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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).
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)
Secondary ID [1] 260107 0
None
Universal Trial Number (UTN)
Trial acronym
DITPA01-10
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Allan-Herndon-Dudley Syndrome (AHDS) 265783 0
Condition category
Condition code
Human Genetics and Inherited Disorders 265937 265937 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 264523 0
Treatment: Drugs
Comparator / control treatment
Nil
Control group
Uncontrolled

Outcomes
Primary outcome [1] 266689 0
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.
Timepoint [1] 266689 0
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.
Secondary outcome [1] 276203 0
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.
Timepoint [1] 276203 0
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.

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.
Minimum age
0 Years
Maximum age
12 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Other forms of hypo- or hyperthyroidism.
2. Critical clinical condition.
3. Congenital heart disease or cardiac arrhythmias.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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
Phase 0
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
1/06/2011
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
10
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 265012 0
Commercial sector/Industry
Name [1] 265012 0
Zarion Pharmaceuticals
Country [1] 265012 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Zarion Pharmaceuticals
Address
Level 1, 74 Kingsway
Glen Waverley VIC 3150
Country
Australia
Secondary sponsor category [1] 264106 0
None
Name [1] 264106 0
Address [1] 264106 0
Country [1] 264106 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 266984 0
Southern Health HREC B
Ethics committee address [1] 266984 0
Ethics committee country [1] 266984 0
Date submitted for ethics approval [1] 266984 0
01/05/2011
Approval date [1] 266984 0
18/05/2011
Ethics approval number [1] 266984 0
10328B

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 32567 0
Address 32567 0
Country 32567 0
Phone 32567 0
Fax 32567 0
Email 32567 0
Contact person for public queries
Name 15814 0
Robert Gianello
Address 15814 0
Level 1, 74 Kingsway
Glen Waverley VIC 3150
Country 15814 0
Australia
Phone 15814 0
61403 174 863
Fax 15814 0
Email 15814 0
rg@pmp-vic.com
Contact person for scientific queries
Name 6742 0
Robert Gianello
Address 6742 0
Level 1, 74 Kingsway
Glen Waverley VIC 3150
Country 6742 0
Australia
Phone 6742 0
61403174863
Fax 6742 0
Email 6742 0
rg@pmp-vic.com

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.