Triac Trial II in MCT8 Deficiency Patients

• Status: Recruiting
• Trial end date: 2024-11-01
• Target enrollment: 0
• Participant gender: Male

Summary

This study will investigate the effect of treatment with tiratricol (also called Triac) in young boys (≤30 months) with MCT8 deficiency (also called the Allan-Herndon-Dudley syndrome (AHDS)). The hypothesis tested is that treatment with tiratricol will have a beneficial effect on the hypothyroid state in the brain as well as the hyperthyroid state in peripheral organs and tissues in these patients. Patients will be treated for 24 months with tiratricol, treatment effect on neurodevelopment impairment caused by hypothyroidism and the thyrotoxicosis will be evaluated at an interim analysis after 12 months treatment and after 24 months treatment.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Erasmus Medical Center; Rare Thyroid Therapeutics International AB
• Collaborator: Erasmus Medical Center
• Treatments: Chlorpheniramine, phenylpropanolamine drug combination; Guaifenesin; Phenylpropanolamine

Criteria

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 participant should be male and have a pathogenic mutation in the MCT8 gene.

Exclusion Criteria:

• Previous treatment with tiratricol.

• Previous treatment with a combination of Propylthiouracil (PTU) and Levothyroxine (LT4).

• Previous treatment with LT4 for a period longer than three months. Treatment with LT4 within three months of baseline visit.

• Major illness or recent major surgery (within four weeks of baseline visit 1) unrelated to MCT8 deficiency.

• Known allergic reactions to components of the IMP. Patients with galactose intolerance, Lapp lactase deficiency or malabsorption of glucose or galactose (the IMP contains lactose).

• Treatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit 1.