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"congenital neuronal ceroid lipofuscinosis" OR "Neuronal Ceroid-Lipofuscinoses"
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"congenital neuronal ceroid lipofuscinosis" OR "Neuronal Ceroid-Lipofuscinoses" (25 records)
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1 | NCT01399047 | Completed Has Results |
Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis |
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Interventional | Phase 2 |
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Other |
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19 | All | 6 Years to 25 Years (Child, Adult) | NCT01399047 | 3908 | JUMP | July 2011 | November 2015 | November 2015 | July 21, 2011 | January 23, 2017 | January 23, 2017 |
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2 | NCT01966757 | Completed | Neuronal Ceroid Lipofuscinosis and Associated Sleep Abnormalities |
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Observational |
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Other |
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57 | All | Child, Adult, Senior | NCT01966757 | IRB13-00376 | July 2013 | July 2016 | July 2016 | October 22, 2013 | July 26, 2016 |
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3 | NCT03307304 | Recruiting | Investigations of Juvenile Neuronal Ceroid Lipofuscinosis |
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Observational |
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NIH |
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50 | All | Child, Adult, Senior | NCT03307304 | 180002 18-CH-0002 |
November 27, 2017 | December 31, 2030 | December 31, 2030 | October 11, 2017 | April 17, 2018 |
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4 | NCT01238315 | Withdrawn | Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis |
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Interventional | Phase 1 |
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Industry |
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0 | All | 6 Months to 6 Years (Child) | NCT01238315 | CL-N03-NCL | November 2010 | April 2011 | April 2011 | November 10, 2010 | January 15, 2015 |
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5 | NCT01873924 | Recruiting | Clinical and Neuropsychological Investigations in Batten Disease |
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Observational |
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Other |
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400 | All | 1 Year to 40 Years (Child, Adult) | NCT01873924 | Batten Study | August 2004 | July 2020 | July 2020 | June 10, 2013 | October 25, 2017 |
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6 | NCT00337636 | Completed | Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL) |
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Interventional | Phase 1 |
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Industry |
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6 | All | 18 Months to 12 Years (Child) | NCT00337636 | CL-N001-05 | May 2006 | February 2009 | September 2009 | June 16, 2006 | January 15, 2015 |
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7 | NCT01035424 | Active, not recruiting | Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis |
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Observational |
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Other / NIH |
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75 | All | 2 Years to 18 Years (Child, Adult) | NCT01035424 | 0901010186 U54NS065768 R01NS061848 5R01NS061848-04 LDN 6716 |
March 2010 | April 2020 | April 2025 | December 18, 2009 | December 14, 2017 |
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8 | NCT01161576 | Active, not recruiting | Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) |
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Interventional | Phase 1 |
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Other / NIH |
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25 | All | 2 Years to 18 Years (Child, Adult) | NCT01161576 | 0810010013 UL1TR000457-06 |
August 2010 | December 2017 | August 2032 | July 13, 2010 | December 26, 2017 |
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9 | NCT00151216 | Active, not recruiting | Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis |
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Interventional | Phase 1 |
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Other |
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10 | All | 3 Years to 18 Years (Child, Adult) | NCT00151216 | 0401007010 OBA-RAC 0312-619 |
June 2004 | June 2019 | June 2019 | September 8, 2005 | May 24, 2017 |
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10 | NCT01414985 | Active, not recruiting | AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis |
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Interventional | Phase 1 Phase 2 |
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Other |
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8 | All | 3 Years to 18 Years (Child, Adult) | NCT01414985 | 1005011054 IND 13951 |
April 15, 2010 | December 2020 | December 2022 | August 11, 2011 | January 9, 2018 |
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11 | NCT01907087 | Completed | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease |
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Interventional | Phase 1 Phase 2 |
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Industry |
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24 | All | 3 Years to 16 Years (Child) | NCT01907087 | 190-201 | September 2013 | November 2015 | March 2016 | July 24, 2013 | April 27, 2016 |
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12 | NCT03285425 | Recruiting | Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae |
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Observational |
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Other |
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15 | All | 2 Years to 25 Years (Child, Adult) | NCT03285425 | IRB16-00554 | Batten'sCLN6 | January 2017 | January 2020 | January 2020 | September 18, 2017 | September 18, 2017 |
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13 | NCT02725580 | Recruiting | Batten CLN6 Gene Therapy |
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Interventional | Phase 1 Phase 2 |
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Other |
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12 | All | 1 Year and older (Child, Adult, Senior) | NCT02725580 | IRB15-00963 | March 2016 | March 2019 | March 2019 | April 1, 2016 | January 23, 2018 |
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14 | NCT00028262 | Completed Has Results |
Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis |
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Interventional | Phase 4 |
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NIH |
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10 | All | 6 Months to 3 Years (Child) | NCT00028262 | 010086 01-CH-0086 |
February 2001 | November 2013 | November 2013 | December 18, 2001 | October 27, 2016 | January 21, 2015 |
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15 | NCT00151268 | Completed | Genotype - Phenotype Correlations of LINCL |
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Observational |
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Other |
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18 | All | 2 Years to 18 Years (Child, Adult) | NCT00151268 | 0401007011 | March 2004 | September 2009 | September 2009 | September 8, 2005 | July 29, 2016 | |||||
16 | NCT01698229 | Terminated | Collection of Cerebrospinal Fluid in Healthy Children |
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Observational |
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Other |
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32 | All | 2 Years to 8 Years (Child) | NCT01698229 | 1111012024 | December 2011 | December 2015 | December 2015 | October 2, 2012 | May 4, 2016 |
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17 | NCT02485899 | Active, not recruiting | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease |
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Interventional | Phase 1 Phase 2 |
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Industry |
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23 | All | 3 Years to 16 Years (Child) | NCT02485899 | 190-202 | February 2015 | July 2020 | January 2021 | June 30, 2015 | November 21, 2017 |
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18 | NCT02678689 | Enrolling by invitation | A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease |
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Interventional | Phase 2 |
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Industry |
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15 | All | up to 17 Years (Child) | NCT02678689 | 190-203 | February 2016 | July 2022 | January 2023 | February 10, 2016 | November 21, 2017 |
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19 | 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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20 | 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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21 | 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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22 | NCT02435940 | Recruiting | Inherited Retinal Degenerative Disease Registry |
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Observational |
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Other |
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20000 | All | Child, Adult, Senior | NCT02435940 | FFB-Registry-01 | June 2014 | June 2037 | June 2037 | May 6, 2015 | September 14, 2017 |
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23 | 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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24 | 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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25 | NCT01793168 | Recruiting | Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford |
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Observational |
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Other |
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20000 | All | Child, Adult, Senior | NCT01793168 | 03-10-014 Hypersomnia Foundation National Ataxia Foundation 4p- Support Group CdLS Foundation Hyperacusis Research Limited Kabuki Syndrome Network Kawasaki Disease Foundation Klippel-Feil Syndrome Freedom Leiomyosarcoma Direct Research MSS Support Group ML4 Foundation Stickler Involved People IWSA Soft Bones PWN4PWN aHUS Klippel-Feil Syndrome Alliance American MEN Support Kleine-Levin Syndrome All Things Kabuki WSS Foundation BIVA ABDA PROS Foundation (HLH) |
CoRDS | July 2010 | December 2100 | December 2100 | February 15, 2013 | October 2, 2017 |
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† Study has passed its completion date and status has not been verified in more than two years.