Condition category
Cancer
Date applied
05/07/2013
Date assigned
05/07/2013
Last edited
22/08/2013
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Contact information

Type

Scientific

Primary contact

Dr Dominic Culligan

ORCID ID

Contact details

Cornhill Road
Aberdeen
AB25 2ZN
United Kingdom
dominic.culligan@nhs.net

Additional identifiers

EudraCT number

2011-004559-38

ClinicalTrials.gov number

Protocol/serial number

13706

Study information

Scientific title

Acronym

Deferasirox for early iron loading in transfusion-dependant MDS

Study hypothesis

Myelodysplastic Syndromes (MDS) cause a failure of the bone marrow, which does not produce enough blood cells (red cells, white cells and platelets). This is because the bone marrow contains too many abnormal cells (dysplastic cells) which function poorly.

Many patients with MDS do not produce enough red blood cells, which leads to anaemia. This means that they receive regular blood transfusions to treat the anaemia and alleviate symptoms. However, blood is rich in iron and repeated transfusions may cause a build-up of excess iron. Although iron is an essential part of the blood, an excess of iron may affect the way in which the organs in the body function. This includes the liver and heart. This situation is called iron overload.

The aim of this study is to test how effective, safe and tolerable a drug called Deferasirox (also called Exjade®) is when used to treat rising iron levels in patients with MDS. The study treatment will aim to control the iron levels in the blood, which steadily increase after receiving regular blood transfusions. It is not intended to treat MDS. Normally doctors will wait until the level of iron in the blood significantly increases before considering starting treatment for iron overload, but in this study we would like to give Deferasirox treatment early rather than waiting for the iron levels to rise until a high level is reached and organ damage begins. In summary, we are looking at the feasibility of starting treatment early, before overload begins.

Ethics approval

12/NE/0220

Study design

Non-randomised; Interventional; Design type: Process of Care, Treatment

Primary study design

Interventional

Secondary study design

Non randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Condition

Topic: National Cancer Research Network, Blood; Subtopic: Haematological Oncology, Blood (all Subtopics); Disease: Unknown Primary Site, Non-malignant haematology

Intervention

Deferasirox, oral deferasirox for patients with transfusion dependant, low risk MDS and early iron loading; Study entry: registration only

Intervention type

Drug

Phase

Phase II

Drug names

Deferasirox

Primary outcome measures

Time to reach a ferritin of 1500 ug/l; Timepoint(s): Treatment is for 12 months and follow up for 24 months

Secondary outcome measures

Not provided at time of registration

Overall trial start date

25/01/2013

Overall trial end date

25/01/2015

Reason abandoned

Eligibility

Participant inclusion criteria

1. At least 18 years old
2. Written informed consent
3. MDS with:
3.1. Baseline haemoglobin concentration < 11 g/dl and clinically requiring red cell transfusion with a frequency of at least 2 units every 6 weeks for the receding 12 week period.
3.2. Serum ferritin > 300 ug/l but < 1000 ug/l in absence of ongoing inflammation (CRP < 3 x ULN)
3.3. Serum creatinine < 1.2 x ULN and/or creatinine clearance > 40 ml/min
3.4. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) < 2.5 ULN
3.5. International Prognostic Scoring System (IPSS) Low/INT-1 previously untreated or having failed a therapeutic trail of erythropoetic stimulating agents (ESA) or other active MDS drug therapy, or alternatively lost their response to such therapy
3.6. IPSS INT-2 with <10% blasts and lacking a complex karyotype or monosomy 7 (and with stable blood counts from diagnosis to study entry)
Target gender: male & female

Participant type

Patient

Age group

Not Specified

Gender

Both

Target number of participants

Planned Sample Size: 54; UK Sample Size: 54

Participant exclusion criteria

1. Active treatment for MDS, including erythropoetic stimulating agents (ESA), 5-azacitidine, antilymphocyte globulin and low dose chemotherapy such as cytarabine during the trial and within the last 8 weeks
2. Life expectancy of less than 1 year
3. Known HIV positive
4. Active infection
5. Use of prior investigational agents within 6 weeks
6. Pregnancy or lactation
7. Other severe concurrent medical illness that limit the patient’s prognosis to <1 year, or psychiatric disorders
8. Concurrent active or previous malignancy, within the last 3 years, except controlled, localised prostate cancer on hormone therapy or basal cell carcinoma or cervical carcinoma in situ or completely resected colonic polyps carcinoma in situ
9. Ongoing inflammation as measured by C-reactive protein (CRP) > 3 x ULN
10. Serum creatinine > 1.2 x ULN and/or creatinine clearance <40 mls/min
11. ALT or AST >2.5 ULN
12. History of drug/alcohol abuse or non-compliance

Recruitment start date

25/01/2013

Recruitment end date

25/01/2015

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Cornhill Road
Aberdeen
AB25 2ZN
United Kingdom

Sponsor information

Organisation

University of Birmingham (UK)

Sponsor details

Institute for Cancer studies
Edgbaston
Birmingham
B15 2TT
United Kingdom

Sponsor type

University/education

Website

Funders

Funder type

Charity

Funder name

Leukaemia & Lymphoma Research (UK) Grant Codes: 11019

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Funder name

Novartis Oncology (Switzerland); Grant Codes: CICL670AGB05T

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes