Haematology Trials - includes Bone Marrow Transplant, Myelodysplasic Syndrome and Other Haematology Trials

The following list provides a brief description of haematology trials that are recruiting participants in Western Australia. If you would like more information please follow the links provided, contact one of the trial sites or speak with your doctor.

Please note that this list is based on information provided to the Cancer Council by WA hospitals and may not include all clinical trials that are running in WA.

Where ‘N/A' appears - this means the lacking information has not been provided to date to the Cancer Council.

 

 

Bone Marrow Transplant

 

Reach 2

Registered Title A Phase III Randomized Open-label Multi-center Study of Ruxolitinib Versus Best Available Therapy in Patients With Corticosteroid-refractory Acute Graft vs. Host Disease After Allogeneic Stem Cell Transplantation
Purpose The purpose of the study is to assess the efficacy of Ruxolitinib when added to immunosuppression therapy in patients with Grade II-IV steroid refractory acute graft vs. host disease.
Lay Summary To evaluate the safety and efficacy of ruxolitinib compared to Best Available Therapy in patients with corticosteroid-refractory acute graft vs. host disease after allogeneic stem cell transplantation
WA Trial Sites

Fiona Stanley Hospital Haematology
Ph. (08) 6152 6540

Links  US National Library of Medicine

Acknowledgements:US National Library of Medicine

 

[Return to top]

 

Myelodysplasic Syndrome

CC-486-MDS-006 Myelodysplastic Disorders Study (Not Recruiting)

Registered Title

A Phase 2, International, Multicenter, Randomized, Open-label, Parallel Group to Evaluate the Efficacy and Safety of Cc-486 (Oral Azacitidine) Alone in Combination With Durvalumab (MEDI4736) in Subjects With Myelodysplastic Syndromes Who Fail to Achieve an Objective Response to Treatment With Azacitidine for Injection or Decitabine.

Purpose

Evaluate the safety and efficacy of CC-486 and Durvalumab in Subjects with Myelodysplastic Syndromes who failed to achieve an objective response post iHMA treatment.

Lay Summary

N/A

WA Trial Sites

Royal Perth Hospital logo

RPH Haematology
Ph. (08) 9224 2405

Links

US National Library of Medicine

 

MDS-MSC01 Myelodysplastic Disorders Study

Registered Title

Mesenchymal stromal cells as therapy for Myelodysplaisa of Low and Intermediate IPSS prognostic risk.

Purpose

N/A

Lay Summary

Myelodysplasia is a malignant disease of bone marrow failure. It occurs due to abnormal production of blood components (cells). It presents with low blood counts (anaemia, thrombocytopenia, neutropenia) and its complications include fatigue, bleeding, infections and transformation to acute myeloid leukaemia. Life expectancy is significantly reduced by this disease. Prognosis in MDS is classified from low to high risk based on the IPSS (WHO, 2001) which better predicts overall survival and progression to acute leukaemia. Low and intermediate-1 risk patients with MDS (considered for this study) would have median overall survival of 3.4-5.7 years. Standard care for patients with MDS is supportive (i.e. blood transfusions, treatment of infections). There are significant impacts to quality of life for this disorder due to frequent hospital presentations, transfusion, infections and a lack of treatment options.

WA Trial Sites

Royal Perth Hospital logo

RPH Haematology
Ph. (08) 9224 2405

Links

N/A

 

 

Other Haematology Trials

 

PCYC-1140-IM

Registered Title  Ibrutinib in Combination With Corticosteroids Versus Placebo in Combination With Corticosteroids in Subjects With New Onset Chronic Graft Versus Host Disease (cGVHD) (INTEGRATE)
Purpose  To evaluate the safety and efficacy of ibrutinib in combination with prednisone in subjects with newly diagnosed moderate to severe cGVHD.
Lay Summary  N/A
WA Trial Links

Fiona Stanley Hospital Haematology
Ph. (08) 6152 6540

Links  US National Library of Medicine

Acknowledgements: US National Library of Medicine

 

ALXN-1210-PNH-301 Paroxysmal Nocturnal Hemoglobinuria Study (Not Recruiting)

Registered Title

A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naive Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH).

Purpose

The purpose of this study is to assess ALXN1210 compared to eculizumab in adult patients with PNH who have not previously used a complement inhibitor.

Lay Summary

N/A

WA Trial Sites

Perth Blood Institute Logo

Perth Blood Institute
3/95 Monash Avenue, Nedlands, WA
info@pbi.org.au
Ph. (08) 9200 4904

Links

US National Library of Medicine

 

Aplastic Anaemia Registry

Registered Title

Aplastic Anaemia Registry (AA).

Purpose

The aims of the Aplastic Anaemia Registry are to: 1) Better define the incidence of AA in Australia; 2) Provide information on the range of therapeutic strategies being employed in the treatment of AA patients, including IST, HSCT and supportive care; 3) Explore factors influencing clinical outcomes; 4) Investigate the relationship of PNH clones to progress of disease and response to therapy; 5) Better define optimal management of AA patients Inform and inspire future hypothesis-driven research in this area.

Lay Summary

N/A

WA Trial Sites

Sir Charles Gairdner Hospital Logo

Haematology Care Centre
Sir Charles Gairdner Hospital
Ph. (08) 6383 3207
Fax (08) 9346 4432
Email: louise.hay@health.wa.gov.au

Royal Perth Hospital logo

RPH Haematology
Ph. (08) 9224 2405

Fiona Stanley Hospital Haematology
Ph. (08) 6152 6540

Links

N/A

 

APMAT1 Microangiopathic Thrombocytopenia Study

Registered Title

A Multi-centre, Observational Study by the Asian-Pacific Microangiopathic Thrombocytopenia (APMAT) Network To Determine The Clinical Characteristics, Laboratory Features, Treatments, and Clinical Outcomes in Patients with Microangiopathic Thrombocytopenia.

Purpose

This is an observational study of patients who have previously been diagnosed and treated for Microangiopathic Thrombocytopenia (MAT). The study will collect clinical and laboratory data to assess the disease characteristics, laboratory features, treatments, and clinical outcome.

Lay Summary

N/A

WA Trial Sites

Perth Blood Institute Logo

Perth Blood Institute
3/95 Monash Avenue, Nedlands, WA
info@pbi.org.au
Ph. (08) 9200 4904

Links

Australian New Zealand Clinical Trials Registry

 

 

C16011 - A study of MLN9708 in patients with Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis

Registered Title

A Phase 3 Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of Dexamethasone Plus MLN9708 or Physician's Choice of Treatment Administered to Patients with Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis.

Purpose

The purpose of this study is to find out if MLN9708 plus dexamethasone improves against systemic light chain amyloidosis better than the physician's choice chemotherapy treatment. Physician's choice chemotherapy treatment is a treatment regimen that is commonly used to treat amyloidosis patients. The study will also determine if MLN9708 plus dexamethasone can change the chance that the amyloidosis will cause heart and/or kidneys to begin to work poorly needing care in the hospital or of dying.

Another purpose of this study is to see how differences in DNA, RNA and proteins related to the disease may influence the way people respond to MLN9708. This information may be used by the Sponsor of this study, Millennium Pharmaceuticals, its agents and its affiliated companies for research related to analysis and development of MLN9708 and associated disease states, for example:

  • to develop a better understanding of how people's genes affect the safety and effectiveness of MLN9708

  • to help develop new ways to monitor and treat cancer

  • to generate information needed for research, development and regulatory approval of diagnostic tests related to diseases or conditions that MLN9708 might treat

Lay Summary

This is a phase 3, randomized, controlled, open-label, multicenter study of the oral formulation of dexamethasone plus MLN9708 compared with treatment chosen by the investigator from a prespecified list of regimens available in clinical practice. Treatment options will include: dexamethasone alone, dexamethasone plus an alkylating agent (melphalan or cyclophosphamide), or dexamethasone plus an immunomodulatory drug (IMiD, thalidomide or lenalidomide) in patients with relapsed or refractory AL amyloidosis. Crossover to the investigational treatment arm is not permitted during participation in this study.

WA Trial Sites

Sir Charles Gairdner Hospital Logo

Haematology Care Centre
Sir Charles Gairdner Hospital
Ph. (08) 6383 3207
Fax (08) 9346 4432
Email: louise.hay@health.wa.gov.au

Links

US National Library of Medicine

Acknowledgements: US National Library of Medicine

 

HERCULES Acquired Thrombotic Thrombocytopenic Purpura Study

Registered Title

A Phase III Double-blind, Randomized, Parallel Group, Multicenter Placebo-controlled Trial to Study the Efficacy and Safety of Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura.

Purpose

The study is a phase III, double blind, placebo-controlled, randomized study to evaluate the efficacy and safety of caplacizumab treatment in more rapidly curtailing ongoing microvascular thrombosis when administered in addition to standard of care treatment in subjects with an acute episode of acquired TTP.

Lay Summary

N/A

WA Trial Sites

Royal Perth Hospital logo

RPH Haematology
Ph. (08) 9224 2405

Links

US National Library of Medicine

 

MEPO Study

Registered Title

Mepolizumab Compassionate Use Supply Program (SB240563.MHE104317).

Purpose

The purpose of this programme is to provide a treatment option to patients with life-threatening Hypereosinophilic Syndrome (HES) whose symptoms are not controlled with other therapies and to provide continued access to mepolizumab for subjects who received clinical benefit in Study MHE100901 following termination of the study.

Lay Summary

Eligible patients will receive a supply of Mepolizumab for monthly infusions with up to 10mg/kg (maximum dose 750mg or 10mg/kg if body weight less than 45kg) Mepolizumab IV for an initial three months of treatment. The duration and frequency of additional treatment with Mepolizumab will be determined based on the subject's response to the initial three months of treatment as demonstrated by significant lowering of eosinophil level and/or decreased signs and symptoms of HES.

WA Trial Sites

Sir Charles Gairnder Hospital Logo

Haematology Care Centre
Sir Charles Gairdner Hospital
Ph. (08) 6383 3207
Fax (08) 9346 4432
Email: louise.hay@health.wa.gov.au

Links

US National Library of Medicine

Acknowledgements: US National Library of Medicine

 

 MPN01 Registry

Registered Title

Australasian Leukaemia and Lymphoma Group Myeloproliferative Neoplasms Registry.

Purpose

The aim of this registry is collect information on patients in Australia and New Zealand who have been diagnosed with a myeloproliferative neoplasm (polycythaemia vera, essential thrombocythaemia, primary myelofibrosis, chronic eosinophilic leukaemia) or a related disorder (hypereosinophilic syndrome, refractory anaemia with ringed sideroblasts associated with a marked thrombocytosis) and to understand the nature of the disease in the local area.

Lay Summary

The registry is an observational study and participating does not mandate the performance of investigations and does not recommend any treatment. The registry will collect information at the time the blood disorder was diagnosed which will include information about the haematological disease, the presence of comorbidities (other illnesses), family history of blood disorders, complications present in the disease and any therapies started.

More information will be collected for the registry every 12 months on the progress of the disease, its therapy and any complications that may have developed for as long as the registry remains open.

Patients will be offered the opportunity to complete a quality-of-life assessment (MPNSAF: myeloproliferative neoplasm symptom assessment form) within the first three months of diagnosis and on an annual basis.
There will not be any requirement to attend any additional appointments through participation in the MPN01 registry.

Patients will be asked to provide consent to obtain additional blood, bone marrow & saliva samples If they wish to take part in the optional scientific studies.

WA Trial Sites

Sir Charles Gairdner Hospital logo

Haematology Care Centre
Sir Charles Gairdner Hospital
Ph. (08) 6383 3207
Fax (08) 9346 4432
Email: louise.hay@health.wa.gov.au

Links

Australian New Zealand Clinical Trials Registry

 

PNH Registry

Registered Title

A global, observational, non-interventional study collecting effectiveness, safety and quality of life data on patients with Paroxysmal Nocturnal Haemoglobinuria (PNH) Disease.

Purpose

The Registry will evaluate safety data specific to the use of Soliris,and will collect data to characterize the progression of PNH as well as clinical outcomes, mortality and morbidity in Soliris and non-Soliris treated patients. The registry also aims to raise PNH awareness in the medical community and subject/potential subject population.

Lay Summary

The registry will enrol patients treated with Soliris for any reason, as well as patients with PNH. Clinical data will be collected from patients enrolled in this study. Participants will also be required to complete six monthly quality of life questionnaires.

WA Trial Sites

Sir Charles Gairdner Hospital Logo

Haematology Care Centre,
Sir Charles Gairdner Hospital         
Ph. (08) 6383 3207
Fax (08) 9346 4432
Email: louise.hay@health.wa.gov.au

Fiona Stanley Hospital Logo

Fiona Stanley Hospital Haematology
Ph. (08) 6152 6540

Perth Blood Institute Logo

Perth Blood Institute
3/95 Monash Avenue, Nedlands, WA
info@pbi.org.au
Ph. (08) 9200 4904

Links

PNH Registry website

US National Library of Medicine

Acknowledgements: US National Library of Medicine

 

TTP Registry

Registered Title

Thrombotic Thrombocytopenic Purpura (TTP) Registry.

Purpose

The aims of the TTP Registry are to:
  • Better define the incidence, natural history, specific clinical characteristics, and clinical outcome of patients with TTP and HUS.
  • Provide information on the range of therapeutic strategies employed in the treatment of TTP and HUS patients.
  • Explore factors influencing clinical outcomes.
  • Help define optimal management of patients with TTP and HUS.
  • Inform and inspire future hypothesis-driven research in this area.

Lay Summary

The TTP Registry will be a register of patients who develop TTP in any clinical setting. Clinical data collection will be undertaken by clinicians in specialist units at participating hospitals. Data management and analysis will be undertaken by the Department of Epidemiology and Preventive Medicine (DEPM), Monash University and interpreted with the input of Transfusion Medicine Specialists at ARCBS and specialist clinicians on the Registry Steering Committee. The Registry began collecting data in December 2008.

Patients are identified either by the treating clinician, or by ARCBS clinicians as a result of referral for provision of blood components for therapy. Patient liaison and registration will take place in participating hospitals. Recruitment strategies take advantage of the fact that all patients will require blood component therapy provided by ARCBS, and patients are largely managed by a small group of specialised clinicians in a limited number of major centres with apheresis facilities. Registry staff will maintain close interaction with key individuals working in relevant hospital clinical care areas to ensure notification of all patients.

WA Trial Sites

Sir Charles Gairnder Hospital Logo

Haematology Care Centre
Sir Charles Gairdner Hospital
Ph. (08) 6383 3207
Fax (08) 9346 4432
Email: louise.hay@health.wa.gov.au

Links

N/A

 

[Return to top]

 

 

[Return to List of Clinical Trials]