Clinical Trials on MPS - Mucopolysaccharidosis

Total 807 results

Shire

Completed

Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)

Hunter Syndrome

Mexico
Takeda
Takeda Development Center Americas, Inc.

Active, not recruiting

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Hunter Syndrome

United States, Germany, Malaysia, Philippines, Thailand, Dominican Republic, Serbia, Vietnam
University of California, San Francisco
Duke University

Recruiting

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases (IUERT)

Wolman Disease | MPS IVA | Pompe Disease Infantile-Onset | Gaucher Disease, Type 2 | MPS VI | MPS I | Gaucher Disease, Type 3 | MPS II | Mps VII

United States
University of Pittsburgh

Recruiting

Longitudinal Study of Neurodegenerative Disorders

Lysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditions

United States
Takeda

Available

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Hunter Syndrome

United States, Australia, Mexico, Spain, United Kingdom
Green Cross Corporation

Completed

Safety and Efficacy of Hunterase (GC1111)

Hunter Syndrome

Korea, Republic of
Shire

Completed

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

Hunter Syndrome

United States, United Kingdom
Aldagen

Terminated

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases (UCBT-002)

Mucopolysaccharidosis | Inborn Errors of Metabolism | Lysosomal Storage Disorders | Inherited Metabolic Diseases | Peroxisomal Storage Diseases

United States
Gwynn Long, M.D.
ADC Therapeutics S.A.

Terminated

ADCT-301 in Patients With R/R AML, MDS, or MDS/MPN

Acute Myeloid Leukemia (AML) | Myelodysplastic Syndrome (MDS) | Myeloproliferative Neoplasm (MDS/MPN)

United States
Shire

Completed

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

Hunter Syndrome

United States, Spain, Canada, United Kingdom, Mexico, Australia, France
Shire

Completed

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

Hunter Syndrome

United States, United Kingdom
Shire

Completed

A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®

Hunter Syndrome

United States, United Kingdom
Shire

Completed

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

Hunter Syndrome

United Kingdom, United States, Brazil
University of British Columbia
Medical University of Graz; Hospital de Clinicas de Porto Alegre

Unknown

Natural History of Morquio B and Late-Onset of GM1 Gangliosidosis

Morquio B Disease | GM1 Gangliosidosis Type III

Canada
Takeda

Recruiting

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

Hunter Syndrome

Canada, France
Jacqueline Garcia, MD
National Institutes of Health (NIH)

Recruiting

Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN

Acute Myeloid Leukemia (AML) | Chronic Myelomonocytic Leukemia (CMML) | Myelodysplastic Syndrome (MDS) | Hematopoietic Stem Cell Transplant | MDS/Myeloproliferative Neoplasm-unclassifiable (MDS/MPN-unclassifiable)

United States
Masonic Cancer Center, University of Minnesota

Recruiting

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditions

United States
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, United Kingdom, Turkey, Greece, France, Australia, Belgium, Bulgaria, Canada, Germany, Israel, Italy, Malaysia, Spain, Sweden, Taiwan, Thailand, Tunisia, Lebanon, China, Netherlands
Shire

Completed

An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment

Hunter Syndrome

United States, United Kingdom, Canada
Johannes Gutenberg University Mainz

Unknown

Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer. (MPSORA)

Mucopolysaccharidosis | Fabry Disease

Germany
Shire

Completed

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment (AIM-IT)

Hunter Syndrome

Spain, United States, Canada, United Kingdom, France, Australia, Mexico
Assiut University

Unknown

Prognostic Value of Serum Erythropoietin Level,Ferritin Level and Fibrinogen in Adult Low Risk MDS

MDS
Groupe Francophone des Myelodysplasies
Novartis

Unknown

A Study of Combined Deferasirox, Vitamin D and Azacytidine in High Risk MDS (GFM-EXVD-AZA)

MDS

France, Belgium
Stanford University
National Institutes of Health (NIH)

Completed

Non-Myeloablative Allogeneic Transplant for Myelodysplastic Syndromes and Myeloproliferative Disorders

Myelodysplastic Syndromes | Myeloproliferative Disorders | Myelodysplastic Syndromes (MDS) | Blood and Marrow Transplant (BMT) | Myeloproliferative Disorders (MPD)

United States
Zhejiang Provincial Hospital of TCM

Unknown

Study on High-risk MDS Patients Based on RNA-seq Technology (MDS&RNA-seq)

MDS

China
University Hospital Tuebingen

Recruiting

Combination Salvage Therapy With Venetoclax and Decitabine in Relapsed/Refractory AML (VenSwitch)

AML | MDS

Germany
The Second Hospital of Shandong University

Not yet recruiting

Allogenic NK Cell Transfusion Azacitidine MDS-EB

MDS-EB

China
Groupe Francophone des Myelodysplasies
Astex Pharmaceuticals, Inc.

Active, not recruiting

ASTX727 and Donor Lymphocyte Infusions After Allogenic Stem Cell Transplantation in Very High Risk MDS or AML Patients

AML | MDS

France
Boehringer Ingelheim

Not yet recruiting

Brightline-3: A Study to Find Out Whether Brigimadlin in Combination With Ezabenlimab Helps People With Advanced Soft Tissue Sarcoma

Advanced Soft Tissue Sarcoma | Undifferentiated Pleomorphic Sarcoma (UPS) | Myxofibrosarcoma (MFS)
Rigshospitalet, Denmark

Recruiting

Innate Donor Effector Allogeneic Lymphocyte Infusion After Stem Cell Transplantation: the IDEAL Trial (IDEAL)

AML/MDS

Denmark
GWT-TUD GmbH

Recruiting

Comparison of Therapies Before Stem Cell Transplantation in Patients With Higher Risk MDS and Oligoblastic AML (PALOMA)

AML | MDS

Germany, Austria
University of Texas Southwestern Medical Center

Withdrawn

Effects of Growth Hormone in Chronically Ill Children

HIV Infections | Cerebral Palsy | Crohn Disease | Hurler Syndrome | Juvenile Rheumatoid Arthritis

United States
Joanne Kurtzberg, MD
The Marcus Foundation

Recruiting

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (DUOC-01)

Mucopolysaccharidosis II | Brain Diseases, Metabolic, Inborn | Adrenoleukodystrophy | Batten Disease | Leukodystrophy, Metachromatic | Leukodystrophy, Globoid Cell | Alpha-Mannosidosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | Neimann Pick Disease | Sanfilippo Mucopolysaccharidoses

United States
Montefiore Medical Center

Recruiting

Metabolically Optimized, Non-cytotoxic Low Dose Weekly Decitabine/Venetoclax in MDS and AML

AML | MDS | CMML

United States
M.D. Anderson Cancer Center

Recruiting

A Phase II Study of Vibecotamab (XmAb14045) for MRD- Positive AML and MDS After Hypomethylating Agent Failure

AML | MDS

United States
University of Florence

Active, not recruiting

Pilot Study of CC 486 (Oral Azacitidine) Plus BSC as Maintenance After sc Azacitidine in Elderly HR-IPSS-R MDS Patients (FLO_CC-486-)

MDS | CMML

Italy
PhenoSolve, LLC
Jacobi Medical Center; Boston Clinical Trials

Completed

Prevalence of Lidocaine Ineffectiveness in Adults With and Without ADHD

ADHD | PMS

United States
Northwest Women's and Children's Hospital, Xi'an...

Unknown

Gonadotrophin Releasing Hormone Agonist Addition for Luteal Support in In-vitro Fertilization and Embryo Transfer Cycles

GnRHa;LPS
Tehran University of Medical Sciences

Unknown

Evaluation of an Oral Contraceptive Containing 30 mg Ethinyloestradiol and 150 mg Desogestrel on Women's Health

Women With PMS

Iran, Islamic Republic of
Novartis Pharmaceuticals

Completed

Efficacy and Safety of Enteric-coated Mycophenolate Sodium in Heart Transplant Recipients

Patients Successfully Completing the 12-month Treatment Period of the Core Study (de Novo Heart Recipients) Who Were Interested of Being Treated With EC-MPS
Assiut University

Not yet recruiting

Accuracy And Safety of Trans Pedicular Screw Fixation of The Sub-axial Cervical Spine Using A Triad- Dependent Technique

Pedicle Screw | CPS
Masonic Cancer Center, University of Minnesota

Terminated

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Sphingolipidoses | Niemann-Pick Disease, Type C | Krabbe Disease | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Hurler's Syndrome | Maroteaux-Lamy Syndrome | Wolman's Disease | Niemann-Pick Disease Type B

United States
Memorial Sloan Kettering Cancer Center

Recruiting

PREPARE: Patient Centered Perioperative Experience

Minimally Invasive Surgery (MIS) Hysterectomy

United States
Astellas Pharma Global Development, Inc.
Basilea Pharmaceutica International Ltd

Completed

A Study to Assess the Effect of Multiple Doses of Isavuconazole on the Pharmacokinetics of a Single Dose of Mycophenolate Mofetil in Healthy Adult Subjects

Healthy Subjects | Pharmacokinetics of Isavuconazole | Pharmacokinetics of Plasma Mycophenolic Acid (MPA) | Pharmacokinetics of Plasma Phenolic Glucuronide of MPA (MPAG)

United States
Shalvata Mental Health Center

Completed

A Study on the Effect of Methylphenidate on Creativity of Healthy Adults

MPH Influence on Creativity
Duke University

Terminated

Myeloablative Allogeneic Stem Cell Transplantation Using a Naive T-Cell Depleted Peripheral Blood Stem Cell Graft

MDS | NHL | ALL | ANLL
SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt)

Recruiting

Registry Gangliosidoses

Gangliosidoses | Galactosialidosis | Sialidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Morquio B Disease | Gm2-Gangliosidosis, Variant B1 | GM2 Activator Deficiency

Germany
Center for International Blood and Marrow Transplant...
Cellular Dynamics International, Inc. - A FUJIFILM Company

Terminated

Generation of an Induced Pluripotent Stem (iPS) Cell Bank Immune Matched to a Majority of the US Population

iPS Cell Manufacture and Banking
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Maroteaux-Lamy Disease (BioMaroteaux) (BioMaroteaux)

Lung Diseases | Eye Abnormalities | Lysosomal Storage Disease | Macroglossia | Obstructive Sleep Apnoea

Germany, India, Sri Lanka, Egypt
Bellicum Pharmaceuticals

No longer available

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

Hurler Syndrome | Inborn Errors of Metabolism | Metachromatic Leukodystrophy | Inherited Metabolic Disorder | Lysosomal Storage Disorder

United States

Clinical Trials on MPS - Mucopolysaccharidosis

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Medical Conditions

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CROs in Guatemala

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