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Results for “Sanfilippo Syndrome (MPS III)”

49 trials

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Showing 20 of 49 results

Not applicableStudy completedNCT01586871
What this trial is testing

Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network

Who this might be right for
Mucopolysaccharidoses
University of Minnesota 1,147
Large-scale testing (Phase 3)Enrolling By InvitationNCT04360265
What this trial is testing

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

Who this might be right for
Mucopolysaccharidosis IIIAMPS IIIASanfilippo Syndrome+1 more
Ultragenyx Pharmaceutical Inc 41
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
Large-scale testing (Phase 3)UnknownNCT03612869
What this trial is testing

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

Who this might be right for
Mucopolysaccharidosis Type IIIA
LYSOGENE 20
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
Not applicableStudy completedNCT01938014
What this trial is testing

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Who this might be right for
Mucopolysaccharidosis Type I (MPS I)Mucopolysaccharidosis Type II (MPS II)Mucopolysaccharidosis Type III (MPS III)+2 more
University of Chicago 19
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 applicableStudy completedNCT05523206
What this trial is testing

A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)

Who this might be right for
Mucopolysaccharidosis III-A
Sanguine Biosciences 15
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)Active Not RecruitingNCT06095388
What this trial is testing

Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

Who this might be right for
Mucopolysaccharidosis III-A
JCR Pharmaceuticals Co., Ltd. 15
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
Testing effectiveness (Phase 2)Study completedNCT00383448
What this trial is testing

HSCT for High Risk Inherited Inborn Errors

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more
Masonic Cancer Center, University of Minnesota 38
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
Testing effectiveness (Phase 2)Ended earlyNCT04088734
What this trial is testing

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

Who this might be right for
MPS IIIASanfilippo SyndromeSanfilippo A+1 more
Ultragenyx Pharmaceutical Inc 5
Not applicableNot Yet RecruitingNCT07712003
What this trial is testing

Natural History to Assess Disease in Patients With MPS IIIC

Who this might be right for
Sanfilippo Syndrome Type CMucopolysaccharidosis (MPS) IIIC
Phoenix Nest 35
Early research (Phase 1)Active Not RecruitingNCT06567769
What this trial is testing

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Who this might be right for
Sanfilippo Syndrome Type A
GC Biopharma Corp 9
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)Ended earlyNCT02350816
What this trial is testing

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

Who this might be right for
Sanfilippo SyndromeMucopolysaccharidosis (MPS)
Shire 17
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