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Results for “Mucopolysaccharidosis I”

106 trials

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Showing 20 of 106 results

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 completedNCT03161171
What this trial is testing

Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type III+2 more
University Hospital Heidelberg 34
Not applicableNo Longer AvailableNCT03639844
What this trial is testing

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

Who this might be right for
Hurler SyndromeInherited Metabolic DisorderLysosomal Storage Disorder+2 more
Bellicum Pharmaceuticals
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
Not applicableEnded earlyNCT04008849
What this trial is testing

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 3
Not applicableLooking for participantsNCT07361536
What this trial is testing

Cardiac Structure and Function in MPS

Who this might be right for
MPS IMPS IV AMPS - Mucopolysaccharidosis+1 more
Children's Hospital of Orange County 240
Not applicableWithdrawnNCT00286689
What this trial is testing

Effects of Growth Hormone in Chronically Ill Children

Who this might be right for
Hurler SyndromeCerebral PalsyJuvenile Rheumatoid Arthritis+2 more
University of Texas Southwestern Medical Center
Large-scale testing (Phase 3)Study completedNCT00146770
What this trial is testing

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 45
Testing effectiveness (Phase 2)Ended earlyNCT00668564
What this trial is testing

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Who this might be right for
Hurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more
Masonic Cancer Center, University of Minnesota 18
Testing effectiveness (Phase 2)Study completedNCT03411161
What this trial is testing

S 81694 Plus Paclitaxel in Metastatic Breast Cancer

Who this might be right for
Metastatic Breast CancerMetastatic Triple Negative Breast Cancer
Institut de Recherches Internationales Servier 22
Early research (Phase 1)Study completedNCT02371226
What this trial is testing

Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I

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)Active Not RecruitingNCT05665166
What this trial is testing

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II

Who this might be right for
Mucopolysaccharidosis II
University of Manchester 5
Testing effectiveness (Phase 2)Study completedNCT00741338
What this trial is testing

Immune Tolerance Study With Aldurazyme® (Laronidase)

Who this might be right for
Mucopolysaccharidosis I
Genzyme, a Sanofi Company 7
Testing effectiveness (Phase 2)Study completedNCT03128593
What this trial is testing

JR-141 in Patients With Mucopolysaccharidosis Type II

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 14
Testing effectiveness (Phase 2)Study completedNCT03406962
What this trial is testing

MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 8
Testing effectiveness (Phase 2)Study completedNCT00004454
What this trial is testing

Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)

Who this might be right for
Mucopolysaccharidosis II
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 2
Early research (Phase 1)Study completedNCT00638547
What this trial is testing

Intrathecal Enzyme Replacement for Hurler Syndrome

Who this might be right for
Hurler Syndrome
Masonic Cancer Center, University of Minnesota 26
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
Post-approval studies (Phase 4)Ended earlyNCT00418821
What this trial is testing

The Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 2
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