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Results for “Mucopolysaccharidosis IIIB”

20 trials

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Showing 20 of 20 results

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
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
Not applicableAvailableNCT07733856
What this trial is testing

Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)

Who this might be right for
Mucopolysaccharidosis Type IIIBMPS IIIB (Sanfilippo B Syndrome)
Spruce Biosciences
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 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
Large-scale testing (Phase 3)Not Yet RecruitingNCT07579910
What this trial is testing

Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)

Who this might be right for
MPS IIIB
Spruce Biosciences 14
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 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 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 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
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)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 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
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07818759
What this trial is testing

Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB

Who this might be right for
Sanfilippo Syndrome (MPS III)
NeuroGT 9
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
Testing effectiveness (Phase 2)Looking for participantsNCT07640984
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)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)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
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