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Trial registered on ANZCTR


Registration number
ACTRN12613000751774
Ethics application status
Approved
Date submitted
3/07/2013
Date registered
5/07/2013
Date last updated
5/07/2013
Type of registration
Prospectively registered

Titles & IDs
Public title
In children with inflammatory bowel disease and iron deficiency, does intravenous iron therapy with Ferric Carboxymaltose significantly improve cognitive function, quality of life and iron status.

Scientific title
In children with inflammatory bowel disease and iron deficiency, does intravenous iron therapy with Ferric Carboxymaltose signficantly improve cognitive function, quality of life and iron status.
Secondary ID [1] 282780 0
nil
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Paediatric Inflammatory Bowel Disease 289542 0
Iron deficiency anaemia
289547 0
Iron deficiency without anaemia 289553 0
Condition category
Condition code
Oral and Gastrointestinal 289864 289864 0 0
Inflammatory bowel disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
In Inflammatory bowel disease patients with iron deficiency, baseline iron status will be obtained, along with quality of life data (using IMPACT III questionnaire) and cognitive function (WISC). A single intravenous iron infusion with Ferric Carboxymaltose (15mg/kg up to a maxmum dose of 500mg, in 100mL N/Saline) will be administered, and 8 weeks following this iron status, quality of life and cognitive function will be obtained, and results compared. If iron deficiency is ongoing, a second infusion of Ferric Carboxymaltose will be administered.
Intervention code [1] 287453 0
Treatment: Drugs
Comparator / control treatment
Given the known detrimental effect of iron deficiency in paediatric patients, it would be unethical to have a control group receiving no therapy. As such, all patients with iron deficiency will receive intravenous iron.
Control group
Uncontrolled

Outcomes
Primary outcome [1] 289926 0
Demonstrate that treatment of iron deficiency and iron deficiency anaemia with Ferric Carboxymaltose is safe and effective in paediatric inflammatory bowel disease in patients six and over.

This outcome will be demonstrated with improvement in iron studies at 8 weeks following infusion
Safety data will be obtained at the completion of the trial, by collating adverse drug reactions, which in adult data and children 14 years and over have been well established. Most common side effects are : Headache (less than 3.3 in 100), gastrointestinal upset ( 1 in 100), flushing (less than 1 in 100), hypersensitivity reactions (less than 1 in 100). There have been no documented cases of anaphylaxis.
Timepoint [1] 289926 0
At eight weeks following intravenous iron infusion
Primary outcome [2] 289933 0
Demonstrate that the treatment of iron deficiency to correction with Ferric Carboxymaltose improves cognitive function and health related quality of life.

This outcome will be measured by comparing baseline quality of life and cognitive function data with data obtained at 8 weeks following infusion. Quality of life data will be obtained using the IMPACT III questionnaire; cognitive function using WISC-R.
Timepoint [2] 289933 0
At eight weeks following intravenous iron infusion.
Secondary outcome [1] 303580 0
1 - To show that Iron deficiency and iron deficiency anaemia are prevalent in paediatric inflammatory bowel disease

This data will be obtained through blood tests performed at baseline, as part of routine clinical care.
Timepoint [1] 303580 0
At baseline, prior to iron infusion.
Secondary outcome [2] 303598 0
2 - Show that iron deficiency and iron deficiency anaemia adversely impact cognitive function and health related quality of life in paediatric inflammatory bowel disease, as measured b the WISC-R and Impact III questionnaires respectively.

This will be obtained at baseline through the quality of life and cognitive function questionnaire.
Timepoint [2] 303598 0
At baseline, prior to iron infusion.

Eligibility
Key inclusion criteria
Paediatric patients with documented inflammatory bowel disease (Crohn's disease or Ulcerative colitis) being treated at the Royal Children's Hospital, with iron deficiency as defined by international criteria (ferritin less than 30, TS less than 16% or ferritin less than 100 with coexisting inflammation)
Minimum age
6 Years
Maximum age
18 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Patients will be excluded if:
1 - They have a haemoglobinopathy
2 - They received a blood transfusion in the previous 6 weeks
3 - Patient or family do not give consent for participation in the study
4 - They have advanced liver disease
5 - They have acute severe colitis

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
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 4
Type of endpoint(s)
Statistical methods / analysis
The patient cohort was determined by the number of paediatric inflammatory bowel disease patients with coexisting iron deficiency at the Royal Children's Hospital. This number approximates 50. We will recruit any patient fulfilling this criteria during the study time period.

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 1198 0
Royal Children's Hospital - Herston
Recruitment postcode(s) [1] 7060 0
4006 - Herston

Funding & Sponsors
Funding source category [1] 287550 0
Self funded/Unfunded
Name [1] 287550 0
Address [1] 287550 0
Country [1] 287550 0
Primary sponsor type
Hospital
Name
Gastroenterology Department, Royal Children's Hospital - Brisbane
Address
Royal Children's Hospital Brisbane
Gastroenterology Department
Cnr Bramston Tce and Herston Rd,
Herston 4006 QLD.
Country
Australia
Secondary sponsor category [1] 286302 0
None
Name [1] 286302 0
Address [1] 286302 0
Country [1] 286302 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 289530 0
Children's Health Services QLD Human Research Ethics Committee
Ethics committee address [1] 289530 0
Level 3, Royal Children's Hospital (RCH) Foundation Bldg, Royal Children's Hospital. Cnr Bramston Tce and Herston Road, Herston. QLD. 4029

Ethics committee country [1] 289530 0
Australia
Date submitted for ethics approval [1] 289530 0
Approval date [1] 289530 0
12/03/2013
Ethics approval number [1] 289530 0
HREC/12/QRCH/226

Summary
Brief summary
This study aims to define the role of iron deficiency and iron deficiency anaemia in Paediatric Inflammatory Bowel Disease. Iron deficiency with or without anaemia is prevalent in inflammatory bowel disease (IBD) and an adverse impact on quality of life and cognitive function. These effects are likely to be equally if not more severe in the paediatric population. Correction of iron deficiency in IBD is difficult because of ongoing losses and because oral iron is poorly tolerated and bioavailability is impaired in states of inflammation. Given this, intravenous iron preparations are increasingly being used to correct iron deficiency. Previous preparations were complicated by a high rate of anaphylaxis and of iron toxicity which limited the doses administered leading to incomplete correction. We aim to show that the treatment of iron deficiency and iron deficiency anaemia in children using a newer, safer compound with no reported anaphylaxis nor iron toxicity, Ferric Carboxymaltose, is safe and effective in children and that treatment to correction of iron deficiency improves quality of life. This compound has been safely and effectively given, in corrective doses to over 10,000 patients aged 14 and above and there is no reason to indicate it is not equally safe and efficacious in younger children. We also aim to demonstrate improvements in cognitive function after correction of iron deficiency and that the administration of ferric carboxymaltose does not significantly increase markers of lipid peroxidation in paediatric inflammatory bowel disease. The patients included in this prospective open study will be children (six and older) treated for Inflammatory Bowel disease at the Royal Children's Hospital in Brisbane, with documented iron deficiency or iron deficiency anaemia (as defined by published guidelines). Baseline records of iron status (Ganzoni formula, FBC, IS) and quality of life will be obtained through blood tests and the IMPACT III QOL questionnaire (previously validated). A cognitive assessment will also be recorded by a qualified psychologist using a WISC-R. Treatment with Ferric Carboxymaltose will be undertaken (dose 15mg/kg body weight up to 500mg as per current hospital infusion guidelines for patients aged 14 and over. Following this, repeat iron status and quality of life information will be obtained at 8 weeks, with a repeat assessment of cognitive function. Primary endpoints will be defined as correction of total iron stores and improvement in cognitive function and QOL measures.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 41210 0
Dr Nicholas Carman
Address 41210 0
Gastroenterology Department
Level 1 Coles Bldg
Royal Children's Hospital
Cnr Bramston Tce and Herston Rd
Herston, QLD 4029
Country 41210 0
Australia
Phone 41210 0
+61736367887
Fax 41210 0
Email 41210 0
nicholas_carman@health.qld.gov.au
Contact person for public queries
Name 41211 0
Dr Nicholas Carman
Address 41211 0
Gastroenterology Department
Level 1 Coles Bldg
Royal Children's Hospital
Cnr Bramston Tce and Herston Rd
Herston, QLD 4029
Country 41211 0
Australia
Phone 41211 0
+61736367887
Fax 41211 0
Email 41211 0
nicholas_carman@health.qld.gov.au
Contact person for scientific queries
Name 41212 0
Dr Nicholas Carman
Address 41212 0
Gastroenterology Department
Level 1 Coles Bldg
Royal Children's Hospital
Cnr Bramston Tce and Herston Rd
Herston, QLD 4029
Country 41212 0
Australia
Phone 41212 0
+61736367887
Fax 41212 0
Email 41212 0
nicholas_carman@health.qld.gov.au

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