99 studies found for:    "mucopolysaccharidosis type IV"
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Rank Status Study
21 Available Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant
Conditions: Acute Lymphoblastic Leukemia;   Acute Myeloid Leukemia;   Chronic Myeloid Leukemia;   Myelodysplastic Syndrome;   Lymphomas;   Bone Marrow Failure;   Hemoglobinopathy;   Immune Deficiency;   Osteopetrosis;   Cytopenias;   White Blood Cell Abnormalities;   Red Blood Cell Abnormalities
Intervention: Biological: CD34+ enriched, T Cell Depleted donor stem cell product
22 Completed Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial
Condition: Rubinstein-Taybi Syndrome
Interventions: Drug: sodium valproate;   Drug: Placebo
23 Enrolling by invitation Immunodeficiency in Cartilage-hair Hypoplasia: Sub-project on Safety of Vaccination Against Chickenpox
Condition: Cartilage-hair Hypoplasia
Intervention: Biological: Varilrix
24 Active, not recruiting BMN 110 Phase 3B in Australian Patients
Condition: Mucopolysaccharidosis IVA (Morquio A Syndrome)
Intervention: Drug: BMN 110
25 Completed Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Intervention: Drug: BPS804
26 Completed Marrow Mesenchymal Cell Therapy for Osteogenesis Imperfecta: A Pilot Study
Condition: Osteogenesis Imperfecta
Intervention: Biological: Bone marrow transplant
27 Completed Zoledronic Acid in Children (1 -17 Years) With Severe Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Intervention: Drug: Zoledronic Acid
28 Terminated
Has Results
Stem Cell Transplantation for Children Affected With Osteopetrosis
Condition: Osteopetrosis
Interventions: Procedure: Stem Cell Transplantation;   Device: Miltenyi Biotec CliniMACS;   Drug: Systemic chemotherapy and antibodies
29 Completed Efficacy and Safety of Alendronate in Chinese Children or Adolescents With Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Intervention: Drug: Alendronate
30 Completed Studies on Abnormal Bone From Patients With Polyostotic Fibrous Dysplasia and McCune Albright Syndrome
Condition: Polyostotic Fibrous Dysplasia
Intervention:
31 Terminated A Two Centre Study to Assess the Stability and Long-term Performance of the C-Stem™ AMT in a Total Primary Hip Replacement
Conditions: Rheumatoid Arthritis;   Osteoarthritis;   Post-traumatic Arthritis;   Collagen Disorders;   Avascular Necrosis;   Traumatic Femoral Fractures;   Nonunion of Femoral Fractures;   Congenital Hip Dysplasia;   Slipped Capital Femoral Epiphysis.
Intervention: Device: C-Stem™ AMT Femoral Component (standard and high off-set variants)
32 Recruiting The Effect of Treatment With Teriparatide and Zoledronic Acid in Patients With Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
Interventions: Drug: Zoledronic acid;   Drug: Teriparatide;   Drug: placebo zoledronic acid;   Drug: placebo teriparatide
33 Enrolling by invitation A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
Condition: Achondroplasia
Intervention:
34 Terminated
Has Results
Reduced Intensity AlloTransplant For Osteopetrosis
Condition: Osteopetrosis
Interventions: Procedure: Stem Cell or Umbilical Cord Blood Transplantation;   Drug: Campath, Busulfan, Clofarabine;   Procedure: Total Lymphoid Irradiation
35 Completed Treatment of Severe Osteogenesis Imperfecta by Allogeneic Bone Marrow Transplantation
Condition: Osteogenesis Imperfecta
Interventions: Other: Bone Marrow Cell Transplantation;   Radiation: Irradiation, Total Body;   Drug: Cyclophosphamide;   Drug: Cyclosporin;   Procedure: Mesenchymal Stem Cell Transplantation;   Drug: Busulfan
36 Terminated A Multi-centre Study to Assess the Long-term Performance of the Summit™ Hip in Primary Total Hip Replacement
Conditions: Rheumatoid Arthritis;   Osteoarthritis;   Post-traumatic Arthritis;   Collagen Disorders;   Avascular Necrosis;   Traumatic Femoral Fractures;   Nonunion of Femoral Fractures;   Congenital Hip Dysplasia;   Slipped Capital Femoral Epiphysis
Intervention: Device: Summit Tapered Hip System
37 Terminated A Randomised Study to Compare Metal Ion Release and Long-term Performance of the Pinnacle™ Cup With a Ceramic-on-Metal or a Metal-on-Metal Bearing
Conditions: Osteoarthritis;   Post-traumatic Arthritis;   Collagen Disorders;   Avascular Necrosis;   Congenital Hip Dysplasia;   Slipped Capital Femoral Epiphysis
Intervention: Device: Pinnacle™ Acetabular System
38 Recruiting Evaluation of Skeletal Maturity for Slipped Capital Femoral Epiphysis
Condition: Slipped Capital Femoral Epiphysis
Intervention: Radiation: Radiographic skeletal age assessment
39 Completed Issues Surrounding Prenatal Genetic Testing for Achondroplasia
Conditions: Achondroplasia;   Dwarfism
Intervention:
40 Terminated Multi-Centre Study to Assess the Long-term Performance of the DePuy ASR™ System in Resurfacing and Primary Total Hip Replacement
Conditions: Rheumatoid Arthritis;   Osteoarthritis;   Post-traumatic Arthritis;   Collagen Disorders;   Avascular Necrosis;   Nonunion of Femoral Fractures;   Congenital Hip Dysplasia;   Slipped Capital Femoral Epiphysis
Interventions: Device: DePuy ASR™ Hip System;   Device: DePuy ASR™ XL Head / ASR™ Acetabular Cup System

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Indicates status has not been verified in more than two years