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Results for “MPS III”

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Showing 20 of 0 results

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
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
Large-scale testing (Phase 3)Study completedNCT00069641
What this trial is testing

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

Who this might be right for
Mucopolysaccharidosis II
Shire 96
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
Large-scale testing (Phase 3)Ended earlyNCT00748969
What this trial is testing

Clinical Trial of Growth Hormone in MPS I, II, and VI

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis VI
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 2
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
Not applicableStudy completedNCT02315014
What this trial is testing

Daily Protein Intake Distribution and MPS (ARCIII)

Who this might be right for
Overweight
Société des Produits Nestlé (SPN) 20
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 completedNCT01594294
What this trial is testing

An Evaluation of Marketed Contact Lens Disinfectant Systems on Eyelid Tissues

Who this might be right for
Myopia
Alcon Research 100
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
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
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 applicableActive Not RecruitingNCT03655223
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
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