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Results for “Mucopolysaccharidosis Type I”

97 trials

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Showing 20 of 97 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
Testing effectiveness (Phase 2)Study completedNCT01043640
What this trial is testing

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Who this might be right for
MucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more
Masonic Cancer Center, University of Minnesota 46
Not applicableStudy completedNCT00004738
What this trial is testing

Genetic Analysis of the Chiari I Malformation

Who this might be right for
SyringomyeliaType I Arnold Chiari Malformation
National Institute of Neurological Disorders and Stroke (NINDS) 152
Testing effectiveness (Phase 2)Active Not RecruitingNCT03153319
What this trial is testing

Study to Evaluate the Safety and Efficacy of Adalimumab 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 14
Early research (Phase 1)Ended earlyNCT00786968
What this trial is testing

Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

Who this might be right for
Spinal Cord CompressionMucopolysaccharidosis IHurler-Scheie Syndrome+2 more
Patricia I. Dickson, M.D. 3
Early research (Phase 1)Study completedNCT00692926
What this trial is testing

Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells

Who this might be right for
MDSAnemia, AplasticInborn Errors of Metabolism+2 more
Joanne Kurtzberg, MD 37
Early research (Phase 1)Study completedNCT01173016
What this trial is testing

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

Who this might be right for
Hurler Syndrome
Masonic Cancer Center, University of Minnesota 11
Not applicableStudy completedNCT03576729
What this trial is testing

MRS to Determine Neuroinflammation and Oxidative Stress in MPS I

Who this might be right for
Mucopolysaccharidosis Type I
University of Minnesota 30
Post-approval studies (Phase 4)Study completedNCT00144768
What this trial is testing

A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 25
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 completedNCT02067650
What this trial is testing

Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis

Who this might be right for
MPS IMPS IV
Children's Hospital of Eastern Ontario 18
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
Large-scale testing (Phase 3)Active Not RecruitingNCT06149403
What this trial is testing

Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

Who this might be right for
MPS-IH (Hurler Syndrome)
Orchard Therapeutics 41
Early research (Phase 1)Study completedNCT02597114
What this trial is testing

Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181

Who this might be right for
Mucopolysaccharidosis I
ArmaGen, Inc 3
Not applicableStudy completedNCT05073783
What this trial is testing

Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting

Who this might be right for
Pompe DiseaseMucopolysaccharidosis Type I (MPS I)
Sanofi 57
Testing effectiveness (Phase 2)Study completedNCT04227600
What this trial is testing

JR-171 in Patients With Mucopolysaccharidosis I

Who this might be right for
Mucopolysaccharidosis I
JCR Pharmaceuticals Co., Ltd. 18
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
Not applicableLooking for participantsNCT04528355
What this trial is testing

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+2 more
Paul Szabolcs 50
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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