139. 先天性大脳白質形成不全症
［臨床試験数：10，薬物数：7（DrugBank：2），標的遺伝子数：2，標的パスウェイ数：2］
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.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02396459 (ClinicalTrials.gov) | March 2020 | 7/3/2015 | Triac Trial II in MCT8 Deficiency Patients | Tiratricol Treatment of Children With Monocarboxylate Transporter 8 Deficiency: Triac Trial II | Allan-Herndon-Dudley Syndrome | Drug: Triac | Rare Thyroid Therapeutics International AB | Erasmus Medical Center | Not yet recruiting | N/A | 30 Months | Male | 18 | Phase 2 | Netherlands |
| 2 | JPRN-jRCTs031180348 | 01/04/2018 | 18/03/2019 | 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 | 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 Ken | NULL | Complete | 5age | 20age | Male | 10 | N/A | Japan |
| 3 | EUCTR2014-000178-20-CZ (EUCTR) | 04/02/2016 | 23/06/2015 | Therapy 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 Centre | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 40 | Phase 2 | Czech Republic;Belgium;Germany;Italy | ||
| 4 | EUCTR2014-000178-20-IT (EUCTR) | 01/02/2016 | 16/10/2015 | Therapy 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 Centre | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 40 | Phase 2 | Germany;Italy | ||
| 5 | EUCTR2014-000178-20-FR (EUCTR) | 14/01/2016 | 14/12/2015 | Therapy 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 Centre | Centre Hospitalier de Toulouse | Not Recruiting | Female: no Male: yes | 40 | Phase 2 | France;Czech Republic;Canada;Belgium;Australia;South Africa;Germany;Italy | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | EUCTR2014-000178-20-BE (EUCTR) | 12/08/2015 | 22/06/2015 | Therapy 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 Centre | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 40 | Phase 2 | Canada;Belgium;Australia;Chile;South Africa;Germany;Italy | ||
| 7 | NCT02060474 (ClinicalTrials.gov) | October 2014 | 5/2/2014 | 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 | Completed | N/A | 99 Years | Male | 46 | Phase 2 | Netherlands |
| 8 | NCT01391637 (ClinicalTrials.gov) | June 2011 | 6/7/2011 | 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. | NULL | Completed | N/A | N/A | Male | 4 | United States | |
| 9 | NCT01005004 (ClinicalTrials.gov) | November 2009 | 28/10/2009 | 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. | NULL | Completed | 6 Months | 5 Years | Male | 4 | Phase 1 | United States |
| 10 | EUCTR2014-000178-20-DE (EUCTR) | 05/10/2015 | Therapy 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 Centre | NULL | Not Recruiting | Female: no Male: yes | 40 | Phase 2 | France;Czech Republic;Canada;Belgium;Australia;Israel;South Africa;Germany;Italy |