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Results for “Hurler-Scheie Syndrome”

22 trials

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Showing 20 of 22 results

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
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
Not applicableStudy completedNCT00852358
What this trial is testing

Intrathecal Enzyme Therapy for Cognitive Decline in MPS I

Who this might be right for
Cognitive DeclineMucopolysaccharidosis IHurler-Scheie Syndrome+2 more
Patricia I. Dickson, M.D. 9
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)WithdrawnNCT05665036
What this trial is testing

Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy

Who this might be right for
MPS IMPS1-SMPS1-HS
Sigilon Therapeutics, Inc.
Large-scale testing (Phase 3)Study completedNCT00912925
What this trial is testing

Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

Who this might be right for
Mucopolysaccharidosis IHurlers SyndromeHurler-Scheie Syndrome
Genzyme, a Sanofi Company 45
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 applicableStudy completedNCT00144794
What this trial is testing

Mucopolysaccharidosis I (MPS I) Registry

Who this might be right for
Mucopolysaccharidosis I (MPS I)
Genzyme, a Sanofi Company 1,406
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)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
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
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
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)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
Post-approval studies (Phase 4)Study completedNCT00144781
What this trial is testing

A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 34
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
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 applicableEnded earlyNCT02095015
What this trial is testing

Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)

Who this might be right for
Mucopolysaccharidosis (MPS)
Shire 159
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
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