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Trial registered on ANZCTR
Registration number
ACTRN12611000472976
Ethics application status
Approved
Date submitted
4/05/2011
Date registered
6/05/2011
Date last updated
13/06/2012
Type of registration
Prospectively registered
Titles & IDs
Public title
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).
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Scientific title
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)
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Secondary ID [1]
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None
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Universal Trial Number (UTN)
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Trial acronym
DITPA01-10
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Allan-Herndon-Dudley Syndrome (AHDS)
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Condition category
Condition code
Human Genetics and Inherited Disorders
265937
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0
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
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.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
Nil
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Control group
Uncontrolled
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Outcomes
Primary outcome [1]
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To determine the safety and tolerability of orally administered DITPA in patients with AHDS.
Safety Assessments will include: Physical examination including weight; Vital signs; Neurological examination and imaging (including Electroencephalogram (EEG) and Magnetic Resonance Imaging (MRI); Echocardiogram (ECHO), Electrocardiogram (ECG), Holter 24h ECG; Cranial and Abdominal ultrasounds; Thyroid function tests (including TSH, fT3 and fT4); Bone metabolism; Blood gas analysis; Blood chemistry; Haematology; Urine dipstick and Adverse Events.
Patients will be closely monitored for possible risks, side effects and discomforts including:
-Arrhythmia (irregular or abnormal heart beat rhythm)
-Tachycardia (rapid heart rate)
-Hypotension (abnormally low blood pressure)
-Dyspnoea (difficulty in breathing)
-Weight Loss
-Reduction in Appetite
-Diarrhea
-Change in blood test results
-Accumulation and damage to every organ
-Carcinogenicity.
-Having a blood sample taken may cause some discomfort or bruising. Sometimes, the blood vessel may swell, or blood may clot in the blood vessel. Rarely, there could be a minor infection or bleeding. If this happens, it can be easily treated.
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Assessment method [1]
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Timepoint [1]
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Physical exams and vital signs will be performed at baseline then twice daily for a minimum of 2 days upon commencement of treatment, then on a weekly basis for the first month, followed fortnightly up to month 3 and monthly up to month 12.
Blood chemistry/haematology and blood gas analysis will be performed at baseline then every second day during the first 4 days of treatment, then weekly for the first month, followed fortnightly up to month 3 and monthly up month 6. Lipid studies are performed at baseline, 6 weeks, 3 months, 6 months and 12 months.
Echocardiogram (ECHO) and Electrocardiogram (ECG) will be performed at baseline then months 1 and 6. A Holter 24h ECG recording will be performed at baseline, after 4 days of treatment and at months 1, 3, 6 and 12.
Adverse events will be recorded as they occur throughout the duration of the study.
An abdominal ultrasound will be performed at baseline and at month 6 and 12.
Bone age Xray will be performed at baseline, 6 months and 12 months.
DXA bone density scan will be performed at baseline and 12 months.
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Secondary outcome [1]
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To determine the pharmacodynamics and biochemical response of orally administered DITPA in patients with ADHS.
Pharmacodynamic and Response Assessments will include: Thyroid function biomarkers; Levels of DITPA in blood; weight gain; neurological examination; growth parameters and feeding pattern; neuroanatomical development; neurophysical developement; vision and hearing; motor function assessment; neurodevelopment assessment.
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Assessment method [1]
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Timepoint [1]
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Thyroid function biomarkers will be measured at baseline, once daily for 4 days into treatment, then on a weekly basis for the first month, followed fortnightly up to month 3 and monthly up to month 12.
DITPA levels in blood will be measured on Day 1 and 4 of treatment, then on a weekly basis for the first month, followed fortnightly up to month 3 and monthly up to month 12.
Neurological exams and growth parameters/feeding patterns will be performed at baseline, then on day 2, then weekly for the first month, followed fortnightly up to month 3 and monthly up to month 12.
An MRI will be performed at baseline and month 12.
An EEG will be performed at baseline and after 6 months.
A Vision and hearing assessment will be performed at baseline and at 6 months.
Motor Function will be assessed at baseline and at 6 months.
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Eligibility
Key inclusion criteria
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. Medically stable according to the treating physician.
3. Informed parental consent.
4. Aged between 0 and 12 years.
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Minimum age
0
Years
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Maximum age
12
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Other forms of hypo- or hyperthyroidism.
2. Critical clinical condition.
3. Congenital heart disease or cardiac arrhythmias.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 0
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Type of endpoint/s
Safety/efficacy
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Date of first participant enrolment
Anticipated
1/06/2011
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Actual
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
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Sample size
Target
10
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Funding & Sponsors
Funding source category [1]
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Commercial sector/Industry
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Name [1]
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Zarion Pharmaceuticals
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Address [1]
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Level 1, 74 Kingsway
Glen Waverley VIC 3150
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Country [1]
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Australia
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Primary sponsor type
Commercial sector/Industry
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Name
Zarion Pharmaceuticals
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Address
Level 1, 74 Kingsway
Glen Waverley VIC 3150
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Country
Australia
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Secondary sponsor category [1]
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None
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Name [1]
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Address [1]
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Country [1]
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Ethics approval
Ethics application status
Approved
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Ethics committee name [1]
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Southern Health HREC B
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Ethics committee address [1]
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Southern Health HREC B, Monash Medical Centre, 246 Centre Rd, Clayton, Vic, 3168
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Ethics committee country [1]
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Date submitted for ethics approval [1]
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01/05/2011
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Approval date [1]
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18/05/2011
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Ethics approval number [1]
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10328B
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Summary
Brief 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.
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Trial website
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Robert Gianello
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Address
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Level 1, 74 Kingsway
Glen Waverley VIC 3150
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Country
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Australia
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Phone
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61403 174 863
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Name
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Robert Gianello
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Address
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Level 1, 74 Kingsway
Glen Waverley VIC 3150
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Country
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Australia
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Phone
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61403174863
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Fax
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Email
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[email protected]
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No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
Documents added manually
No documents have been uploaded by study researchers.
Documents added automatically
No additional documents have been identified.
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