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 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.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02396459June 20198 April 2019Triac Trial II in MCT8 PatientsEffects 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 IIAllan-Herndon-Dudley SyndromeDrug: TriacErasmus Medical CenterNot recruitingN/A18 YearsMale30Phase 2Netherlands
2JPRN-jRCTs03118034801/04/20182 April 2019Effectiveness of Curcumin for PMDEffectiveness of Curcumin (an active integrant in turmeric) for Pelizaeus-Merzbacher disease: an exploratory study - Effectiveness of Curcumin for PMDPelizaeus-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 InoueRecruiting520Male10N/ANone
3NCT02060474October 201429 April 2019Thyroid Hormone Analog Therapy in MCT8 Deficiency: Triac Trial PatientsThyroid Hormone Analog Therapy of Patients With Severe Psychomotor Retardation Caused by Mutations in the MCT8 Thyroid Hormone Transporter: The Triac Trial.Allan-Herndon-Dudley SyndromeDrug: TriacErasmus Medical CenterZonMw: The Netherlands Organisation for Health Research and DevelopmentNot recruitingN/A99 YearsMale46Phase 2Netherlands
4NCT01391637June 201123 May 2016Long-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;PMDBiological: HuCNS-SC transplant in the lead-in phaseStemCells, Inc.Not recruitingN/AN/AMale4Phase 1United States
5NCT01005004November 200919 February 2015Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) SubjectsPhase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD)Pelizaeus-Merzbacher DiseaseBiological: HuCNS-SC cellsStemCells, Inc.Not recruiting6 Months5 YearsMale4Phase 1United States

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