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Results for “Mucopolysaccharidosis IH”

38 trials

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Showing 20 of 38 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 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 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 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
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
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 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
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT03488394
What this trial is testing

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant

Who this might be right for
Mucopolysaccharidosis IH
Orchard Therapeutics 8
Testing effectiveness (Phase 2)Temporarily pausedNCT03580083
What this trial is testing

RGX-111 Gene Therapy in Patients With MPS I

Who this might be right for
Mucopolysaccharidosis Type I (MPS I)Hurler SyndromeHurler-Scheie Syndrome
REGENXBIO Inc. 21
Testing effectiveness (Phase 2)Study completedNCT03053089
What this trial is testing

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

Who this might be right for
Mucopolysaccharidosis I
ArmaGen, Inc 21
Testing effectiveness (Phase 2)Study completedNCT00730314
What this trial is testing

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Who this might be right for
Sickle Cell DiseaseThalassemiaAnemia+9 more
Children's Hospital Los Angeles 25
Early research (Phase 1)Study completedNCT01917708
What this trial is testing

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Who this might be right for
Hurler SyndromeFanconi AnemiaGlanzmann Thrombasthenia+12 more
Emory University 10
Not applicableStudy completedNCT01873911
What this trial is testing

Neurobehavioral Phenotypes in MPS III

Who this might be right for
Sanfilippo Syndrome Type ASanfilippo Syndrome Type BHurler Syndrome
University of Minnesota 30
Testing effectiveness (Phase 2)Study completedNCT00176917
What this trial is testing

Stem Cell Transplantation for Hurler

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIMannosidosis+1 more
Masonic Cancer Center, University of Minnesota 41
Testing effectiveness (Phase 2)Study completedNCT00146757
What this trial is testing

A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old

Who this might be right for
Mucopolysaccharidosis IHurler SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 20
Not applicableStudy completedNCT01870375
What this trial is testing

Longitudinal Studies of Brain Structure and Function in MPS Disorders

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type VI+2 more
University of Minnesota 100
Not applicableLooking for participantsNCT05619900
What this trial is testing

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more
University of California, San Francisco 250
Not applicableActive Not RecruitingNCT05634512
What this trial is testing

Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

Who this might be right for
Hematopoietic Cell TransplantationMucopolysaccharidosis Type I
Masonic Cancer Center, University of Minnesota 13
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