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Tiratricol · 1 trial · 1 indication
To evaluate the effects of tiratricol on neurodevelopment in young MCT8 deficiency patients, measured by the Gross Motor Function Measure (GMFM)-88 assessment. Potential result values range from 0 to 100%, the latter being representative for a 4-year old healthy child. A high score is equivalent to better/more neurodevelopment and is therefore a better outcome than a low score.
To evaluate the effect of tiratricol treatment on neurodevelopment measured by the Bayley Scales of Infant Development (BSID-III) Gross Motor Skill Domain score. Potential total raw scores range from 0-72, and can be age-adjusted before analysis. A high score is equivalent to better/more neurodevelopment and is therefore a better outcome than a low score
| Arm | Type | Description |
|---|---|---|
| MCT8 deficient patients | EXPERIMENTAL | Tiratricol (Triac) treatment |
| Name | Type | Description |
|---|---|---|
| Tiratricol | DRUG | Tiratricol, individually titrated dose |
Inclusion Criteria: * Signed and dated informed consent form from the parents or legal guardian. * Parents stated willingness to comply with all study procedures and availability for the duration of the study. * The participant should be aged between 0 and 30 months on the day of inclusion. * The p...
Tiratricol is an investigational small molecule being developed for Allan-Herndon-Dudley Syndrome, a rare genetic disorder. It is currently in Phase 2 clinical development and has not been approved by the FDA.
Tiratricol is a small molecule designed to treat Allan-Herndon-Dudley Syndrome, a rare disease caused by mutations in the MCT8 transporter. The drug is being studied for its ability to address the thyroid hormone transport deficiency associated with this condition.
Tiratricol is being developed by cbdMD, Inc., a company traded on the NYSE American under the ticker symbol YCBD. The company is conducting clinical trials to evaluate the drug for Allan-Herndon-Dudley Syndrome.
Tiratricol is in Phase 2 clinical development. It is an investigational drug and has not received FDA approval. The ongoing Phase 2 trial is active but not recruiting participants.
Tiratricol is being studied in a Phase 2 clinical trial with the identifier NCT02396459, titled 'Triac Trial II in MCT8 Deficiency Patients.' The trial is enrolling 22 male participants with Allan-Herndon-Dudley Syndrome across the United States, Czechia, Germany, and the Netherlands.
Tiratricol is also known as Triac, as reflected in the clinical trial title 'Triac Trial II in MCT8 Deficiency Patients.' The drug is being investigated under this alternative name for the treatment of Allan-Herndon-Dudley Syndrome.