations