Plain English Summary
Trial website
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
A phase 2 study of the efficacy and safety of Deferasirox administered at early iron loading in patients with transfusion-dependent myelodysplastic syndromes
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 Deferasirox treatment is given early rather than waiting for the iron levels to rise until a high level is reached and organ damage begins. In summary, this study is 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 study
Trial setting
Hospitals
Trial type
Treatment
Patient information sheet
Not available in web format, please use contact details to request a participant 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 measure
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 (if study stopped)
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
Adult
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 patients 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
Funders
Funder type
Charity
Funder name
Leukaemia and Lymphoma Research (Grant Codes: 11019)
Alternative name(s)
Funding Body Type
private sector organisation
Funding Body Subtype
other non-profit
Location
United Kingdom
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
Basic results (scientific)
Publication list