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"cholesteryl ester storage disease" OR "Lipid Metabolism, Inborn Errors"
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"cholesteryl ester storage disease" OR "Lipid Metabolism, Inborn Errors" (571 records)
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1 | NCT02372513 | Completed | National Lysosomal Acid Lipase Deficiency Study |
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Observational |
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Other / Industry |
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810 | All | 3 Months to 18 Years (Child, Adult) | NCT02372513 | LALD-TR | LAL-D | January 2015 | January 31, 2017 | March 1, 2017 | February 26, 2015 | November 13, 2017 |
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2 | NCT01528917 | Completed | An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype |
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Observational |
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Industry |
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49 | All | 5 Years and older (Child, Adult, Senior) | NCT01528917 | LAL-2-NH01 | June 2011 | January 2013 | May 2013 | February 8, 2012 | July 20, 2016 |
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3 | NCT02112994 | Completed | Safety and Efficacy Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency |
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Interventional | Not Applicable |
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Industry |
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31 | All | 8 Months and older (Child, Adult, Senior) | NCT02112994 | LAL-CL06 2011-004287-30 |
May 2014 | December 2017 | December 2017 | April 14, 2014 | February 14, 2018 |
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4 | NCT02376751 | No longer available | An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency |
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Expanded Access |
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Industry | All | 8 Months and older (Child, Adult, Senior) | NCT02376751 | LAL-EA01 | March 3, 2015 | June 8, 2016 | ||||||||||
5 | 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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6 | 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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7 | NCT01358370 | Completed | A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype |
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Observational |
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Industry |
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40 | All | Child, Adult, Senior | NCT01358370 | LAL-1-NH01 | November 2010 | January 2013 | March 2013 | May 23, 2011 | June 27, 2016 |
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8 | NCT02926872 | Terminated | Screening for Lysosomal Acid Lipase Deficiency |
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Observational |
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Industry |
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22 | All | 2 Years to 16 Years (Child) | NCT02926872 | ALX-LAL-502 | November 2016 | June 5, 2017 | June 5, 2017 | October 6, 2016 | June 16, 2017 |
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9 | 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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10 | NCT01716728 | Unknown † | Identification of Undiagnosed Lysosomal Acid Lipase Deficiency |
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Interventional | Not Applicable |
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Other / Industry |
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60 | All | Child, Adult, Senior | NCT01716728 | 2012P000418 | August 2012 | August 2014 | October 30, 2012 | August 13, 2013 |
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11 | NCT01757184 | Active, not recruiting Has Results |
A Multicenter Study of SBC-102 (Sebelipase Alfa) in Patients With Lysosomal Acid Lipase Deficiency/ ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) |
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Interventional | Phase 3 |
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Industry |
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66 | All | 4 Years and older (Child, Adult, Senior) | NCT01757184 | LAL-CL02 | January 2013 | January 2019 | January 2019 | December 28, 2012 | March 20, 2017 | April 18, 2016 |
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12 | NCT01488097 | Active, not recruiting Has Results |
Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency |
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Interventional | Phase 2 |
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Industry |
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8 | All | 18 Years to 65 Years (Adult) | NCT01488097 | LAL-CL04 | November 2011 | March 2014 | June 2017 | December 8, 2011 | March 6, 2017 | May 9, 2016 |
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13 | NCT01307098 | Completed | Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Patients With Lysosomal Acid Lipase Deficiency |
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Interventional | Phase 1 |
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Industry |
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9 | All | 18 Years to 65 Years (Adult) | NCT01307098 | LAL-CL01 | May 2011 | January 2012 | January 2012 | March 2, 2011 | July 27, 2016 |
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14 | NCT02383641 | Recruiting | Biomarker for Wolman Disease |
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Observational |
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Other / Industry |
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100 | All | 2 Months and older (Child, Adult, Senior) | NCT02383641 | BWD 04-2015 | BioWolman | March 2015 | March 2020 | March 2020 | March 9, 2015 | July 5, 2017 |
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15 | NCT02707211 | Unknown † | Anti-oxLDL IgM Antibodies as a Novel Therapy for Metabolic Lipid Diseases |
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Interventional | Early Phase 1 |
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Other |
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20 | All | 2 Years to 64 Years (Child, Adult) | NCT02707211 | Pfizer-Prevenar13-2015 | June 2016 | February 2017 | February 2017 | March 14, 2016 | March 15, 2016 |
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16 | NCT02345421 | Terminated | A Study to Identify and Characterize LAL-D Patients in High-risk Populations |
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Observational |
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Industry |
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640 | All | 2 Years and older (Child, Adult, Senior) | NCT02345421 | LAL-CSS01 | December 2014 | September 2015 | October 2015 | January 26, 2015 | May 24, 2016 |
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17 | NCT01884220 | Completed | Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up |
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Observational |
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Other / NIH |
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4 | All | Child, Adult, Senior | NCT01884220 | LDN6706 U54NS065768 |
November 2010 | May 2014 | May 2014 | June 21, 2013 | July 28, 2015 |
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18 | NCT02851550 | Recruiting | Assessement of the Prevalence of Lysosomal Acid Lipase Deficiency in Liver Post-transplant Patients |
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Observational |
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Other |
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100 | All | 18 Years and older (Adult, Senior) | NCT02851550 | 69HCL16_0485 | LALTH-2 | December 2015 | December 2017 | June 2018 | August 1, 2016 | August 1, 2016 |
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19 | NCT02852304 | Recruiting | Assessment of the Prevalence of Lysosomal Acid Lipase Deficiency in Patients Waiting for a Liver Transplant. |
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Observational |
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Other |
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100 | All | 18 Years and older (Adult, Senior) | NCT02852304 | 69HCL16_0428 | LALTH-1 | October 2015 | October 2017 | March 2018 | August 2, 2016 | August 2, 2016 |
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20 | NCT01791452 | Unknown † | Novel Association of Cholesterol Ester Storage Disease Due to Lysosomal Acid Lipase Deficiency and Non-Alcoholic Fatty Liver Disease: A Prospective Clinical Study |
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Observational |
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Other |
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All | 18 Years to 80 Years (Adult, Senior) | NCT01791452 | 0042-12-ZIV | February 2014 | February 15, 2013 | February 15, 2013 |
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21 | NCT00092898 | Completed | An Investigational Drug Study to Lower Non-Cholesterol Sterol Levels Associated With Sitosterolemia (0653-062)(COMPLETED) |
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Interventional | Phase 3 |
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Industry |
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30 | All | 18 Years to 85 Years (Adult, Senior) | NCT00092898 | 0653-062 MK0653-062 2004_038 |
October 2004 | September 2005 | October 2005 | September 28, 2004 | March 10, 2017 | |||
22 | NCT00092820 | Completed | Sitosterolemia Extension Study (0653-004)(COMPLETED) |
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Interventional | Phase 3 |
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Industry |
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58 | All | 10 Years and older (Child, Adult, Senior) | NCT00092820 | P02243 2004_033 |
February 12, 2001 | September 18, 2001 | November 30, 2004 | September 28, 2004 | May 11, 2017 | |||
23 | NCT00092807 | Completed | Sitosterolemia Extension Study (0653-003)(COMPLETED) |
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Interventional | Phase 3 |
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Industry |
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37 | All | 10 Years and older (Child, Adult, Senior) | NCT00092807 | P02257 2004_032 |
February 12, 2001 | September 18, 2001 | November 30, 2004 | September 28, 2004 | May 11, 2017 | |||
24 | NCT00092833 | Terminated | Investigational Drug in Patients With Hypercholesterolemia or in Patients With Sitosterolemia (0653-026)(COMPLETED) |
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Interventional | Phase 3 |
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Industry |
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49 | All | 8 Years and older (Child, Adult, Senior) | NCT00092833 | 0653-026 2004_034 |
July 2002 | May 2005 | May 2005 | September 28, 2004 | February 28, 2017 | |||
25 | NCT00004481 | Completed | Genetic Study of Sitosterolemia |
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Observational |
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NIH / Other |
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All | Child, Adult, Senior | NCT00004481 | NCRR-M01RR01070-0470 MUSC-HR-8022 |
November 1999 | October 19, 1999 | June 24, 2005 |
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26 | NCT01633489 | Recruiting | Lysosomal Acid Lipase (LAL) Deficiency Registry |
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Observational |
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Industry |
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1000 | All | Child, Adult, Senior | NCT01633489 | ALX-LALD-501 | ALX-LALD-501 | December 2012 | June 2029 | June 2029 | July 4, 2012 | March 2, 2018 |
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27 | NCT01782027 | Suspended | Mendelian Reverse Cholesterol Transport Study |
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Interventional | Not Applicable |
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Other |
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30 | All | 18 Years to 75 Years (Adult, Senior) | NCT01782027 | 815075 | October 2012 | January 2019 | January 2019 | February 1, 2013 | July 27, 2017 |
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28 | NCT00260299 | Unknown † | Dietary Cholesterol and Defects in Cholesterol Synthesis in Mevalonate Kinase Deficiency |
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Observational |
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Other |
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15 | All | Child, Adult, Senior | NCT00260299 | MKD dietary study | February 2005 | December 2012 | December 1, 2005 | March 23, 2012 |
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29 | 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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30 | NCT03198897 | Recruiting | Biomarker for Homozygous Familial Hypercholesterolemia |
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Observational |
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Other / Industry |
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20 | All | 2 Months and older (Child, Adult, Senior) | NCT03198897 | BHFH 04-2017 | BioHoFH | June 1, 2017 | June 2020 | July 2020 | June 26, 2017 | July 26, 2017 |
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31 | 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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32 | 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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33 | NCT00383448 | Completed | HSCT for High Risk Inherited Inborn Errors |
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Interventional | Phase 2 |
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Other |
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39 | All | up to 70 Years (Child, Adult, Senior) | NCT00383448 | MT2006-14 0606M87246 |
September 2006 | September 2014 | September 2014 | October 3, 2006 | April 24, 2015 |
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34 | 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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35 | NCT03393130 | Not yet recruiting | Perioperative Research Into Memory, Genomics in the Intensive Therapy Unit: Alzheimer's |
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Observational |
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Other |
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150 | All | 18 Years to 25 Years (Adult) | NCT03393130 | C&W17/059 | PRIMoGenITA | February 1, 2018 | February 1, 2019 | December 1, 2019 | January 8, 2018 | January 8, 2018 | |||
36 | NCT01916512 | Unknown † | Relationship Between CETP Deficiency and Atherosclerosis in Patients With Hyperalphalipoproteinemia |
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Observational |
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Other / Industry |
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1500 | All | 40 Years to 100 Years (Adult, Senior) | NCT01916512 | INFINITY | INFINITY | February 2012 | March 2016 | August 5, 2013 | April 15, 2015 |
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37 | NCT01948648 | Not yet recruiting | Effects of Fish Oil and Colesevelam |
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Interventional | Not Applicable |
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Other |
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16 | All | 12 Years and older (Child, Adult, Senior) | NCT01948648 | B2013:075 | October 2016 | December 2017 | December 2017 | September 23, 2013 | August 31, 2016 |
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38 | NCT01584206 | Unknown † | Sitosterolemia Metabolism |
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Interventional | Not Applicable |
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Other |
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8 | All | 16 Years to 99 Years (Child, Adult, Senior) | NCT01584206 | B2011:051 | STAIR7002 | April 2012 | August 2013 | December 2016 | April 24, 2012 | March 10, 2016 |
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39 | NCT02635269 | Recruiting | Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy |
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Interventional | Not Applicable |
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Other |
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128 | All | 15 Years to 80 Years (Child, Adult, Senior) | NCT02635269 | Metabolic Myopathy H-15015150 |
January 2016 | August 2020 | December 2020 | December 18, 2015 | September 1, 2017 |
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40 | NCT00694109 | Completed Has Results |
An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia |
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Interventional | Phase 3 |
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Industry |
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144 | All | 12 Years and older (Child, Adult, Senior) | NCT00694109 | 301012-CS6 2005-003450-10 |
April 2008 | September 2014 | September 2014 | June 10, 2008 | September 9, 2016 | December 21, 2015 |
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41 | NCT00607373 | Completed Has Results |
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia |
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Interventional | Phase 3 |
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Industry |
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51 | All | 12 Years and older (Child, Adult, Senior) | NCT00607373 | 301012CS5 2005-003449-15 |
RADICHOL 1 | July 2007 | March 2009 | March 2009 | February 5, 2008 | September 9, 2016 | March 21, 2013 |
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42 | NCT00477594 | Completed Has Results |
Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia |
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Interventional | Phase 2 |
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Industry |
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21 | All | 12 Years and older (Child, Adult, Senior) | NCT00477594 | 301012-CS17 2007-001024-12 |
May 2007 | March 2011 | July 2011 | May 24, 2007 | September 9, 2016 | April 5, 2013 |
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43 | NCT00362180 | Completed Has Results |
Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration |
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Interventional | Phase 2 |
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Industry |
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38 | All | 18 Years to 65 Years (Adult) | NCT00362180 | 301012-CS10 2005-005783-90 |
July 2006 | March 2010 | September 2010 | August 9, 2006 | October 21, 2016 | June 24, 2015 |
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44 | NCT02327364 | Enrolling by invitation | Natural History of Pearson Syndrome |
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Observational |
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Other / NIH |
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25 | All | Child, Adult, Senior | NCT02327364 | NAMDC7408 | March 2014 | September 2020 | September 2020 | December 30, 2014 | November 8, 2017 | |||||
45 | NCT02104336 | Terminated | Phase 2 Study of EPI-743 in Children With Pearson Syndrome |
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Interventional | Phase 2 |
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Industry |
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10 | All | up to 18 Years (Child, Adult) | NCT02104336 | EPI743-13-024 | August 2014 | November 2015 | February 2016 | April 4, 2014 | March 21, 2016 |
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46 | NCT00983788 | Completed | Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects |
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Interventional | Phase 2 |
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Other |
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12 | All | 18 Years to 70 Years (Adult, Senior) | NCT00983788 | EudraCT: 2008-006704-27 | Bezafibrate | October 2009 | September 2011 | September 2011 | September 24, 2009 | May 30, 2012 |
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47 | NCT02298673 | Recruiting | Biomarker for Mucolipidosis Disorder Type I, II, III or IV |
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Observational |
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Other / Industry |
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50 | All | 2 Months and older (Child, Adult, Senior) | NCT02298673 | BML 04-2014 | BioML | November 2014 | November 2019 | December 2019 | November 24, 2014 | February 6, 2018 |
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48 | NCT02416661 | Recruiting | LYSO-PROVE - Determine the Prognostic Value of Lyso-Gb1 for Monitoring the Progress of Gaucher Disease |
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Observational |
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Other / Industry |
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250 | All | 6 Months and older (Child, Adult, Senior) | NCT02416661 | LP 03-2015 | LYSO-PROVE | March 2015 | April 2019 | May 2019 | April 15, 2015 | May 5, 2017 |
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49 | NCT02918032 | Recruiting | International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases |
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Observational |
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Other |
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100 | All | Child, Adult, Senior | NCT02918032 | TRICVD1315 | January 2014 | December 2018 | October 2020 | September 28, 2016 | December 19, 2017 |
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50 | NCT02830763 | Enrolling by invitation | Clinical Study on the Safety of CNT-02 for TGCV and NLSD-M |
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Interventional | Not Applicable |
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Other |
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10 | All | 20 Years and older (Adult, Senior) | NCT02830763 | TRICVD1527 UMIN000023043 |
September 5, 2016 | May 2021 | May 2021 | July 13, 2016 | December 19, 2017 |
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† Study has passed its completion date and status has not been verified in more than two years.