What side effects are associated with this type of intervention?

Treatments for Allan-Herndon-Dudley Syndrome (AHDS) that are similar to tiratricol (Triac), which acts as a thyroid hormone analog, can have side effects including increased heart rate, weight loss, anxiety, tremors, bone loss, muscle weakness, and potential cardiovascular issues. Careful monitoring and dosage adjustments are essential to manage these side effects effectively.

What prior FDA approvals exist?

There are no specific FDA-approved treatments mentioned for Allan-Herndon-Dudley Syndrome (AHDS) in the provided context. The trial studying tiratricol (Triac) aims to address the hypothyroid state in the brain and hyperthyroid state in peripheral tissues in AHDS patients, but no prior FDA approvals for similar treatments are noted.

What other types of research exist?

As of now, there are no specific novel alternatives to Tiratricol (Triac) explicitly mentioned for Allan-Herndon-Dudley Syndrome (AHDS) in the provided context. Patients should consult with their healthcare providers to explore any emerging therapies or clinical trials that may be available in 2024.

← Back to Search

Thyroid Hormone Analog

Triac for Allan-Herndon-Dudley Syndrome

Phase 2

Waitlist Available

Led By W.E. Visser, MD, PhD

Research Sponsored by Rare Thyroid Therapeutics International AB

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

The participant should be aged between 0 and 30 months on the day of inclusion

Must not have

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)

Previous treatment with tiratricol

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 96 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial will test tiratricol in young boys with MCT8 deficiency. Tiratricol can bypass the defective transporter to deliver thyroid hormone, potentially improving brain development and reducing harmful effects in other parts of the body. Tiratricol is a thyroid hormone analog that can potentially access the brain in the absence of MCT8 and restore at least a subset of cerebral thyroid hormone actions.

Who is the study for?

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.

What is being tested?

The Triac Trial II studies the effects of tiratricol (Triac) on brain hypothyroidism and peripheral hyperthyroidism in patients with MCT8 deficiency. The treatment lasts for 96 weeks, assessing its impact on neurodevelopmental impairment, with an option to continue for two more years.

What are the potential side effects?

While specific side effects of Triac aren't listed here, common ones may include symptoms related to altering thyroid hormone levels such as changes in metabolism, heart rate fluctuations, weight changes, fatigue or restlessness.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am a male with a confirmed MCT8 gene mutation.

Select...

I am between 0 and 30 months old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am not allergic to the trial medication and do not have issues digesting lactose.

Select...

I have been treated with tiratricol before.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 96 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~96 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 96 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Bayley Scales of Infant Development III Gross Motor Skill Domain score

Gross Motor Function Measure 88 (GMFM 88) total score

Secondary study objectives

Bayley Scales of Infant Development III score.

Blood pressure

Body weight

+5 more

Other study objectives

Estimate the elimination half-life of tiratricol in young children, reported in hours (optional and provided a medical reason prevails).

Estimate the maximum serum concentration of tiratricol in young children, reported in nmol/L (optional and provided a medical reason prevails).

Evaluate the effect of tiratricol treatment on brain function (optional) (BERA)

+27 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: MCT8 deficiency patientsExperimental Treatment1 Intervention

Triac treatment

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Tiratricol

Not yet FDA approved

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The primary treatment for Allan-Herndon-Dudley Syndrome (AHDS) involves the use of thyroid hormone analogs like tiratricol (Triac). These analogs mimic the action of thyroid hormones, aiming to correct the hypothyroid state in the brain and the hyperthyroid state in peripheral tissues. This dual action is crucial for AHDS patients because it helps to normalize thyroid hormone levels across different body systems, potentially improving neurodevelopmental outcomes and reducing peripheral thyrotoxicosis. Effective management of these imbalances is essential for improving the quality of life and overall health of individuals with AHDS.

Effectiveness and safety of the tri-iodothyronine analogue Triac in children and adults with MCT8 deficiency: an international, single-arm, open-label, phase 2 trial.