139. 先天性大脳白質形成不全症 ［臨床試験数：5，薬物数：4（DrugBank：1），標的遺伝子数：0，標的パスウェイ数：0］
Searched query = "Congenital cerebral hypomyelination", "Congenital cerebral white matter aplasia", "Pelizaeus Merzbacher disease", "Pelizaeus Merzbacher like disease 1", "Pelizaeus Merzbacher like disease type 1", "Hypomyelination with atrophy of the basal ganglia and cerebellum", "18q syndrome", "Chromosome 18q deletion syndrome", "Allan Herndon Dudley syndrome", "Mitochondrial Hsp60 chaperonopathy", "Salla disease", "Diffuse cerebral hypomyelination with cerebellar atrophy and hypoplasia of the corpus callosum", "Hypomyelination and congenital cataract", "Ataxia, delayed dentition, and hypomyelination", "Peripheral demyelinating neuropathy", "Central dysmyelinating leukodystrophy", "Waardenburg syndrome", "Hirschsprung disease"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02396459 | June 2019 | 8 April 2019 | Triac Trial II in MCT8 Patients | Effects of the Thyroid Hormone Analog Triac on the Neurocognitive Phenotype in Patients With Severe Psychomotor Retardation Caused by Mutations in the MCT8 Thyroid Hormone Transporter: The Triac Trial II | Allan-Herndon-Dudley Syndrome | Drug: Triac | Erasmus Medical Center | Not recruiting | N/A | 18 Years | Male | 30 | Phase 2 | Netherlands | |
| 2 | JPRN-jRCTs031180348 | 01/04/2018 | 2 April 2019 | Effectiveness of Curcumin for PMD | Effectiveness of Curcumin (an active integrant in turmeric) for Pelizaeus-Merzbacher disease: an exploratory study - Effectiveness of Curcumin for PMD | Pelizaeus-Merzbacher disease Neurology/Pediatrics;D020371;Neurology/Pediatrics | Low dose curcumin (1mg/kg/day, oral, once a day) for 2 months followed by high dose curcumin (4mg/kg/day, oral, once a day) for 10 months. | Ken Inoue | Recruiting | 5 | 20 | Male | 10 | N/A | None | |
| 3 | NCT02060474 | October 2014 | 29 April 2019 | Thyroid Hormone Analog Therapy in MCT8 Deficiency: Triac Trial Patients | Thyroid Hormone Analog Therapy of Patients With Severe Psychomotor Retardation Caused by Mutations in the MCT8 Thyroid Hormone Transporter: The Triac Trial. | Allan-Herndon-Dudley Syndrome | Drug: Triac | Erasmus Medical Center | ZonMw: The Netherlands Organisation for Health Research and Development | Not recruiting | N/A | 99 Years | Male | 46 | Phase 2 | Netherlands |
| 4 | NCT01391637 | June 2011 | 23 May 2016 | Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD) | Long-Term Follow-Up Safety and Preliminary Efficacy Study of Human Central Nervous System Stem Cell (HuCNS-SC®) Transplantation in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD) | Pelizaeus-Merzbacher Disease;PMD | Biological: HuCNS-SC transplant in the lead-in phase | StemCells, Inc. | Not recruiting | N/A | N/A | Male | 4 | Phase 1 | United States | |
| 5 | NCT01005004 | November 2009 | 19 February 2015 | Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects | Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD) | Pelizaeus-Merzbacher Disease | Biological: HuCNS-SC cells | StemCells, Inc. | Not recruiting | 6 Months | 5 Years | Male | 4 | Phase 1 | United States |