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Trial registered on ANZCTR

Registration number
Ethics application status
Date submitted
Date registered
Date last updated
Type of registration
Prospectively registered

Titles & IDs
Public title
A Clinical Study to Determine the Safety and Tolerability of Orally Administered 3,5-Diiodothyropropionic Acid in Patients with Allan-Herndon-Dudley Syndrome (6 month-2 years after commencement of treatment).
Scientific title
A Multi-Centre, Open-Label Study to Determine the Safety, Tolerability and Pharmacodynamics of Orally Administered 3,5-Diiodothyropropionic Acid (DITPA) in 10 Patients with Allan-Herndon-Dudley Syndrome (AHDS)
Secondary ID [1] 273303 0
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Allan-Herndon-Dudley Syndrome (AHDS). 279075 0
Condition category
Condition code
Neurological 279264 279264 0 0
Other neurological disorders

Study type
Description of intervention(s) / exposure
DITPA (3,5-Diiodothyropropionic Acid).
Doses ranging 1.5 to 4.75 mg/kg/day.
Oral dosing with the daily dose divided into 3 equal parts at 8 hour intervals, for the duration of the study.
Duration of study is 18 months.
Dose changes will be at the discretion of the PI, on a case by case basis.
Intervention code [1] 283650 0
Treatment: Drugs
Comparator / control treatment
No treatment
Control group

Primary outcome [1] 279888 0
Improvement of the symptoms and parameters associated with AHDS (including normalization of the thyroid hormone levels, and/or weight gain, and/or preventing of further neurological deterioration). The tests are tabulated in the protocol. In brief these are:
Safety and Tolerability Assessments
* Physical examination including weight
* Vital signs (heart rate (including sleeping heart rate), non-invasive blood pressure, respiratory rate, and temperature)
* Neurological examination and imaging (including EEG and MRI)
* Echocardiogram, ECG, Holter 24h ECG
* Cranial and abdominal ultrasounds
* Thyroid function tests (including TSH, fT3, rT3 and fT4, and thyroglobulin)
* Bone metabolism
* Blood gas analysis
* Blood chemistry (including urea, electrolytes, creatinine, creatinine kinase, uric acid, bone metabolites, ferritin, sex-hormone binding globulin and liver function tests)
* Haematology
* Urine dipstick
* Adverse Events

Pharmacodynamic and Response Assessments

* Levels of TSH, fT3, rT3 and fT4 and thyroglobulin in blood
* Levels of DITPA in blood
* Weight gain
* Neurological examination
* Growth parameters and Feeding pattern
* Neuroanatomical development
* Neurophysical development
* Vision and Hearing
* Motor function assessment
* Neurodevelopment assessment
Timepoint [1] 279888 0
Assessments at 2-monthly intervals between 6-12 months from starting treatment. Then assessments at 3-monthly intervals between 12-24 months from starting treatment.
Secondary outcome [1] 294687 0
Timepoint [1] 294687 0

Key inclusion criteria
The protocol has not been registered elsewhere.

1. 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.
2. Commenced on treatment of DITPA as per protocol DITPA 01-10 (HREC no. 10328B). The continuation will be a protocol titled "A Multi-Centre, Open-Label Study to Determine the Safety, Tolerability and Pharmacodynamics of Orally Administered 3,5-Diiodothyropropionic Acid (DITPA) in Patients with Allan-Herndon-Dudley Syndrome (AHDS) (6 months – 2 years after commencement of treatment)"
3. Medically stable according to the treating physician.
4. Informed parental consent.
Minimum age
0 Years
Maximum age
12 Years
Can healthy volunteers participate?
Key exclusion criteria
1. Other forms of hypo- or hyperthyroidism.
2. Critical clinical condition.

Study design
Purpose of the study
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Enrolment follows the determination that the subject has met the inclusion criteria
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?

Intervention assignment
Single group
Other design features
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment status
Date of first participant enrolment
Date of last participant enrolment
Date of last data collection
Sample size
Accrual to date
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 284134 0
Commercial sector/Industry
Name [1] 284134 0
Zarion Pharmaceuticals P/L
Address [1] 284134 0
Level 1, 74 Kingsway, Glen Waverley, 3150, Victoria
Country [1] 284134 0
Primary sponsor type
Commercial sector/Industry
Zarion Pharmaceuticals P/L
Level 1, 74 Kingsway, Glen Waverley, 3150, Victoria
Secondary sponsor category [1] 269094 0
Name [1] 269094 0
Address [1] 269094 0
Country [1] 269094 0

Ethics approval
Ethics application status
Ethics committee name [1] 272099 0
Southern Health HREC B
Ethics committee address [1] 272099 0
Monash Medical Centre, 246 Clayton Rd., Clayton, 3168
Ethics committee country [1] 272099 0
Date submitted for ethics approval [1] 272099 0
Approval date [1] 272099 0
Ethics approval number [1] 272099 0

Brief summary
Allan-Herndon-Dudley Syndrome (AHDS) that is a rare condition that usually results in severe physical and mental impairment and for which no licensed treatment is available. The study aims to provide an experimental treatment for children with AHDS. It is proposed that the daily oral administration of a compound called 3-5,Diiodothyropropionic Acid (referred to as DITPA) be provided to children with AHDS as the experimental treatment. We hypothesise that DITPA will improve the symptoms and abnormal thyroid function associated with AHDS.
Trial website
Trial related presentations / publications
Public notes

Principal investigator
Name 33336 0
Address 33336 0
Country 33336 0
Phone 33336 0
Fax 33336 0
Email 33336 0
Contact person for public queries
Name 16583 0
Mr Robert Gianello
Address 16583 0
8 Grandview Grove
Country 16583 0
Phone 16583 0
Fax 16583 0
Email 16583 0
Contact person for scientific queries
Name 7511 0
Dr. Flora Wong
Address 7511 0
Level 5, Monash Medical Centre,
246 Clayton Road,
Clayton, VIC 3168
Country 7511 0
Phone 7511 0
+61 03 95945482
Fax 7511 0
+61 03 95946115
Email 7511 0

No information has been provided regarding IPD availability
Summary results
No Results