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Thyroid Hormone Analog

Triac for Allan-Herndon-Dudley Syndrome

Phase 2
Waitlist Available
Led By Jan Lebl
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 96 weeks
Awards & highlights

Study Summary

This trialwill study the effects of a drug, tiratricol, on young boys with a thyroid condition called MCT8 deficiency. It will see if the drug helps with brain hypothyroidism and peripheral hyperthyroidism.

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.Check my eligibility
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.See study design
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.

Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Bayley Scales of Infant Development III Gross Motor Skill Domain score
Gross Motor Function Measure 88 (GMFM 88) total score
Secondary outcome measures
Bayley Scales of Infant Development III score.
Blood pressure
Body weight
+5 more
Other outcome measures
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

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

Find a Location

Who is running the clinical trial?

Rare Thyroid Therapeutics International ABLead Sponsor
3 Previous Clinical Trials
37 Total Patients Enrolled
3 Trials studying Allan-Herndon-Dudley Syndrome
37 Patients Enrolled for Allan-Herndon-Dudley Syndrome
Erasmus Medical CenterOTHER
649 Previous Clinical Trials
1,877,681 Total Patients Enrolled
1 Trials studying Allan-Herndon-Dudley Syndrome
46 Patients Enrolled for Allan-Herndon-Dudley Syndrome
Jan LeblPrincipal InvestigatorCharles University and Motol University Hospital

Media Library

Triac (Thyroid Hormone Analog) Clinical Trial Eligibility Overview. Trial Name: NCT02396459 — Phase 2
Allan-Herndon-Dudley Syndrome Research Study Groups: MCT8 deficiency patients
Allan-Herndon-Dudley Syndrome Clinical Trial 2023: Triac Highlights & Side Effects. Trial Name: NCT02396459 — Phase 2
Triac (Thyroid Hormone Analog) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02396459 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Have there been any other scientific investigations regarding individuals with MCT8 deficiency?

"At present, there is one live trial for MCT8 deficiency patients which has not reached Phase 3 yet. This research effort is centered in Berlin, Oregon with 7 different medical sites participating."

Answered by AI

What is the desired outcome of this medical experiment?

"This trial is observing a 96 week timeline, in order to assess the primary outcome of Bayley Scales of Infant Development III Gross Motor Skill Domain score. Additionally, secondary outcomes such as GMFM-88 individual item scores 10 and 24; GMFM Domain B (Sitting) summary score 18-37; Motor Milestone Responder Analysis from Section 2 of HINE; Serum T3 concentrations for biochemical thyrotoxic features evaluation; and tissue specific markers assessing serum sex hormone binding globulin levels will be monitored throughout this period."

Answered by AI

Is this the inaugural clinical experiment of its kind?

"Since 2020, MCT8 deficiency patients have been the focus of extensive research. Initially sponsored by Rare Thyroid Therapeutics International AB in 2020, the first study included 22 participants and successfully completed its Phase 2 clinical trial stage. This organisation is currently running the only active investigation into this condition."

Answered by AI

Does this research initiative have any openings for participants?

"This medical experiment has completed the recruitment process. Initially posted on December 7th 2020, it was last updated October 27 2022. For those seeking alternative clinical trials, there are currently two studies requiring participants: one for Allan-Herndon-Dudley Syndrome and another for MCT8 Deficiency patients."

Answered by AI

How many research subjects are contributing to this clinical experiment?

"This clinical trial is no longer recruiting participants. It was initially advertised on December 7th 2020, and most recently updated on October 27th 2022. However, there are currently two other medical studies that may be of interest: one for Allan-Herndon-Dudley Syndrome patients and another dedicated to MCT8 deficiency sufferers which remain open for recruitment."

Answered by AI

Have any treatments for MCT8 deficiency been officially endorsed by the FDA?

"Considering the Phase 2 trial status, our team at Power gave MCT8 deficiency patients a safety rating of two. At this stage, there is evidence only to support its security and not necessarily efficacy."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Triac Trial II in MCT8 Deficiency Patients
2020. "Triac Trial II in MCT8 Deficiency Patients". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02396459.
~1 spots leftby Jun 2024
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