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Results for “Sanfilippo Syndrome Type B”

22 trials

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Showing 20 of 22 results

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
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 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 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 applicableWithdrawnNCT02298686
What this trial is testing

Biomarker for Sanfilippo Type A-B-C-D Disease (BioSanfilippo)

Who this might be right for
Mental RetardationDevelopmental Delay
CENTOGENE GmbH Rostock
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
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
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
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 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 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
Not applicableStudy completedNCT02493998
What this trial is testing

Mucopolysaccharidosis Type IIIB (MPS IIIB)

Who this might be right for
Mucopolysaccharidosis Type IIIBMucopolysaccharidosis Type 3 BMPS III B+1 more
Allievex Corporation 22
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07640984
What this trial is testing

A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)

Who this might be right for
Mucopolysaccharidosis IIIB
JCR Pharmaceuticals Co., Ltd. 12
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 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)Ended earlyNCT03315182
What this trial is testing

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB

Who this might be right for
Mucopolysaccharidosis Type 3 B
Abeona Therapeutics, Inc 11
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)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
Large-scale testing (Phase 3)Not Yet RecruitingNCT07579910
What this trial is testing

Intracerebroventricular Tralesinidase Alfa in Children With Mucopolysaccharidosis Type IIIB

Who this might be right for
MPS IIIB
Spruce Biosciences 14
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
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