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

Inclusion Criteria4

Proven mutation of the SLC16A2/MCT8 gene, with a presumptive diagnosis of MCT8 Deficiency made based on elevated fT3, decrease of serum rT3 concentrations, low fT4 in the blood.
Medically stable according to the treating physician.
Informed parental consent.
Aged between 0 and 12 years.

Exclusion Criteria3

Other forms of hypo- or hyperthyroidism.
Critical clinical condition.
Congenital heart disease or cardiac arrhythmias.

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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