Triac for Allan-Herndon-Dudley Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a treatment called Triac (a thyroid hormone analog) for young boys with Allan-Herndon-Dudley syndrome (AHDS), a rare condition. An issue with the MCT8 gene causes AHDS, leading to brain development problems and an overactive thyroid in other body parts. The trial will assess whether Triac can improve these symptoms after nearly two years of treatment. Boys under 30 months old with a known MCT8 gene mutation, who haven't received certain thyroid medications for extended periods, are suitable candidates for this trial. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering a chance to contribute to important early findings.
Will I have to stop taking my current medications?
If your child has been treated with LT4 or PTU for less than three months, they can join the study six weeks after stopping these medications. If they have been on these medications for more than three months, they cannot participate.
Is there any evidence suggesting that Triac is likely to be safe for humans?
Research has shown that Triac, a treatment under study for Allan-Herndon-Dudley syndrome (AHDS), appears safe. In a study with children and adults with MCT8 deficiency, the same condition targeted by this trial, Triac was generally well-tolerated, with no major side effects directly linked to the treatment.
In these previous cases, Triac helped lower high levels of thyroid hormone, a key issue in AHDS. While it didn't resolve all problems, its safety profile supports further exploration of its benefits.
Triac is currently in a phase 2 trial, indicating it has already passed initial safety tests. This phase focuses more on the treatment's effectiveness, but safety remains closely monitored. So far, the safety data has been encouraging.12345Why do researchers think this study treatment might be promising?
Unlike the standard of care for Allan-Herndon-Dudley Syndrome, which primarily focuses on managing symptoms, Triac directly targets the underlying problem. This condition is tied to a deficiency in the MCT8 protein, which affects thyroid hormone transport. Triac is a thyroid hormone analog that can bypass this transport issue, potentially correcting the hormone imbalance. Researchers are excited because this could directly address the root cause of the syndrome rather than just alleviating symptoms, offering a more effective treatment option.
What evidence suggests that Triac might be an effective treatment for Allan-Herndon-Dudley syndrome?
Research has shown that Triac, the treatment under study for MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome (AHDS), may help manage symptoms of peripheral thyrotoxicosis, where the body's tissues overreact to thyroid hormones. Studies suggest that Triac can slow the loss of developmental skills in young patients, helping them maintain progress. However, while Triac has been effective for some symptoms, it does not appear to improve thyroid hormone function in the brain. Overall, Triac may offer important benefits for managing certain aspects of MCT8 deficiency.14678
Who Is on the Research Team?
Andrew Bauer, MD
Principal Investigator
Children's Hospital of Philadelphia
W.E. Visser, MD, PhD
Principal Investigator
Erasmus Medical Center
Kristina Sjöblom Nygren, MD
Principal Investigator
Rare Thyroid Therapeutics International AB
Stephen LaFranchi_Nicol
Principal Investigator
Oregon Health& Science University (OHSU) Doernbecher Childrens Hospital
Jan Lebl
Principal Investigator
Charles University and Motol University Hospital
Heiko Krude
Principal Investigator
Charité - Universitätsmedizin Berlin Institut fur experimental paediatrische endokrinologie
Are You a Good Fit for This Trial?
This trial is for young boys aged 0 to 30 months with MCT8 deficiency, also known as Allan-Herndon-Dudley Syndrome. They must have a mutation in the MCT8 gene and their parents or guardians should agree to follow study procedures. Boys who've had certain thyroid treatments or other investigational drugs recently are not eligible.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive tiratricol treatment to evaluate its effect on neurodevelopment impairment and peripheral thyrotoxicosis
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term treatment
Participants may continue treatment for an additional 2 years to evaluate long-term effects
What Are the Treatments Tested in This Trial?
Interventions
- Triac
Find a Clinic Near You
Who Is Running the Clinical Trial?
Rare Thyroid Therapeutics International AB
Lead Sponsor
Erasmus Medical Center
Collaborator