Triac for Allan-Herndon-Dudley Syndrome

Recruiting at 6 trial locations

Overseen By F.S. van Geest, MD

Age: < 18

Sex: Male

Trial Phase: Phase 2

Sponsor: Rare Thyroid Therapeutics International AB

Must not be taking: LT4, PTU, Anti-thyroid

Disqualifiers: Major illness, Recent surgery, Allergies, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 96 weeks

22 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

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.

What data supports the effectiveness of the drug Triac for treating Allan-Herndon-Dudley Syndrome?

Research indicates that Triac (3,3',5 triiodothyroacetic acid) can improve the hormone levels in patients with Allan-Herndon-Dudley Syndrome, which is important for managing the condition. ^{1 2 3 4 5}

Is Triac safe for humans?

The research articles provided do not contain specific safety data for Triac in humans. ^{1 2 3 4 6}

How is the drug Triac different from other treatments for Allan-Herndon-Dudley Syndrome?

Triac is unique because it specifically targets the abnormal thyroid hormone levels seen in Allan-Herndon-Dudley Syndrome, which are caused by mutations in the MCT8 transporter. This approach is different from other treatments that may not directly address the underlying hormone transport issue. ^{1 3 5 6 7}

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

Eligibility Criteria

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

I am a male with a confirmed MCT8 gene mutation.

I am between 0 and 30 months old.

Signed and dated informed consent form from the parents or legal guardian

Exclusion Criteria

I have been treated with thyroid medication for more than 3 months or stopped it at least 6 weeks ago with stable tests.

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

Treatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit 1.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive tiratricol treatment to evaluate its effect on neurodevelopment impairment and peripheral thyrotoxicosis

96 weeks

Regular visits for monitoring and assessment

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term treatment

Participants may continue treatment for an additional 2 years to evaluate long-term effects

2 years

Treatment Details

Interventions

• Triac

Trial Overview 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.

Participant Groups

Group I: MCT8 deficiency patients Experimental Treatment 1 Intervention

Triac treatment

Who Is Running the Clinical Trial?

Rare Thyroid Therapeutics International AB

Lead Sponsor

Trials

4

Recruited

60+

Erasmus Medical Center

Collaborator

Trials

742

Recruited

2,156,000+

References

1. China pubmed.ncbi.nlm.nih.gov

[Clinical and genetic features of five patients with Allan-Herndon-Dudley syndrome]. [2020]

2. Netherlands pubmed.ncbi.nlm.nih.gov

Allan-Herndon-Dudley syndrome in Hong Kong: Implication for newborn screening. [2023]

3. England pubmed.ncbi.nlm.nih.gov

High T3, Low T4 Serum Levels in Mct8 Deficiency Are Not Caused by Increased Hepatic Conversion through Type I Deiodinase. [2020]

4. China pubmed.ncbi.nlm.nih.gov

[A family with Allan-Herndon-Dudley syndrome due to SLC16A2 gene mutation]. [2020]

5. United States pubmed.ncbi.nlm.nih.gov

Allan-Herndon-Dudley syndrome with unusual profound sensorineural hearing loss. [2020]

6. India pubmed.ncbi.nlm.nih.gov

Allan-Herndon-Dudley syndrome. [2009]

7. Chile pubmed.ncbi.nlm.nih.gov

[Allan-Herndon-Dudley syndrome: a diagnosis to rule out in any male infant with undiagnosed hypotonia]. [2022]