Clinical trial NCT02546154: Time to Relapse of Iron Deficiency Anaemia After Standard Treatment With The Intravenous Iron (Monofer®)
Official title: A Non-interventional Study of the Time to Relapse of Iron Deficiency Anaemia After Standard Treatment With the Intravenous Iron Monofer® (10% Iron Isomaltoside 1000)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Iron Deficiency Anaemia
Keywords: Iron
Anaemia
Anaemia, Iron-Deficiency
Kidney Diseases
Inflammatory Bowel Diseases
Fatigue
Lead sponsor: Pharmacosmos A/S
Sponsor class: INDUSTRY
Collaborators: name: BioStata
First posted: 2015-09-10
Enrollment: 359
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients ≥18 years of age diagnosed with iron deficiency anaemia as a consequence of CKD or IBD.

Brief summary: The objective is to monitor and quality assure the efficacy, including effects on quality of life, and safety of Monofer® in Chronic Kidney Disease and Inflammatory Bowel Disease patient populations when Monofer® is used according to the Monofer® label (Summary of Product Characteristics, SPC) in current clinical practice and where standard routines are being followed.

Detailed description: Intravenous (IV) iron is a well-tolerated and efficacious treatment of iron deficiency anaemia in conditions such as Chronic Kidney Disease (CKD) and Inflammatory Bowel Disease (IBD). Several studies in CKD and IBD patients have shown that IV iron is superior to oral iron, most likely explained by decreased uptake and poor compliance (due to gastrointestinal side-effects) when using oral iron.

The patented Iron Isomaltoside 1000 (Monofer®) has been developed to overcome the current limitations of IV iron drugs with regards to safety and convenience of use. The possibility to administer Monofer® at high single doses (up to 20 mg/kg) reduces the number of treatment visits needed for full iron correction, which is cost-effective, resource and time-sparing, and a reduced drug exposure frequency decreases the risk of side-effects such as infusion reactions.

The objective of the study is to monitor and quality assure the efficacy, including effects on quality of life, and safety of Monofer® in CKD and IBD patient populations when Monofer® is used according to the Monofer® label (Summary of Product Characteristics, SPC) in current clinical practice and where standard routines are being followed. The scientific rationale being to fulfill a need for systematic information/auditing on applied practice including both short and long-term experience with the use of IV iron in different hospital settings and in current clinical practice. The outcome will provide an evidence base for optimised treatment procedures in terms of safety and efficacy.

The total duration of the study per site is approximately 21 months, which includes a 6 months enrollment period, a prospective observation period of at least 12 months, and a period of maximum 3 months prior to the last blood test. Patients will only attend hospital visits planned as part of their standard treatment and they will receive treatment as a part of standard care and according to the doctor's discretion. The number of patient visits depends on the number of Monofer® treatment courses needed during the study period. Each patient can receive one or more treatment courses during 12 months after informed consent. The last blood test will be taken after the last Monofer® treatment course which might occur 13-15 months after informed consent. Study termination will occur once the 12 months observational period has been completed for all patients and the last blood test has been collected from the last Monofer® treated patient in the study. Each treatment course can consist of one or more Monofer® administrations. For each administration of Monofer® either intravenous infusion or injection can be used. Pre- and post-treatment blood tests according to standard treatment and quality of life assessments for fatigue symptoms (FACIT-Fatigue and IBD-F Scales) are a part of the Monofer® treatment course. Laboratory assessments, i.e. anaemia work-up/treatment evaluation, shall be a part of local standard practice. The protocol does not accept any additional samples outside current local standard practice to be taken.

Clinical data management will be performed in accordance with applicable standards and data cleaning procedures. The collected data will systematically be entered into an electronic case report form (eClinicalOS, myEDC, license by BioStata Aps Denmark). The sources of information are the relevant treatment and laboratory results obtained from patient records as well as the quality of life questionnaires for fatigue symptoms. The data will be evaluated by the Pharmacosmos Medical Affairs teams and an external Data Management vendor located in a country within the European Union.

Eligibility criteria: Inclusion Criteria:

* Patients diagnosed with iron deficiency anaemia as a consequence of CKD or IBD (on the basis of local definition or clinical judgement), treated on the doctor's discretion with Monofer® as standard treatment according to current practice

Exclusion Criteria:

* Patients diagnosed with both CKD and IBD

Primary outcomes:
measure: Time to relapse of iron deficiency anaemia
timeFrame: From screening until 12 months
Secondary outcomes:
measure: Change in scores for fatigue symptoms
timeFrame: Immediately before to earliest 4 weeks after each treatment course during 12 months
measure: Change in anaemia-related blood parameter levels (haemoglobin, iron parameters)
timeFrame: Immediately before to earliest 4 weeks after each treatment course during 12 months
measure: IV iron dose (total needed dose per treatment course)
timeFrame: From screening until 12 months
measure: Number and seriousness of Adverse Drug Reactions
timeFrame: From screening until 12 months
Other outcomes:
Locations:
city: Exeter
country: United Kingdom
facility: Royal Devon and Exeter Hospital NHS Foundation Trust
Study officials:
affiliation: Pharmacosmos A/S
name: Sylvia Simon, PhD
role: STUDY_CHAIR
affiliation: Royal Devon and Exeter Hospital NHS Foundation Trust
name: Jason Moore, BMedSci, BMBS, FRCP
role: PRINCIPAL_INVESTIGATOR
References:
citation: Kalra PA, Bhandari S, Spyridon M, Davison R, Lawman S, Mikhail A, Reaich D, Pritchard N, McCafferty K, Moore J. NIMO-CKD-UK: a real-world, observational study of iron isomaltoside in patients with iron deficiency anaemia and chronic kidney disease. BMC Nephrol. 2020 Dec 10;21(1):539. doi: 10.1186/s12882-020-02180-2.