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Results for “Mucopolysaccharidosis Type 3 B”

23 trials

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Showing 20 of 23 results

Testing effectiveness (Phase 2)Study completedNCT02324049
What this trial is testing

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

Who this might be right for
Mucopolysaccharidosis IIIB
Alexion Pharmaceuticals, Inc. 11
Testing effectiveness (Phase 2)UnknownNCT03784287
What this trial is testing

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

Who this might be right for
Mucopolysaccharidosis Type IIIBMPS III B
Allievex Corporation 20
Not applicableStudy completedNCT01509768
What this trial is testing

Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

Who this might be right for
Sanfilippo Syndrome Type B
Shire 19
Testing effectiveness (Phase 2)Ended earlyNCT02618512
What this trial is testing

A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

Who this might be right for
Mucopolysaccharidosis III, Type B (MPS IIIB)Sanfilippo B
Alexion Pharmaceuticals, Inc. 3
Testing effectiveness (Phase 2)Study completedNCT03300453
What this trial is testing

Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

Who this might be right for
Sanfilippo Syndrome B
UniQure Biopharma B.V. 4
Testing effectiveness (Phase 2)Study completedNCT02754076
What this trial is testing

A Treatment Study of Mucopolysaccharidosis Type IIIB

Who this might be right for
MPS III BMucopolysaccharidosis Type IIIB
Allievex Corporation 23
Not applicableStudy completedNCT02037880
What this trial is testing

Natural History Studies of Mucopolysaccharidosis III

Who this might be right for
Mucopolysaccharidosis Type IIIAMucopolysaccharidosis Type IIIB
Nationwide Children's Hospital 25
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
Not applicableStudy completedNCT02293382
What this trial is testing

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

Who this might be right for
MPS IIIB (Sanfilippo Syndrome)
Alexion Pharmaceuticals, Inc. 30
Not applicableStudy completedNCT01873911
What this trial is testing

Neurobehavioral Phenotypes in MPS III

Who this might be right for
Sanfilippo Syndrome Type ASanfilippo Syndrome Type BHurler Syndrome
University of Minnesota 30
Not applicableUnknownNCT03227042
What this trial is testing

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Who this might be right for
Mucopolysaccharidosis Type IIIB
Allievex Corporation 44
Not applicableUnknownNCT04320329
What this trial is testing

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

Who this might be right for
Morquio B DiseaseGM1 Gangliosidosis Type III
University of British Columbia 30
Not applicableEnded earlyNCT02293408
What this trial is testing

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

Who this might be right for
MPS IIIB (Sanfilippo B Syndrome)
Alexion Pharmaceuticals, Inc. 30
Not applicableStudy completedNCT02090179
What this trial is testing

Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging

Who this might be right for
MPS IIIB (Sanfilippo B Syndrome)
Alexion Pharmaceuticals, Inc. 5
Testing effectiveness (Phase 2)Looking for participantsNCT06488924
What this trial is testing

An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB

Who this might be right for
Mucopolysaccharidosis III-B
JCR Pharmaceuticals Co., Ltd. 10
Not applicableEnded earlyNCT04655911
What this trial is testing

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

Who this might be right for
Mucopolysaccharidosis III-B
Abeona Therapeutics, Inc 1
Large-scale testing (Phase 3)Ended earlyNCT00654433
What this trial is testing

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

Who this might be right for
Inherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more
Aldagen 40
Post-approval studies (Phase 4)Enrolling By InvitationNCT05492799
What this trial is testing

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

Who this might be right for
MPS III B
Allievex Corporation 15
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