RecruitingEarly Phase 1ACTRN12611000472976

A study to determine the safety, tolerability and effects of orally administered 3,5-diiodothyropropionic acid (DITPA) in patients with Allan-Herndon-Dudley-Syndrome (AHDS).

A multi-centre, open-label study to determine the safety, tolerability and pharmacodynamics of orally administered 3,5-diiodothyropropionic acid (DITPA) in patients aged 3 to 12 with Allan-Herndon-Dudley Syndrome (AHDS)

Sponsor

Zarion Pharmaceuticals

Enrollment

10 participants

Start Date

Jun 1, 2011

Study Type

Interventional

Conditions

Allan-Herndon-Dudley Syndrome (AHDS)

Summary

There is currently no approved curative therapy for infants with MCT8 deficiency. Treatment for individuals with this disease aims to provide relief for any symptoms (e.g., treatment with propylthiouracil to reduce the symptoms of peripheral hyperthyroidism) and support in the care of the patient. No treatment up to now has shown to alter the ultimate progression of neurological impairment DITPA is a thyroid hormone analogue with an MCT8 independent neuronal uptake. As a thyroid hormone receptor antagonist, DITPA is known to have distinct thryiod hormone effects on the cardiovascular system, such as an increase in left ventricular systolic performance, improvement of diastolic function, and a decrease in peripheral vascular resistance. Furthermore, DITPA is known to decrease serum lipoproteins and body weight.

Eligibility

Sex: Both males and femalesMin Age: 0 YearssMax Age: 12 Yearss

Plain Language Summary

Simplified for easier understanding

This study is testing a drug called DITPA in children with Allan-Herndon-Dudley Syndrome (AHDS), also known as MCT8 deficiency. AHDS is a very rare genetic condition where the brain cannot properly absorb thyroid hormone, leading to severe intellectual disability and muscle problems, while other parts of the body receive too much thyroid hormone. DITPA is a thyroid hormone-like drug that can enter the brain through a different pathway. This is an early-phase safety study to see if the drug is safe and tolerable in children. You may be eligible if: - Your child is between 0 and 12 years old - Your child has a confirmed genetic mutation of the SLC16A2/MCT8 gene - Your child has blood test results consistent with the diagnosis (elevated T3, low T4) - Your child is medically stable You may NOT be eligible if: - Your child has other thyroid disorders not related to AHDS - Your child is critically ill - Your child has a congenital heart defect or heart rhythm problems Talk to your doctor about whether this trial might be right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

3,5-diiodothyropropionic Acid (DITPA) will be administered daily started at a dose of 1.5 mg/kg/d and increased to 4.75 mg/kg/d. The dose may be escalated from 1.5 mg/kg/d in increments of 33%, at th

3,5-diiodothyropropionic Acid (DITPA) will be administered daily started at a dose of 1.5 mg/kg/d and increased to 4.75 mg/kg/d. The dose may be escalated from 1.5 mg/kg/d in increments of 33%, at the discretion of the treating physician. The daily dose will be divided into 3 equal portions given close to 8 hours apart. It is anticipated that DITPA therapy will be lifelong.

Locations(1)

Australia

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ACTRN12611000472976