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Results for “MPS VII”

19 trials

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Showing 19 of 19 results

Large-scale testing (Phase 3)Study completedNCT02432144
What this trial is testing

UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Who this might be right for
Sly SyndromeMPS VIIMucopolysaccharidosis+1 more
Ultragenyx Pharmaceutical Inc 12
Large-scale testing (Phase 3)Study completedNCT02230566
What this trial is testing

UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Who this might be right for
MPS 7Sly SyndromeMucopolysaccharidosis+1 more
Ultragenyx Pharmaceutical Inc 12
Not applicableLooking for participantsNCT06036693
What this trial is testing

MPS (RaDiCo Cohort) (RaDiCo-MPS)

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis III+5 more
Institut National de la Santé Et de la Recherche Médicale, France 1,000
Testing effectiveness (Phase 2)Ended earlyNCT00668564
What this trial is testing

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

Who this might be right for
Hurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more
Masonic Cancer Center, University of Minnesota 18
Not applicableLooking for participantsNCT05619900
What this trial is testing

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more
University of California, San Francisco 250
Not applicableAvailableNCT03775174
What this trial is testing

Expanded Access to Mepsevii

Who this might be right for
MPS VIIMucopolysaccharidosis VIISly Syndrome
Ultragenyx Pharmaceutical Inc
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104
What this trial is testing

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

Who this might be right for
Mucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more
Masonic Cancer Center, University of Minnesota 149
Early research (Phase 1)Looking for participantsNCT04532047
What this trial is testing

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Who this might be right for
MPS IMPS IIMPS IVA+6 more
University of California, San Francisco 10
Testing effectiveness (Phase 2)Study completedNCT01856218
What this trial is testing

An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Who this might be right for
Mucopolysaccharidosis Type 7
Ultragenyx Pharmaceutical Inc 3
Testing effectiveness (Phase 2)Study completedNCT01043640
What this trial is testing

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Who this might be right for
MucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more
Masonic Cancer Center, University of Minnesota 46
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableEnrolling By InvitationNCT05368038
What this trial is testing

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Who this might be right for
Acid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more
Albert Einstein College of Medicine 100,000
Testing effectiveness (Phase 2)Study completedNCT02418455
What this trial is testing

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

Who this might be right for
Sly SyndromeMPS VIIMucopolysaccharidosis+1 more
Ultragenyx Pharmaceutical Inc 8
Not applicableStudy completedNCT01870375
What this trial is testing

Longitudinal Studies of Brain Structure and Function in MPS Disorders

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type VI+2 more
University of Minnesota 100
Not applicableLooking for participantsNCT03604835
What this trial is testing

Mucopolysaccharidosis VII Disease Monitoring Program

Who this might be right for
Mucopolysaccharidosis VIIMPS VIIMPS 7+1 more
Ultragenyx Pharmaceutical Inc 50
Not applicableNo Longer AvailableNCT02097251
What this trial is testing

An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

Who this might be right for
Mucopolysaccharidosis Type 7
Joyce Fox
Not applicableStudy completedNCT02537730
What this trial is testing

BIOCLEAN MPS (Multi-Purpose Solution) VII in Combination With Biofinity Lens: A Two Week Crossover Study

Who this might be right for
Myopia
CooperVision, Inc. 33
Not applicableWithdrawnNCT02298699
What this trial is testing

Biomarker for Sly Disease (MPS VII) (BioSly)

Who this might be right for
Developmental DelaySkeletal AbnormalitiesHepatomegaly+1 more
CENTOGENE GmbH Rostock