139. Congenital cerebral hypomyelination
10 clinical trials,   7 drugs   (DrugBank: 2 drugs),   2 drug target genes,   2 drug target pathways

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. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02396459
(ClinicalTrials.gov)
March 20207/3/2015Triac Trial II in MCT8 Deficiency PatientsTiratricol Treatment of Children With Monocarboxylate Transporter 8 Deficiency: Triac Trial IIAllan-Herndon-Dudley SyndromeDrug: TriacRare Thyroid Therapeutics International ABErasmus Medical CenterNot yet recruitingN/A30 MonthsMale18Phase 2Netherlands
2JPRN-jRCTs031180348
01/04/201818/03/2019Effectiveness of Curcumin for PMDEffectiveness 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
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.Inoue KenNULLComplete5age20ageMale10N/AJapan
3EUCTR2014-000178-20-CZ
(EUCTR)
04/02/201623/06/2015Therapy of MCT8 patients with the thyroid hormone analog Triac.Thyroid hormone analog therapy of patients with severe psychomotor retardation caused by mutations in the MCT8 thyroid hormone transporter: The Triac Trial. - Triac Trial in MCT8 patients This therapuetical trial will be conducted in patient with the Allan-Herndon-Dudley Syndrome (AHDS), casued by mutations in the thyroid hormone transporter MCT8. This results in the characteristic clinical phenotype of severe psychomotor retardation due to local hypothyroidism in the brain, in combination with high serum T3 and high normal serum TSH levels that lead to local hyperthyroidism in tissues that do not dependent on MCT8, resulting in tachycardia, low body weight and muscle wasting.;Therapeutic area: Diseases [C] - Hormonal diseases [C19]Trade Name: Téatrois (Tiratricol)
Product Name: Téatrois
Erasmus Medical CentreNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
40Phase 2Czech Republic;Belgium;Germany;Italy
4EUCTR2014-000178-20-IT
(EUCTR)
01/02/201616/10/2015Therapy of MCT8 patients with the thyroid hormone analog Triac.Thyroid hormone analog therapy of patients with severe psychomotor retardation caused by mutations in the MCT8 thyroid hormone transporter: The Triac Trial. - Triac Trial in MCT8 patients This therapuetical trial will be conducted in patient with the Allan-Herndon-Dudley Syndrome (AHDS), casued by mutations in the thyroid hormone transporter MCT8. This results in the characteristic clinical phenotype of severe psychomotor retardation due to local hypothyroidism in the brain, in combination with high serum T3 and high normal serum TSH levels that lead to local hyperthyroidism in tissues that do not dependent on MCT8, resulting in tachycardia, low body weight and muscle wasting.;Therapeutic area: Diseases [C] - Hormonal diseases [C19]Trade Name: Téatrois (Tiratricol)
Product Name: Téatrois
Erasmus Medical CentreNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
40Phase 2Germany;Italy
5EUCTR2014-000178-20-FR
(EUCTR)
14/01/201614/12/2015Therapy of MCT8 patients with the thyroid hormone analog Triac.Thyroid hormone analog therapy of patients with severe psychomotor retardation caused by mutations in the MCT8 thyroid hormone transporter: The Triac Trial. - Triac Trial in MCT8 patients This therapuetical trial will be conducted in patient with the Allan-Herndon-Dudley Syndrome (AHDS), casued by mutations in the thyroid hormone transporter MCT8. This results in the characteristic clinical phenotype of severe psychomotor retardation due to local hypothyroidism in the brain, in combination with high serum T3 and high normal serum TSH levels that lead to local hyperthyroidism in tissues that do not dependent on MCT8, resulting in tachycardia, low body weight and muscle wasting.;Therapeutic area: Diseases [C] - Hormonal diseases [C19]Trade Name: Téatrois (Tiratricol)
Product Name: Téatrois
Erasmus Medical CentreCentre Hospitalier de ToulouseNot RecruitingFemale: no
Male: yes
40Phase 2France;Czech Republic;Canada;Belgium;Australia;South Africa;Germany;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2014-000178-20-BE
(EUCTR)
12/08/201522/06/2015Therapy of MCT8 patients with the thyroid hormone analog Triac.Thyroid hormone analog therapy of patients with severe psychomotor retardation caused by mutations in the MCT8 thyroid hormone transporter: The Triac Trial. - Triac Trial in MCT8 patients This therapuetical trial will be conducted in patient with the Allan-Herndon-Dudley Syndrome (AHDS), casued by mutations in the thyroid hormone transporter MCT8. This results in the characteristic clinical phenotype of severe psychomotor retardation due to local hypothyroidism in the brain, in combination with high serum T3 and high normal serum TSH levels that lead to local hyperthyroidism in tissues that do not dependent on MCT8, resulting in tachycardia, low body weight and muscle wasting.;Therapeutic area: Diseases [C] - Hormonal diseases [C19]Trade Name: Téatrois (Tiratricol)
Product Name: Téatrois
Erasmus Medical CentreNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
40Phase 2Canada;Belgium;Australia;Chile;South Africa;Germany;Italy
7NCT02060474
(ClinicalTrials.gov)
October 20145/2/2014Thyroid 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 DevelopmentCompletedN/A99 YearsMale46Phase 2Netherlands
8NCT01391637
(ClinicalTrials.gov)
June 20116/7/2011Long-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.NULLCompletedN/AN/AMale4United States
9NCT01005004
(ClinicalTrials.gov)
November 200928/10/2009Study 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.NULLCompleted6 Months5 YearsMale4Phase 1United States
10EUCTR2014-000178-20-DE
(EUCTR)
05/10/2015Therapy of MCT8 patients with the thyroid hormone analog Triac.Thyroid hormone analog therapy of patients with severe psychomotor retardation caused by mutations in the MCT8 thyroid hormone transporter: The Triac Trial. - Triac Trial in MCT8 patients This therapuetical trial will be conducted in patient with the Allan-Herndon-Dudley Syndrome (AHDS), casued by mutations in the thyroid hormone transporter MCT8. This results in the characteristic clinical phenotype of severe psychomotor retardation due to local hypothyroidism in the brain, in combination with high serum T3 and high normal serum TSH levels that lead to local hyperthyroidism in tissues that do not dependent on MCT8, resulting in tachycardia, low body weight and muscle wasting.;Therapeutic area: Diseases [C] - Hormonal diseases [C19]Trade Name: Téatrois (Tiratricol)
Product Name: Téatrois
INN or Proposed INN: Tiratricol
Erasmus Medical CentreNULLNot RecruitingFemale: no
Male: yes
40Phase 2France;Czech Republic;Canada;Belgium;Australia;Israel;South Africa;Germany;Italy