Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2
22 participants
INTERVENTIONAL
2020-12-07
2026-06-30
Brief Summary
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Detailed Description
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Preclinical studies have shown that the T3 analogue tiratricol is transported into cells in an MCT8-independent manner. In animal models mimicking MCT8 deficiency, Triac has been shown to normalize brain development if administrated during early postnatal life.
Recently, Triac Trial I (NCT02060474) has shown that tiratricol treatment in patients with MCT8 deficiency improves key clinical and biochemical features caused by the toxic effects of the high T3 concentrations. No drug related serious adverse events have occurred during Triac Trial I.
This study will investigate the effect of treatment with tiratricol 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 initially be treated for 96 weeks with tiratricol, treatment effect will be evaluated after 96 weeks. After the 96 week treatment period, patients will enter Part II of the trial, evaluating long-term treatment. Patients will be followed for an additional 2 years and treatment effect will be evaluated after 3 years and 4 years respectively from start of treatment.
Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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MCT8 deficiency patients
Triac treatment
Triac
Triac, individually titrated dose
Interventions
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Triac
Triac, individually titrated dose
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 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.
* 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.
30 Months
MALE
No
Sponsors
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Erasmus Medical Center
OTHER
Rare Thyroid Therapeutics International AB
INDUSTRY
Responsible Party
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Principal Investigators
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W.E. Visser, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Erasmus Medical Center
Kristina Sjöblom Nygren, MD
Role: STUDY_DIRECTOR
Rare Thyroid Therapeutics International AB
Stephen LaFranchi_Nicol
Role: PRINCIPAL_INVESTIGATOR
Oregon Health& Science University (OHSU) Doernbecher Childrens Hospital
Jan Lebl
Role: PRINCIPAL_INVESTIGATOR
Charles University and Motol University Hospital
Heiko Krude
Role: PRINCIPAL_INVESTIGATOR
Charité - Universitätsmedizin Berlin Institut fur experimental paediatrische endokrinologie
Andrew Bauer, MD
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital of Philadelphia
Locations
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Oregon Health & Science University (OHSU) Doernbecher Childrens Hospital
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Charles University and Motol University Hospital; The department of peadiatrics of the 2nd faculty of medicine
Prague, , Czechia
Charité - Universitätsmedizin Berlin Institut fur experimental paediatrische endokrinologie
Berlin, , Germany
Erasmus MC
Rotterdam, , Netherlands
Countries
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Other Identifiers
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MCT8-2019-2
Identifier Type: -
Identifier Source: org_study_id