Clinical Trials on Sanfilippo Syndrome Type A (MPS IIIA) in Italy

Total 27 results

Alexion Pharmaceuticals

Terminated

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB

MPS IIIB (Sanfilippo B Syndrome)

Spain, United States, Brazil, United Kingdom, Portugal, Italy
Shire

Terminated

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

Sanfilippo Syndrome | Mucopolysaccharidosis (MPS)

United States, United Kingdom, Spain, Germany, France, Italy, Netherlands
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
Shire

Completed

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

Sanfilippo Syndrome

United States, Spain, United Kingdom, Argentina, France, Germany, Italy, Netherlands
BioMarin Pharmaceutical

Terminated

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

Morquio A Syndrome | MPS IV A | Mucopolysaccharidosis IVA

France, United Kingdom, Taiwan, United States, Argentina, Netherlands, Canada, Brazil, Germany, Italy
BioMarin Pharmaceutical

Completed

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVA

Taiwan, United States, Italy, United Kingdom
BioMarin Pharmaceutical

Completed

A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

MPS IV A

United States, Canada, France, United Kingdom, Taiwan, Argentina, Colombia, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, Germany, Portugal, Italy, Qatar
BioMarin Pharmaceutical
ICON plc

Completed

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) (MARS)

Mucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVA

United States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
BioMarin Pharmaceutical

Completed

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IV A

United States, Canada, France, Taiwan, Argentina, Colombia, Spain, Turkey, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, United Kingdom, Germany, Norway, Portugal, Italy, Qatar
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
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

Completed

A Safety and Efficacy Study of LGH447 in Patients With Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome (MDS)

AML and High Risk MDS

Germany, Italy, France, Netherlands, United States, Australia, Japan
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
Onconova Therapeutics, Inc.
The Leukemia and Lymphoma Society

Completed

Randomized Study of ON 01910.Na in Refractory Myelodysplastic Syndrome Patients With Excess Blasts (ONTIME)

Myelodysplastic Syndromes | Chronic Myelomonocytic Leukemia | MDS | RAEB

United States, Belgium, Germany, Spain, France, Italy
medac GmbH
Syneos Health; Celerion

Completed

Treosulfan-based Conditioning in Paediatric Patients With Haematological Malignancies

Myelodysplastic Syndromes (MDS) | Acute Lymphoblastic Leukaemias (ALL) | Acute Myeloid Leukaemias (AML) | Juvenile Myelomonocytic Leukaemias (JMML)

Germany, United Kingdom, Italy, Austria, Czechia, Poland
Gamida Cell ltd

Completed

Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Cells, in Patients With HM

Hematological Malignancies | Acute Myeloid Leukemia (AML) | Acute Lymphoblastic Leukemia (ALL) | Myelodysplastic Syndrome (MDS)

United States, Singapore, Spain, Netherlands, Italy
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Completed

Tacrolimus and Mycophenolate Mofetil in Preventing Graft-Versus-Host Disease in Patients Who Have Undergone Total-Body Irradiation With or Without Fludarabine Phosphate Followed by Donor Peripheral Blood Stem Cell Transplant for Hematologic Cancer

Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell... and other conditions

United States, Italy
Amgen

Terminated

Safety, Tolerability, Pharmacokinetics and Efficacy of AMG 397 in Subjects With Selected Relapsed or Refractory Hematological Malignancies

Acute Myeloid Leukemia | Multiple Myeloma | AML | Myelodysplastic Syndrome | MDS | Non-Hodgkins Lymphoma | NHL

United States, Australia, Italy, France, Greece, Japan
Constellation Pharmaceuticals
The Leukemia and Lymphoma Society

Active, not recruiting

A Phase 2 Study of CPI-0610 With and Without Ruxolitinib in Patients With Myelofibrosis

Neoplasms | Neoplasms by Histologic Type | Leukemia | Preleukemia | Primary Myelofibrosis | Myeloproliferative Disorders | Myelofibrosis | Precancerous Conditions | Myelodysplastic/Myeloproliferative Neoplasm | Myelodysplastic Syndrome (MDS) | Leukemia, Myelocytic, Acute | Bone Marrow Disease | Hematological Disease and other conditions

Canada, United States, United Kingdom, Germany, Italy, Belgium, France, Netherlands, Poland
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Completed

Fludarabine Phosphate, Low-Dose Total-Body Irradiation, and Donor Stem Cell Transplant Followed by Cyclosporine, Mycophenolate Mofetil, Donor Lymphocyte Infusion in Treating Patients With Hematopoietic Cancer

Stage II Multiple Myeloma | Stage III Multiple Myeloma | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult... and other conditions

United States, Italy
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute...

Completed

Alemtuzumab, Fludarabine Phosphate, and Low-Dose Total Body Irradiation Before Donor Stem Cell Transplantation in Treating Patients With Hematological Malignancies

Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent Adult Diffuse Large Cell Lymphoma | Recurrent Adult Diffuse Mixed Cell Lymphoma | Recurrent... and other conditions

United States, Italy
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Completed

Alemtuzumab, Fludarabine Phosphate, and Total-Body Irradiation Followed by Cyclosporine and Mycophenolate Mofetil in Treating Patients Who Are Undergoing Donor Stem Cell Transplant for Hematologic Cancer

Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent Adult Diffuse Large Cell... and other conditions

United States, Italy
Gamida Cell ltd

Active, not recruiting

Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS

Lymphoma | Acute Leukemia | Hematological Malignancies | Acute Lymphoblastic Leukemia (ALL) | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML) | Chronic Myelogenous Leukemia (CML)

Spain, United States, United Kingdom, Israel, Singapore, Brazil, Italy, France, Netherlands, Portugal
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute...

Completed

Mycophenolate Mofetil and Cyclosporine in Reducing Graft-Versus-Host Disease in Patients With Hematologic Malignancies or Metastatic Kidney Cancer Undergoing Donor Stem Cell Transplant

Clear Cell Renal Cell Carcinoma | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent... and other conditions

United States, Germany, Italy
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute...

Completed

Low-Dose Total Body Irradiation and Donor Peripheral Blood Stem Cell Transplant Followed by Donor Lymphocyte Infusion in Treating Patients With Non-Hodgkin Lymphoma, Chronic Lymphocytic Leukemia, or Multiple Myeloma

Stage II Multiple Myeloma | Stage III Multiple Myeloma | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent Adult Diffuse Large Cell Lymphoma | Recurrent Adult Diffuse Mixed Cell Lymphoma and other conditions

United States, Germany, Italy
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute...

Completed

Fludarabine Phosphate and Total-Body Radiation Followed by Donor Peripheral Blood Stem Cell Transplant and Immunosuppression in Treating Patients With Hematologic Malignancies

Stage II Multiple Myeloma | Stage III Multiple Myeloma | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult... and other conditions

United States, Germany, Italy
Chinook Therapeutics U.S., Inc.

Active, not recruiting

Atrasentan in Patients With Proteinuric Glomerular Diseases (AFFINITY)

Diabetic Kidney Disease | Focal Segmental Glomerulosclerosis | Immunoglobulin A Nephropathy | IgA Nephropathy | Diabetic Nephropathy Type 2 | Alport Syndrome

United States, Spain, Korea, Republic of, Australia, Italy, United Kingdom

Clinical Trials on Sanfilippo Syndrome Type A (MPS IIIA) in Italy

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