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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]

nil

Universal Trial Number (UTN)

Trial acronym

Linked study record

Health condition

Health condition(s) or problem(s) studied:

Paediatric Inflammatory Bowel Disease

Iron deficiency anaemia

Iron deficiency without anaemia

Condition category

Condition code

Oral and Gastrointestinal

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]

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]

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]

At eight weeks following intravenous iron infusion

Primary outcome [2]

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]

At eight weeks following intravenous iron infusion.

Secondary outcome [1]

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]

At baseline, prior to iron infusion.

Secondary outcome [2]

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]

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

Sex

Both males and females

Can healthy volunteers participate?

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

5/08/2013

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]

Royal Children's Hospital - Herston

Recruitment postcode(s) [1]

4006 - Herston

Funding & Sponsors

Funding source category [1]

Self funded/Unfunded

Name [1]

Address [1]

Country [1]

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]

None

Name [1]

Address [1]

Country [1]

Ethics approval

Ethics application status

Approved

Ethics committee name [1]

Children's Health Services QLD Human Research Ethics Committee

Ethics committee address [1]

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]

Australia

Date submitted for ethics approval [1]

Approval date [1]

12/03/2013

Ethics approval number [1]

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

Dr Nicholas Carman

Address

Gastroenterology Department
Level 1 Coles Bldg
Royal Children's Hospital
Cnr Bramston Tce and Herston Rd
Herston, QLD 4029

Country

Australia

Phone

+61736367887

Fax

[email protected]

Contact person for public queries

Name

Nicholas Carman

Address

Gastroenterology Department
Level 1 Coles Bldg
Royal Children's Hospital
Cnr Bramston Tce and Herston Rd
Herston, QLD 4029

Country

Australia

Phone

+61736367887

Fax

[email protected]

Contact person for scientific queries

Name

Nicholas Carman

Address

Gastroenterology Department
Level 1 Coles Bldg
Royal Children's Hospital
Cnr Bramston Tce and Herston Rd
Herston, QLD 4029

Country

Australia

Phone

+61736367887

Fax

[email protected]

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

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