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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03933163
Registration number
NCT03933163
Ethics application status
Date submitted
16/04/2019
Date registered
1/05/2019
Titles & IDs
Public title
Micronised Resveratrol as a Treatment for Friedreich Ataxia
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Scientific title
A Randomised Placebo-controlled Crossover Trial of Micronised Resveratrol as a Treatment for Friedreich Ataxia
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Secondary ID [1]
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36007
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Friedreich Ataxia
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Condition category
Condition code
Neurological
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Other neurological disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Neurological
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Neurodegenerative diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Resveratrol
Other: Resveratrol followed by placebo - 1g micronised resveratrol twice daily for 24 weeks, a wash-out period of 4 weeks, followed by twice daily placebo for 24 weeks.
Other: Placebo followed by Resveratrol - Twice daily placebo for 24 weeks, a wash-out period of 4 weeks, followed by 1g micronised resveratrol twice daily for 24 weeks
Treatment: Drugs: Resveratrol
Drug name: Micronised resveratrol. Dosage form: 500mg capsules. Alternate name: 1,3-Benzenediol, 5-\[2-(4-hydroxyphenyl)ethenyl\]-, (E). Ingredients: 99.50% pure trans-resveratrol. Placebo capsules will be identical in terms of taste, smell, and appearance.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Modified Friedreich Ataxia Rating Scale
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Assessment method [1]
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Change in the Modified Friedreich Ataxia Rating Scale score (score range 0-99) at 24 weeks compared with baseline. Higher scores are indicative of more severe disease.
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Timepoint [1]
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24 weeks
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Secondary outcome [1]
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Nine-Hole Peg Test
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Assessment method [1]
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Change in the Nine-Hole Peg Test at 24 weeks compared with baseline.
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Timepoint [1]
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24 weeks
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Secondary outcome [2]
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Berg Balance Scale
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Assessment method [2]
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Change in the Berg Balance Scale (score range 0-56) at 24 weeks compared with baseline. A higher score indicates lower fall risk.
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Timepoint [2]
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24 weeks
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Secondary outcome [3]
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Ataxia Instrumented Measure-Spoon
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Assessment method [3]
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Change in the Ataxia Instrumented Measure-Spoon at 24 weeks compared with baseline.
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Timepoint [3]
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24 weeks
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Secondary outcome [4]
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Friedreich Ataxia Impact Scale
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Assessment method [4]
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Change in the Friedreich Ataxia Impact Scale at 24 weeks compared with baseline. The Friedreich Ataxia Impact Scale comprises 8 subscales (score range 0-100) that are scored independently. A higher score indicates greater impact of Friedreich ataxia on health and well-being.
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Timepoint [4]
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24 weeks
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Secondary outcome [5]
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Modified Fatigue Impact Scale
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Assessment method [5]
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Change in the Modified Fatigue Impact Scale (score range 0-24) at 24 weeks compared with baseline. Higher scores indicate a greater impact of fatigue on an individual's activities.
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Timepoint [5]
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24 weeks
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Secondary outcome [6]
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Measures of speech
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Assessment method [6]
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Change in measures of speech (reading a paragraph, produce a prolonged vowel sound for 5 seconds, count from one to 20, and produce a 1-minute monologue on a pre-specified topic) at 24 weeks compared with baseline.
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Timepoint [6]
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24 weeks
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Secondary outcome [7]
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Measures of hearing
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Assessment method [7]
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Change in measures of hearing using the Listening in Spatialized Noise Test (LiSN-S) at 24 weeks compared with baseline.
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Timepoint [7]
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24 weeks
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Secondary outcome [8]
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Cardiac parameters measured by echocardiography
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Assessment method [8]
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Change in left ventricular global longitudinal strain at 24 weeks compared to baseline.
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Timepoint [8]
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24 weeks
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Secondary outcome [9]
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Cardiac parameters measured by ECG
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Assessment method [9]
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Change in QRS duration at lead V5 at 24 weeks compared to baseline.
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Timepoint [9]
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24 weeks
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Secondary outcome [10]
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Frataxin levels
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Assessment method [10]
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Change in frataxin levels at 24 weeks compared to baseline.
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Timepoint [10]
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24 weeks
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Secondary outcome [11]
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mRNA levels
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Assessment method [11]
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Change in PGC-1a mRNA levels and Nrf2 mRNA levels at 24 weeks compared to baseline.
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Timepoint [11]
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24 weeks
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Secondary outcome [12]
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Plasma F2-isoprostane levels
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Assessment method [12]
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Change in plasma F2-isoprostane levels at 24 weeks compared to baseline.
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Timepoint [12]
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24 weeks
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Eligibility
Key inclusion criteria
1. Age =16 years.
2. Diagnosis of FRDA, genetically documented to be due to homozygosity for a GAA repeat expansion in intron 1 of FXN.
3. Functional stage on the Ataxia subscale of the full FARS of 1 or higher (a score of 1 is assigned if the subject has "Minimal signs detected by the physician during screening. Can run or jump without loss of balance. No disability."), and total mFARS score of = 65.
4. Adequate end organ function defined as follows: (i) total bilirubin <2x upper limit of normal unless attributable to Gilbert disease, (ii) ALT and AST <1.5x upper limit of normal, (iii) Creatinine <2x upper limit of normal, (iv) neutrophils >1.5x10^9/L, (v) platelets >10^6/µL.
5. Written informed consent provided.
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Minimum age
16
Years
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Maximum age
No limit
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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. Non-elective hospitalisation within the past 60 days that could be of concern in the investigator's judgment. Any hospitalisation in the previous 60 days will be assessed and if in the investigator's judgement it could compromise the individual or the study, that person will not be recruited. Examples include if the individual is hospitalised for management of cardiac morbidity such as uncontrolled arrhythmia or angina or for orthopaedic surgery for a lower limb fracture.
2. Women who are pregnant or lactating or men and women of childbearing potential who are unwilling to use contraception for the duration of the study.
3. FRDA due to compound heterozygosity for an expanded GAA repeat and a point mutation/ deletion in the FXN gene.
4. Current or recent (in last 12 months) arrhythmias including: atrial fibrillation, atrial flutter, sinus tachycardia >120/min, sinus bradycardia <50/min. Symptomatic paroxysmal arrhythmia which is recurring frequently. Cardiac insufficiency (by New York Heart Association >2). Reduced LV ejection fraction (<50%) in the last six months.
5. Medical illness that in the judgment of the investigator would jeopardise the safe completion of the study. Examples include cancer, chronic inflammatory disease, severe diabetes (type I or II, HbA1c >8%), chronic liver insufficiency, epilepsy, thrombocytosis.
6. Evidence of end organ dysfunction through failure to meet one or more parameters in inclusion criterion number 4.
7. Prior invasive cancer (excluding localised basal cell or squamous cell skin cancer).
8. Known hypersensitivity to resveratrol.
9. Use of any investigational agent within 30 days of enrolment.
10. Use of antioxidants such as vitamin E, coenzyme Q10 or idebenone within 30 days prior to enrolment.
11. Concomitant use of medications with potential for clinically relevant drug interactions. This includes medications with a narrow therapeutic range that are metabolised by the cytochrome P450 3A4, 2D6 or 2C9 systems e.g. warfarin, amiodarone.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled 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
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Crossover
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
23/05/2019
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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
28/03/2024
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Sample size
Target
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Accrual to date
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Final
25
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
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Recruitment hospital [1]
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Royal North Shore Hospital - St Leonards
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Recruitment hospital [2]
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University of Queensland Centre for Clinical Research - Herston
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Recruitment hospital [3]
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Murdoch Children's Research Institute - Parkville
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Recruitment hospital [4]
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Royal Perth Hospital - Perth
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Recruitment postcode(s) [1]
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2065 - St Leonards
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Recruitment postcode(s) [2]
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4029 - Herston
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Recruitment postcode(s) [3]
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3052 - Parkville
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Recruitment postcode(s) [4]
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6000 - Perth
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Funding & Sponsors
Primary sponsor type
Other
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Name
Murdoch Childrens Research Institute
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The aim of this study is to assess the efficacy of micronised resveratrol as a treatment for FRDA, in terms of reducing the severity of ataxia symptoms at 24 weeks, through a randomised blinded, placebo controlled crossover trial.
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Trial website
https://clinicaltrials.gov/study/NCT03933163
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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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Martin B Delatycki, PhD, MBBS
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Address
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Murdoch Children's Research Institute
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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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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 scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
The de-identified data set collected for analysis of the study will be available six months after publication of the primary outcome.
The study protocol, analysis plan and consent forms will also be available. The data may be obtained from the Murdoch Children's Research Institute by contacting
[email protected]
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Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR), Analytic code
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When will data be available (start and end dates)?
6 months after publication of primary outcome
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Available to whom?
Prior to releasing any data the following are required: a data access agreement must be signed between relevant parties and there must be an agreement around appropriate acknowledgement and any additional costs involved must be covered. Data will only be shared with a recognised research institution which has approved the proposed analysis plan.
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Available for what types of analyses?
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How or where can data be obtained?
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT03933163