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Recruiting, Not yet recruiting, Available Studies | "Niemann-Pick Diseases"
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Recruiting, Not yet recruiting, Available Studies | "Niemann-Pick Diseases" (13 records)
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1 | NCT03471143 | Recruiting | Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C |
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Interventional | Phase 1 Phase 2 |
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Other / NIH |
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12 | All | up to 6 Months (Child) | NCT03471143 | 201708114 1U01HD090845-01 |
March 2018 | February 2022 | February 2022 | March 20, 2018 | March 27, 2018 |
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2 | NCT01306604 | Recruiting | Biomarker for Niemann Pick Type C Disease |
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Observational |
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Other / Industry |
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80 | All | Child, Adult, Senior | NCT01306604 | BN04/2011 | BioNPC | March 2011 | September 2018 | October 2018 | March 2, 2011 | May 4, 2017 |
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3 | NCT00344331 | Recruiting | Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C |
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Observational |
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NIH |
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300 | All | Child, Adult, Senior | NCT00344331 | 060186 06-CH-0186 |
June 19, 2006 | June 26, 2006 | April 4, 2018 |
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4 | NCT02292654 | Recruiting | Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency |
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Interventional | Phase 1 Phase 2 |
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Industry |
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20 | All | up to 17 Years (Child) | NCT02292654 | DFI13803 U1111-1160-6469 |
ASCEND-Peds | May 1, 2015 | June 2019 | September 2019 | November 17, 2014 | April 12, 2018 |
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5 | NCT02004691 | Recruiting | Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency |
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Interventional | Phase 2 Phase 3 |
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Industry |
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36 | All | 18 Years and older (Adult, Senior) | NCT02004691 | DFI12712 2015‐000371‐26 U1111-1142-5963 |
ASCEND | December 18, 2015 | January 4, 2024 | January 4, 2024 | December 9, 2013 | April 12, 2018 |
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6 | NCT02939547 | Recruiting | Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1) |
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Interventional | Phase 1 |
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Industry |
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12 | All | 18 Years and older (Adult, Senior) | NCT02939547 | CTD-TCNPC-101 | October 11, 2017 | February 2019 | February 2019 | October 20, 2016 | March 23, 2018 |
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7 | NCT02912793 | Recruiting | Study of Pharmacokinetics and Preliminary Efficacy in Patients With Niemann-Pick C1 |
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Interventional | Phase 1 Phase 2 |
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Industry |
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12 | All | 2 Years and older (Child, Adult, Senior) | NCT02912793 | CTD-TCNP-201 | March 20, 2017 | August 2019 | August 2019 | September 23, 2016 | April 17, 2018 |
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8 | NCT03201627 | Recruiting | Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease |
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Interventional | Phase 1 Phase 2 |
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Other |
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7 | All | 7 Years to 40 Years (Child, Adult) | NCT03201627 | XH-17-010 | July 1, 2017 | August 30, 2018 | December 30, 2018 | June 28, 2017 | June 28, 2017 |
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9 | NCT02120235 | Recruiting | Investigating Lysosomal Storage Diseases in Minority Groups |
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Observational |
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Other |
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20000 | All | up to 100 Years (Child, Adult, Senior) | NCT02120235 | 14-CFCT-11 | February 2014 | February 2018 | December 2018 | April 22, 2014 | April 4, 2017 |
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10 | NCT03333200 | Recruiting | Longitudinal Study of Neurodegenerative Disorders |
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Observational |
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Other |
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1500 | All | Child, Adult, Senior | NCT03333200 | PRO11050036 | January 11, 2012 | January 2030 | January 2035 | November 6, 2017 | February 1, 2018 |
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11 | NCT02014246 | Recruiting | Genetic Characterization of Movement Disorders and Dementias |
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Observational |
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NIH |
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12000 | All | 18 Years and older (Adult, Senior) | NCT02014246 | 999903329 03-AG-N329 |
February 12, 2003 | December 31, 2018 | December 31, 2018 | December 18, 2013 | April 6, 2018 |
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12 | NCT02254863 | Recruiting | UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells |
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Interventional | Phase 1 |
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Other |
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12 | All | up to 22 Years (Child, Adult) | NCT02254863 | Pro00050198 | DUOC-01 | September 2014 | September 2018 | September 2018 | October 2, 2014 | March 16, 2018 |
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13 | NCT02000310 | Recruiting | Molecular and Cellular Mechanisms of Lysosomal Storage Diseases |
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Observational |
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Other |
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80 | All | up to 100 Years (Child, Adult, Senior) | NCT02000310 | 13-CFCT-07 | November 2013 | December 2017 | March 2018 | December 4, 2013 | September 28, 2016 |
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† Study has passed its completion date and status has not been verified in more than two years.