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Triac Trial II in MCT8 Deficiency Patients

NCT02396459 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 22

Last updated 2026-02-20

No results posted yet for this study

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 initially be treated for 96 weeks with tiratricol, treatment effect on neurodevelopment impairment caused by hypothyroidism and peripheral thyrotoxicosis will be evaluated after 96 weeks treatment. Patients will be offered to continue on treatment for an additional 3 years.

Conditions

  • Allan-Herndon-Dudley Syndrome

Interventions

DRUG

Tiratricol

Tiratricol, individually titrated dose

Sponsors & Collaborators

Principal Investigators

  • W.E. Visser, MD, PhD · Erasmus Medical Center

  • Kristina Sjöblom Nygren, MD · Rare Thyroid Therapeutics International AB

  • Lindsey Nicol · Oregon Health& Science University (OHSU) Doernbecher Childrens Hospital

  • Jan Lebl · Charles University and Motol University Hospital

  • Heiko Krude · Charité - Universitätsmedizin Berlin Institut fur experimental paediatrische endokrinologie

  • Andrew Bauer, MD · Children's Hospital of Philadelphia

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
30 Months
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-12-07
Primary Completion
2027-07-18
Completion
2027-08-18
FDA Drug
Yes

Countries

  • United States
  • Czechia
  • Germany
  • Netherlands

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02396459 on ClinicalTrials.gov