139. 先天性大脳白質形成不全症 Congenital cerebral hypomyelination Clinical trials / Disease details


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

  
5 trials found
No.TrialIDDate_
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PhaseCountries
1EUCTR2014-000178-20-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
2EUCTR2014-000178-20-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
3EUCTR2014-000178-20-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
4EUCTR2014-000178-20-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
5EUCTR2014-000178-20-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