Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II

Phase 2

Recruiting

• Conditions: Allan-Herndon-Dudley syndrome; MCT8 deficiency; 10029299; 10043739; 10029317

• Registration Number: NL-OMON52600
• Lead Sponsor: Rare Thyroid Therapeutics International AB

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 9 September 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 1

Inclusion Criteria

1. Signed and dated informed consent form from the parents or legal guardian.
2. Parents stated willingness to comply with all study procedures and
availability for the duration
of the study.
3. The participant should be aged between 0 and 30 months on the day of
inclusion.
4. The participant should be male and have a pathogenic mutation in the MCT8
gene.

Exclusion Criteria

1. Previous treatment with tiratricol.
2. Previous treatment with LT4 and/or PTU and/or other anti-thyroid medication
for a period longer than three months. Patients previously treated with LT4 for
a shorter period than 3 months may be included in the study (baseline visit)
six weeks (or longer) after last dose of LT4 if two consecutive analyses show
stable TFT*. Patients treated with PTU and/or other anti-thyroid medication for
a shorter period than three months may be included in the study (baseline
visit) six weeks (or longer) after last dose.
3. Major illness or recent major surgery (within four weeks of baseline visit
1) unrelated to MCT8
deficiency.
4. 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).
5. Treatment with another investigational drug or participation in other
interventional trial within
three months prior to baseline visit 1.
*Stable TFT (T3, T4, fT4), determined as a maximal variation of 20%, should be
demonstrated at two separate occasions at least two weeks apart, measured on
the same platform.

Study & Design

• Study Type: Interventional
• Study Design: Not specified