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Results for “Mucopolysaccharidosis IH”

37 trials

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Showing 20 of 37 results

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
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
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
Not applicableWithdrawnNCT02298712
What this trial is testing

Biomarker for Hurler Disease (BioHurler)

Who this might be right for
Mucopolysaccharidosis Type IGargoylismMetabolism, Inborn Errors
CENTOGENE GmbH Rostock
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
Testing effectiveness (Phase 2)Study completedNCT03513328
What this trial is testing

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

Who this might be right for
Bone Marrow Failure SyndromeThalassemiaSickle Cell Disease+13 more
University of Florida 6
Not applicableEnded earlyNCT01572636
What this trial is testing

Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

Who this might be right for
Mucopolysaccharidosis Type IHMPS IHurler Syndrome
Masonic Cancer Center, University of Minnesota 20
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
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
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
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
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
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
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
Testing effectiveness (Phase 2)WithdrawnNCT02715505
What this trial is testing

Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)

Who this might be right for
Inherited Metabolic Disorders IMD
Novartis Pharmaceuticals
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
Early research (Phase 1)Looking for participantsNCT05682144
What this trial is testing

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

Who this might be right for
Mucopolysaccharidosis IH/SMucopolysaccharidosis IS
Immusoft of CA, Inc. 11
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)Active Not RecruitingNCT02171104
What this trial is testing

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Who this might be right for
Mucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more
Masonic Cancer Center, University of Minnesota 149
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
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