35 studies found for:    "Mucopolysaccharidosis type I"
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Rank Status Study
21 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
22 Recruiting Human Placental-Derived Stem Cell Transplantation
Conditions: Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Adrenoleukodystrophy;   Niemann-Pick Disease;   Metachromatic Leukodystrophy;   Wolman Disease;   Krabbe's Disease;   Gaucher's Disease;   Fucosidosis;   Batten Disease;   Severe Aplastic Anemia;   Diamond-Blackfan Anemia;   Amegakaryocytic Thrombocytopenia;   Myelodysplastic Syndrome;   Acute Myelogenous Leukemia;   Acute Lymphocytic Leukemia
Intervention: Drug: Human Placental Derived Stem Cell
23 Recruiting Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
Conditions: Severe Combined Immunodeficiency;   Malignancy, Hematologic;   Neuroblastoma;   Neoplasm;   Mucopolysaccharidosis I
Intervention: Procedure: Venipuncture
24 Completed Stem Cell Transplant w/Laronidase for Hurler
Conditions: Mucopolysaccharidosis I;   Hurler Syndrome
Interventions: Procedure: Stem Cell Transplant;   Drug: Laronidase ERT
25 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
26 Active, not recruiting Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
Conditions: Hurler Syndrome (MPS I);   Hurler-Scheie Syndrome With Early Neurologic Involvement and/or Sensitization to Enzyme Replacement Therapy (ERT);   Hunter Syndrome (MPS II);   Sanfilippo Syndrome (MPS III);   Krabbe Disease (Globoid Leukodystrophy);   Metachromatic Leukodystrophy (MLD);   Adrenoleukodystrophy (ALD and AMN);   Sandhoff Disease;   Tay Sachs Disease;   Pelizaeus Merzbacher (PMD);   Niemann-Pick Disease;   Alpha-mannosidosis
Intervention: Biological: Enriched Hematopoetic Stem Cell Transplantation/novel platform technology
27 Active, not recruiting Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network
Conditions: MPS I;   MPS II;   MPS VI;   Mucopolysaccharidoses
Intervention:
28 Recruiting Intrathecal Enzyme Replacement for Hurler Syndrome
Condition: Hurler Syndrome
Intervention: Drug: IRT Laronidase
29 Enrolling by invitation Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis
Conditions: MPS I;   MPS IV
Intervention:
30 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
31 Recruiting Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
Condition: Hurler Syndrome
Intervention: Drug: Laronidase
32 Recruiting Longitudinal Study of Bone Disease in Children With Mucopolysaccharidoses (MPS) I, II, and VI
Condition: Mucopolysaccharidoses
Intervention:
33 Terminated ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Conditions: Inherited Metabolic Diseases;   Lysosomal Storage Disorders;   Peroxisomal Storage Diseases;   Inborn Errors of Metabolism;   Mucopolysaccharidosis
Intervention: Biological: ALD-101
34 Recruiting Reduced Intensity Conditioning in Patients Aged ≤30 With Non-Malignant Disorders Undergoing Cord Blood Transplantation
Conditions: Primary Immunodeficiency Syndromes;   Congenital Bone Marrow Failure Syndromes;   Inherited Metabolic Disorders (IMD);   Hereditary Anemias;   Patients With Sickle Disease Presenting Specific Symptoms
Interventions: Drug: Hydroxyurea;   Drug: Alemtuzumab;   Drug: Fludarabine;   Drug: Melphalan;   Drug: Thiotepa
35 Recruiting Phase I Dose Escalation of Oral BAY1161909 in Combination With Intravenous Paclitaxel
Condition: Medical Oncology
Interventions: Drug: BAY1161909;   Drug: Paclitaxel

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Indicates status has not been verified in more than two years