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36 studies found for:    HURLER SYNDROME
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Rank Status Study
1 Active, not recruiting Intrathecal Enzyme Replacement for Hurler Syndrome
Condition: Hurler Syndrome
Intervention: Drug: IRT Laronidase
2 Recruiting Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
Conditions: Mucopolysaccharidosis Type IH;   MPS I;   Hurler Syndrome
Intervention: Drug: Laronidase
3 Active, not recruiting Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
Condition: Hurler Syndrome
Intervention: Drug: Laronidase
4 Completed Stem Cell Transplant w/Laronidase for Hurler
Conditions: Mucopolysaccharidosis I;   Hurler Syndrome
Interventions: Procedure: Stem Cell Transplant;   Drug: Laronidase ERT
5 Not yet recruiting Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)
Condition: Hurler Syndrome, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Krabbe, Cerebral Adrenoleukodystrophy
Intervention: Drug: Umbilical cord blood transplantation with HSC835
6 Recruiting Biomarker for Patients With Hurler Disease or High-grade Suspicion for Hurler Disease
Conditions: Mucopolysaccharidosis Type I;   MPS I;   Hurler-Scheie Syndrome;   Gargoylism, Hurler Syndrome
Intervention:
7 Completed Neurobehavioral Phenotypes in MPS III
Conditions: Sanfilippo Syndrome Type A;   Sanfilippo Syndrome Type B;   Hurler Syndrome
Intervention:
8 Completed
Has Results
Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I
Conditions: Mucopolysaccharidosis I;   Hurlers Syndrome;   Hurler-Scheie Syndrome
Interventions: Biological: rhIDU (recombinant human-Alpha-L-Iduronidase);   Biological: Placebo
9 Completed
Has Results
Stem Cell Transplantation for Hurler
Conditions: Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Mannosidosis;   Mucolipidosis Type II (I-cell Disease)
Interventions: Procedure: Stem Cell Transplant;   Drug: Busulfan, Cyclophosphamide, ATG
10 Recruiting Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I
Condition: Mucopolysaccharidosis I
Intervention: Drug: AGT-181 (HIRMAb-IDUA)
11 Completed
Has Results
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
Conditions: Mucopolysaccharidosis I;   Hurler Syndrome;   Hurler-Scheie Syndrome;   Scheie Syndrome
Intervention: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
12 Completed
Has Results
A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
Conditions: Mucopolysaccharidosis I;   Hurler Syndrome;   Hurler-Scheie Syndrome;   Scheie Syndrome
Intervention: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
13 Completed
Has Results
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
Conditions: Mucopolysaccharidosis I;   Hurler's Syndrome;   Hurler-Scheie Syndrome;   Scheie Syndrome
Interventions: Biological: Aldurazyme;   Biological: placebo
14 Completed
Has Results
A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
Conditions: Mucopolysaccharidosis I;   Hurler's Syndrome;   Hurler-Scheie Syndrome;   Scheie Syndrome
Intervention: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
15 Completed A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
Conditions: Mucopolysaccharidosis I;   Hurler's Syndrome;   Hurler-Scheie Syndrome;   Scheie's Syndrome
Intervention: Drug: laronidase
16 Recruiting A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
Conditions: Mucopolysaccharidosis I;   Hurler's Syndrome;   Hurler-Scheie Syndrome;   Scheie
Intervention: Biological: Aldurazyme (laronidase)
17 Unknown  Effects of Growth Hormone in Chronically Ill Children
Conditions: - Hurler Syndrome (MPS-1) With Short Stature and Muscle Wasting;   - Cerebral Palsy With Muscle Wasting;   - Juvenile Rheumatoid Arthritis With Muscle Wasting and Short Stature;   - Crohn’s Disease;   - HIV Infection.
Interventions: Drug: Growth Hormone;   Procedure: Whole body Protein turnover;   Procedure: DEXA scan
18 Completed
Has Results
Immune Tolerance Study With Aldurazyme® (Laronidase)
Condition: Mucopolysaccharidosis I
Interventions: Biological: Laronidase;   Drug: Cyclosporine A (CsA);   Drug: Azathioprine (Aza)
19 Recruiting MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Conditions: Mucopolysaccharidosis I;   Mucopolysaccharidosis II;   Mucopolysaccharidosis VI;   Mucopolysaccharidosis VII;   Hurler Syndrome;   Hunter Syndrome;   Maroteaux Lamy Syndrome;   Sly Syndrome;   Glycoprotein Metabolic Disorders;   Alpha Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Adrenoleukodystrophy;   Peroxisomal Disorders;   Osteopetrosis;   Sphingolipidosis;   Gangliosidosis;   Globoid Cell Leukodystrophy;   Metachromatic Leukodystrophy;   Niemann Pick B;   Niemann Pick C Subtype 2;   I-cell Disease
Interventions: Procedure: blood stem cell transplant;   Drug: Rabbit Anti-Thymocyte Globulin (ATG);   Drug: Fludarabine;   Drug: Busulfan;   Drug: Cyclophosphamide;   Drug: Cyclosporine A (CSA);   Drug: Methylprednisolone;   Drug: Mycophenolate Mofetil (MMF);   Drug: Granulocyte-Colony Stimulating Factor (G-CSF);   Drug: Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF);   Drug: N-acetylcysteine;   Drug: Celecoxib;   Drug: Vitamin E;   Drug: Alpha Lipoic Acid
20 Recruiting BMT Abatacept for Non-Malignant Diseases
Conditions: Hurler Syndrome;   Fanconi Anemia;   Glanzmann Thrombasthenia;   Wiskott-Aldrich Syndrome;   Chronic Granulomatous Disease;   Severe Congenital Neutropenia;   Leukocyte Adhesion Deficiency;   Shwachman-Diamond Syndrome;   Diamond-Blackfan Anemia;   Dyskeratosis-congenita;   Chediak-Higashi Syndrome;   Severe Aplastic Anemia;   Thalassemia Major;   Hemophagocytic Lymphohistiocytosis;   Sickle Cell Disease
Intervention: Drug: Abatacept

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Study has passed its completion date and status has not been verified in more than two years.