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Niemann-Pick Disease
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Niemann-Pick Disease (38 records)
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1 | NCT02124083 | Completed Has Results |
Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1 |
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Interventional | Phase 1 Phase 2 |
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NIH / Other |
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12 | All | 18 Years to 60 Years (Adult) | NCT02124083 | 140102 14-CH-0102 |
April 25, 2014 | December 13, 2016 | December 13, 2016 | April 28, 2014 | February 22, 2018 | February 22, 2018 |
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2 | NCT01899950 | Completed | Longitudinal Study of Cognition With Niemann-Pick Disease, Type C |
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Observational |
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Other / NIH |
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70 | All | 2 Years to 99 Years (Child, Adult, Senior) | NCT01899950 | 11-003868 U54NS065768 LDN6715 |
NPC | March 2011 | September 2014 | September 2014 | July 16, 2013 | January 6, 2016 |
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3 | 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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4 | NCT02612129 | Active, not recruiting | Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C |
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Interventional | Phase 2 Phase 3 |
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Industry |
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50 | All | 2 Years to 18 Years (Child, Adult) | NCT02612129 | CT-ORZY-NPC-002 | June 2016 | June 2018 | June 2020 | November 23, 2015 | July 11, 2017 |
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5 | NCT00975689 | Completed Has Results |
Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine |
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Interventional | Phase 1 Phase 2 |
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NIH / Other |
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35 | All | 12 Months and older (Child, Adult, Senior) | NCT00975689 | 090185 09-CH-0185 |
August 2009 | November 2010 | November 2010 | September 11, 2009 | May 6, 2013 | July 12, 2012 |
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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 | NCT02841358 | Terminated | Screening of Niemann-Pick Disease, Type C in a Psychiatric Population |
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Interventional | Not Applicable |
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Other |
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22 | All | 18 Years and older (Adult, Senior) | NCT02841358 | 38RC13.200 | NPCPsy | December 2013 | June 2016 | December 2016 | July 22, 2016 | January 13, 2017 |
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8 | NCT02435030 | Completed | A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C |
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Observational |
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Industry |
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35 | All | 2 Years to 18 Years (Child, Adult) | NCT02435030 | CT-ORZY-NPC-001 2014-005194-37 |
September 2015 | May 2017 | May 2017 | May 6, 2015 | May 18, 2017 |
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9 | NCT01722526 | Completed | Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients |
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Interventional | Phase 1 |
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Industry |
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5 | All | 18 Years to 65 Years (Adult) | NCT01722526 | DFI13412 2012-003542-32 SPHINGO00812 |
March 2013 | January 2014 | January 2014 | November 7, 2012 | July 30, 2015 |
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10 | 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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11 | 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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12 | NCT00410566 | Terminated | Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) |
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Interventional | Phase 1 |
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Industry |
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11 | All | 18 Years to 65 Years (Adult) | NCT00410566 | SPHINGO00605 | December 2006 | March 2009 | April 2009 | December 13, 2006 | March 19, 2015 |
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13 | NCT00517153 | Completed | Miglustat in Niemann-Pick Type C Disease |
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Interventional | Phase 2 |
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Industry |
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29 | All | 4 Years and older (Child, Adult, Senior) | NCT00517153 | OGT-918-007 | January 2002 | September 2006 | January 2008 | August 16, 2007 | March 26, 2010 |
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14 | NCT02004704 | Enrolling by invitation | A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency |
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Interventional | Phase 2 |
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Industry |
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17 | All | 1 Year and older (Child, Adult, Senior) | NCT02004704 | LTS13632 2013-000051-40 U1111-1141-65868 |
December 4, 2013 | September 1, 2023 | September 1, 2023 | December 9, 2013 | April 4, 2018 |
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15 | 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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16 | 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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17 | 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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18 | 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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19 | NCT00316498 | Completed | Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease |
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Interventional | Phase 1 |
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NIH |
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30 | All | 12 Years and older (Child, Adult, Senior) | NCT00316498 | 030011 03-EI-0011 |
October 17, 2002 | August 15, 2007 | April 20, 2006 | July 2, 2017 |
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20 | NCT01747135 | Completed | Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease |
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Interventional | Phase 1 |
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Industry / NIH |
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14 | All | 2 Years to 25 Years (Child, Adult) | NCT01747135 | 130001 13-CH-0001 |
January 2013 | March 2017 | March 2017 | December 11, 2012 | April 19, 2017 |
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21 | NCT01760564 | Completed | Application of Miglustat in Patients With Niemann-Pick Type C |
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Interventional | Phase 3 |
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Other |
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5 | All | Child, Adult, Senior | NCT01760564 | 200802043M | January 2008 | December 2010 | December 2010 | January 4, 2013 | January 4, 2013 |
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22 | NCT02534844 | Active, not recruiting | Study of VTS-270 (2-hydroxypropyl-β-cyclodextrin) to Treat Niemann-Pick Type C1 (NPC1) Disease |
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Interventional | Phase 2 Phase 3 |
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Industry |
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51 | All | 4 Years to 21 Years (Child, Adult) | NCT02534844 | VTS301 | September 2015 | March 2017 | March 2018 | August 28, 2015 | July 25, 2017 |
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23 | NCT00001972 | Completed | PET Scan of Brain Metabolism in Relation to Age and Disease |
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Observational |
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NIH | 123 | All | Child, Adult, Senior | NCT00001972 | 940205 94-N-0205 |
September 1994 | August 2002 | January 19, 2000 | March 4, 2008 |
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24 | 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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25 | NCT00730314 | Completed | Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells |
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Interventional | Phase 1 Phase 2 |
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Other |
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25 | All | up to 21 Years (Child, Adult) | NCT00730314 | CCI #07-00119 CHLA-#07-00119 |
August 2008 | August 2015 | August 2015 | August 8, 2008 | June 23, 2016 |
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26 | NCT00005900 | Unknown † | Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation |
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Observational |
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Other |
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10 | All | Child, Adult, Senior | NCT00005900 | 199/15111 UMN-MT-1999-18 UMN-MT-9818 |
August 1999 | June 5, 2000 | June 24, 2005 |
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27 | NCT00668564 | Terminated Has Results |
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism |
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Interventional | Phase 2 |
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Other |
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18 | All | up to 21 Years (Child, Adult) | NCT00668564 | MT2008-02 0801M25202 |
March 2008 | February 2010 | February 2010 | April 29, 2008 | December 28, 2017 | July 13, 2011 |
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28 | 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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29 | 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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30 | NCT01586455 | Active, not recruiting | Human Placental-Derived Stem Cell Transplantation |
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Interventional | Phase 1 |
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Other |
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43 | All | up to 55 Years (Child, Adult) | NCT01586455 | NYMC 550 NYMC IRB L-10,733 |
HPDSC | April 2013 | December 2018 | December 2019 | April 26, 2012 | December 22, 2017 |
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31 | NCT01372228 | Active, not recruiting | Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders |
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Interventional | Phase 1 Phase 2 |
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Other |
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30 | All | Child, Adult, Senior | NCT01372228 | ICT-14070-010611 | April 2011 | April 2025 | April 2028 | June 13, 2011 | November 17, 2017 |
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32 | NCT00176904 | Completed Has Results |
Stem Cell Transplant for Inborn Errors of Metabolism |
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Interventional | Phase 2 Phase 3 |
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Other |
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135 | All | Child, Adult, Senior | NCT00176904 | MT1995-01 | January 1995 | June 2010 | June 2010 | September 15, 2005 | December 28, 2017 | June 10, 2011 |
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33 | 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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34 | 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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35 | NCT00654433 | Terminated | ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases |
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Interventional | Phase 3 |
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Industry |
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40 | All | up to 16 Years (Child) | NCT00654433 | UCBT-002 BB-IND#13502 |
UCBT-002 | March 2008 | July 2011 | November 2011 | April 8, 2008 | July 8, 2014 |
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36 | NCT01371825 | Active, not recruiting Has Results |
SurVival of Lysosomal Acid Lipase Deficiency (LAL-D) Infants Treated With SebelipAse aLfa |
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Interventional | Phase 2 Phase 3 |
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Industry |
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9 | All | up to 24 Months (Child) | NCT01371825 | LAL-CL03 | VITAL | May 2011 | August 2014 | April 2018 | June 13, 2011 | November 14, 2017 | April 18, 2016 |
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37 | NCT01473875 | Terminated | Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102 |
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Interventional | Phase 2 Phase 3 |
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Industry |
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10 | All | Child, Adult, Senior | NCT01473875 | LAL-CL05 | November 2011 | December 2014 | January 2015 | November 17, 2011 | February 20, 2017 |
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38 | NCT02193867 | Active, not recruiting | Clinical Trial in Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency |
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Interventional | Phase 2 |
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Industry |
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10 | All | up to 8 Months (Child) | NCT02193867 | LAL-CL08 | June 2014 | April 2018 | April 2018 | July 18, 2014 | September 6, 2017 |
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† Study has passed its completion date and status has not been verified in more than two years.