Clinical Trials on MPS - Mucopolysaccharidosis in Netherlands

Total 41 results

Shire

Completed

Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients

Mucopolysaccharidosis (MPS)

United Kingdom, Netherlands
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
Alexion Pharmaceuticals

Completed

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

MPS IIIB (Sanfilippo Syndrome)

Spain, Netherlands, United Kingdom, United States, Brazil
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
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
BioMarin Pharmaceutical

Completed

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)

United States, France, Australia, Germany, Ireland, Austria, Belgium, Italy, Lithuania, Netherlands, Portugal, Sweden, United Kingdom
Genzyme, a Sanofi Company

Recruiting

Mucopolysaccharidosis I (MPS I) Registry

Mucopolysaccharidosis I (MPS I)

Croatia, United States, Argentina, Australia, Belgium, Brazil, Canada, Denmark, France, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Lebanon, Malaysia, Netherlands, Pakistan, Philippines, Poland, Saudi Arabia, Singapore, Slovaki... and more
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

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
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
LYSOGENE

Completed

Natural History Study of Patients With MPS IIIA

Mucopolysaccharidosis IIIA

Germany, Brazil, United Kingdom, France, Netherlands
LYSOGENE

Active, not recruiting

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (AAVance)

Mucopolysaccharidosis Type IIIA

Germany, United States, United Kingdom, France, Netherlands
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
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC

Completed

A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old

Mucopolysaccharidosis I | Scheie Syndrome | Hurler Syndrome | Hurler-Scheie Syndrome

Netherlands, France, Germany, United Kingdom
Denali Therapeutics Inc.

Enrolling by invitation

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Mucopolysaccharidosis II

United States, Canada, Belgium, Czechia, Netherlands
Shire

Terminated

Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Sanfilippo Syndrome

Netherlands, United Kingdom
Denali Therapeutics Inc.

Completed

A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Mucopolysaccharidosis II

United Kingdom, United States, Italy, Netherlands
Denali Therapeutics Inc.

Recruiting

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) (COMPASS)

Mucopolysaccharidosis II

United States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Colombia, Brazil
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC

Completed

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Mucopolysaccharidosis I | Scheie Syndrome | Hurler's Syndrome | Hurler-Scheie Syndrome

United States, Canada, United Kingdom, Brazil, Germany, Italy, Netherlands
Denali Therapeutics Inc.

Active, not recruiting

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Mucopolysaccharidosis II

United States, Netherlands, Canada, United Kingdom
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
Celgene

Recruiting

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Beta-thalassemia | Myelodysplastic Syndromes (MDS) | Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis

United States, Japan, Israel, United Kingdom, Turkey, Greece, France, Australia, Belgium, Bulgaria, Canada, Germany, Italy, Malaysia, Spain, Sweden, Taiwan, Thailand, Tunisia, China, Lebanon, Netherlands
Stichting Hemato-Oncologie voor Volwassenen Nederland
Swiss Group for Clinical Cancer Research

Active, not recruiting

Tolerability and Efficacy of Midostaurin to 10-day Decitabine in Unfit Adult AML and High Risk MDS Patients (HO155)

AML/MDS

Netherlands, Belgium, Germany, Switzerland
Goethe University
Stichting Hemato-Oncologie voor Volwassenen Nederland; Polish Adult Leukemia... and other collaborators

Terminated

Panobinostat Maintenance After HSCT fo High-risk AML and MDS

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

Germany, Netherlands
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
Bayer

Completed

BetaPlus Survey - Observational Study to Assess Drug Adherence in Patients With Multiple Sclerosis After Conversion to Betaferon® by Using Elements of the BetaPlus Program

Relapsing Remitting MS (RRMS) | Secondary Progressive MS (SPMS)

France, Germany, Korea, Republic of, Saudi Arabia, Spain, Taiwan, Czech Republic, Italy, Jordan, Lebanon, Turkey, Israel, Portugal, Netherlands, Iran, Islamic Republic of
Institut de Recherches Internationales Servier
Celgene Corporation

Active, not recruiting

Safety Study of AG-120 or AG-221 in Combination With Induction and Consolidation Therapy in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) With an IDH1 and/or IDH2 Mutation

Untreated AML | Newly Diagnosed Acute Myeloid Leukemia (AML) | AML Arising From Myelodysplastic Syndrome (MDS) | AML Arising From Antecedent Hematologic Disorder (AHD) | AML Arising After Exposure to Genotoxic Injury

United States, Germany, Netherlands
Incyte Corporation

Completed

A Study of Itacitinib in Combination With Low-Dose Ruxolitinib or Itacitinib Alone Following Ruxolitinib in Participants With Myelofibrosis

MPN (Myeloproliferative Neoplasms)

United States, Netherlands, Austria
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
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
Pfizer
Bristol-Myers Squibb

Completed

Study of Binimetinib + Nivolumab Plus or Minus Ipilimumab in Patients With Previously Treated Microsatellite-stable (MSS) Metastatic Colorectal Cancer With RAS Mutation

MSS | RAS-mutant Colorectal Cancer

United States, Spain, Belgium, United Kingdom, Netherlands
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
Celgene

Completed

Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence (RESUME)

Primary Myelofibrosis | MPN-associated Myelofibrosis

United States, Netherlands, France, Australia, Italy, United Kingdom, Canada, Germany, China, Spain, Belgium, Austria, Poland, Sweden, Japan, Russian Federation
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
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
Swedish Orphan Biovitrum

Recruiting

Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE (EMERALD)

Systemic Lupus Erythematosus | Macrophage Activation Syndrome | Still Disease | Secondary Hemophagocytic Lymphohistiocytosis | SJIA | AOSD | MAS

United States, Belgium, Italy, Netherlands, United Kingdom, China, France, Spain, Czechia, Canada, Sweden, Japan, Germany, Poland
Hoffmann-La Roche

Active, not recruiting

A Study to Evaluate Ocrelizumab Treatment in Participants With Progressive Multiple Sclerosis (CONSONANCE)

Progressive Multiple Sclerosis (PMS)

United States, Canada, Brazil, France, Netherlands, Ireland, Germany, Italy, Poland, Russian Federation, Spain, Colombia, Morocco, Hungary, Denmark, Lebanon, Mexico, Costa Rica, Bosnia and Herzegovina, Czechia, Egypt, Guatemala, Panama and more
InflaRx GmbH

Completed

Study of IFX-1 to Replace Steroids in Patients With Granulomatosis With Polyangiitis and Microscopic Polyangiitis. (IXchange)

Granulomatosis With Polyangiitis (GPA) | Microscopic Polyangiitis (MPA)

Germany, Russian Federation, Belgium, France, Spain, Czechia, Italy, Netherlands, Sweden, Switzerland, United Kingdom
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
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
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

Clinical Trials on MPS - Mucopolysaccharidosis in Netherlands

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