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 |
| Clinical Trials Information System (CTIS) | 2011-004559-38 |
| Protocol serial number | 13706 |
| Sponsor | University of Birmingham (UK) |
| Funders | Leukaemia and Lymphoma Research (Grant Codes: 11019), Novartis Oncology (Switzerland); Grant Codes: CICL670AGB05T |
- 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
Contact information
Dr Dominic Culligan
Scientific
Scientific
Cornhill Road
Aberdeen
AB25 2ZN
United Kingdom
| dominic.culligan@nhs.net |
Study information
| Primary study design | Interventional |
|---|---|
| Study design | Non-randomised; Interventional; Design type: Process of Care, Treatment |
| Secondary study design | Non randomised study |
| Study type | Participant information sheet |
| 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 |
| Study acronym | Deferasirox for early iron loading in transfusion-dependant MDS |
| Study objectives | 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(s) | 12/NE/0220 |
| Health condition(s) or problem(s) studied | 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 / device / biological / vaccine name(s) | Deferasirox |
| Primary outcome measure(s) |
Time to reach a ferritin of 1500 ug/l; Timepoint(s): Treatment is for 12 months and follow up for 24 months |
| Key secondary outcome measure(s) |
Not provided at time of registration |
| Completion date | 25/01/2015 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | All |
| Target sample size at registration | 54 |
| Total final enrolment | 13 |
| Key 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 |
| Key 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 |
| Date of first enrolment | 25/01/2013 |
| Date of final enrolment | 25/01/2015 |
Locations
Countries of recruitment
- United Kingdom
- Scotland
Study participating centre
Cornhill Road
Aberdeen
AB25 2ZN
United Kingdom
AB25 2ZN
United Kingdom
Results and Publications
| Individual participant data (IPD) Intention to share | No |
|---|---|
| IPD sharing plan summary | Not provided at time of registration |
| IPD sharing plan |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Basic results | 21/06/2019 | No | No | ||
| HRA research summary | 28/06/2023 | No | No | ||
| Participant information sheet | Participant information sheet | 11/11/2025 | 11/11/2025 | No | Yes |
| Plain English results | No | Yes |
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.
