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Results for “Hurler Disease”

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Showing 20 of 0 results

Not applicableStudy completedNCT01521429
What this trial is testing

Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI

Who this might be right for
Mucopolysaccharidoses
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 55
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
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 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
Large-scale testing (Phase 3)Study completedNCT00258011
What this trial is testing

Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease

Who this might be right for
Mucopolysaccharidosis IHurler SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 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 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
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
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
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)Ended earlyNCT02702115
What this trial is testing

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

Who this might be right for
MPS I
Sangamo Therapeutics 3
Not applicableWithdrawnNCT02298647
What this trial is testing

Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)

Who this might be right for
Hepato-splenomegalyDysostosis MultiplexSeizures+1 more
CENTOGENE GmbH Rostock
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
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 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)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 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 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
Testing effectiveness (Phase 2)Study completedNCT00176891
What this trial is testing

Stem Cell Transplant w/Laronidase for Hurler

Who this might be right for
Mucopolysaccharidosis IHurler Syndrome
Masonic Cancer Center, University of Minnesota 25
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
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