A phase 2 study of the efficacy and safety of Deferasirox administered at early iron loading in patients with transfusion-dependent myelodysplastic syndromes

ISRCTN ISRCTN62162141
DOI https://doi.org/10.1186/ISRCTN62162141
EudraCT/CTIS number 2011-004559-38
Secondary identifying numbers 13706
Submission date
05/07/2013
Registration date
05/07/2013
Last edited
14/02/2020
Recruitment status
No longer recruiting
Overall study status
Completed
Condition category
Cancer
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data

Plain English summary of protocol

http://www.cancerresearchuk.org/cancer-help/trials/a-study-looking-drug-deferasirox-people-myelodysplastic-syndromes-de-iron

Contact information

Dr Dominic Culligan
Scientific

Cornhill Road
Aberdeen
AB25 2ZN
United Kingdom

Email dominic.culligan@nhs.net

Study information

Study designNon-randomised; Interventional; Design type: Process of Care, Treatment
Primary study designInterventional
Secondary study designNon randomised study
Study setting(s)Hospital
Study typeTreatment
Participant information sheet Not available in web format, please use contact details to request a participant information sheet
Scientific titleA phase 2 study of the efficacy and safety of Deferasirox administered at early iron loading in patients with transfusion-dependent myelodysplastic syndromes
Study acronymDeferasirox for early iron loading in transfusion-dependant MDS
Study objectivesMyelodysplastic 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(s)12/NE/0220
Health condition(s) or problem(s) studiedTopic: National Cancer Research Network, Blood; Subtopic: Haematological Oncology, Blood (all Subtopics); Disease: Unknown Primary Site, Non-malignant haematology
InterventionDeferasirox, oral deferasirox for patients with transfusion dependant, low risk MDS and early iron loading; Study entry: registration only
Intervention typeDrug
Pharmaceutical study type(s)
PhasePhase II
Drug / device / biological / vaccine name(s)Deferasirox
Primary outcome measureTime to reach a ferritin of 1500 ug/l; Timepoint(s): Treatment is for 12 months and follow up for 24 months
Secondary outcome measuresNot provided at time of registration
Overall study start date25/01/2013
Completion date25/01/2015

Eligibility

Participant type(s)Patient
Age groupAdult
Lower age limit18 Years
SexBoth
Target number of participantsPlanned Sample Size: 54; UK Sample Size: 54
Total final enrolment13
Key inclusion criteria1. 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
Key exclusion criteria1. 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
Date of first enrolment25/01/2013
Date of final enrolment25/01/2015

Locations

Countries of recruitment

  • Scotland
  • United Kingdom

Study participating centre

Cornhill Road
Aberdeen
AB25 2ZN
United Kingdom

Sponsor information

University of Birmingham (UK)
University/education

Institute for Cancer studies
Edgbaston
Birmingham
B15 2TT
England
United Kingdom

ROR logo "ROR" https://ror.org/03angcq70

Funders

Funder type

Charity

Leukaemia and Lymphoma Research (Grant Codes: 11019)
Private sector organisation / Other non-profit organizations
Location
United Kingdom
Novartis Oncology (Switzerland); Grant Codes: CICL670AGB05T

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to shareNo
IPD sharing plan summaryNot provided at time of registration
Publication and dissemination planNot provided at time of registration
IPD sharing plan

Study outputs

Output type Details Date created Date added Peer reviewed? Patient-facing?
Plain English results No Yes
Basic results 21/06/2019 No No
HRA research summary 28/06/2023 No No

Editorial Notes

14/02/2020: Cancer Research UK lay results summary link added to Results (plain English).
21/06/2019: Added clinicaltrialsregister.eu link to basic results (scientific). Added total final enrollment.
12/01/2017: No publications found, verifying study status with principal investigator.