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Results for “Sanfilippo Syndrome”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT02053064
What this trial is testing

Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

Who this might be right for
Mucopolysaccharidosis Type III ASanfilippo Disease Type A
LYSOGENE 4
Testing effectiveness (Phase 2)Study completedNCT01155778
What this trial is testing

Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients

Who this might be right for
Mucopolysaccharidosis (MPS)
Shire 12
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
Large-scale testing (Phase 3)Looking for participantsNCT06333041
What this trial is testing

Study of Cannabidiol in Sanfilippo Syndrome

Who this might be right for
Sanfilippo SyndromeMucopolysaccharidosis III
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 35
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 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
Testing effectiveness (Phase 2)Ended earlyNCT01372228
What this trial is testing

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Who this might be right for
Hurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)+9 more
Talaris Therapeutics Inc. 3
Not applicableStudy completedNCT05648851
What this trial is testing

A Natural History Study of Sanfilippo Syndrome Type D

Who this might be right for
Sanfilippo Syndrome Type D
Phoenix Nest 10
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 completedNCT01474343
What this trial is testing

Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

Who this might be right for
Mucopolysaccharidosis Type III ASanfilippo Disease Type A
LYSOGENE 4
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 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
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)Ended earlyNCT01299727
What this trial is testing

Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Who this might be right for
Sanfilippo Syndrome
Shire 12
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 applicableUnknownNCT04918641
What this trial is testing

Natural History Observational Study of MPS IIIa in SMC

Who this might be right for
Sanfilippo Syndrome A
Samsung Medical Center 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
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
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)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
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