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Trial of Deflazacort, a steroid which is not currently commercially available in Australia, for boys with DMD who have failed Prednisone trials

The study will assess if treatment with Prednisolone has a positive effect in maintaining lung function in non-ambulant boys with Duchenne muscular dystrophy

To see which regimen of Prednisone - either a low daily dose or a high dose given on the two days of the weekend - gives the best positive effects (improvement in muscle strength) while minimising adverse effects (such as weight gain and behavioural changes)

Sucrose when given to newborn infants has analgesic properties, especially when used for the relief of procedural pain. The calming effects of the sweet taste, cause the release of naturally occuring endorphins (morphine- like substances). The aim of this study is to show that sucrose has the same pain relieving effects in the infant of a substance dependent mother, compared to healthy (matched) infant.

It has been found that if lymphoma recurs after transplantation it often does so in sites involved prior to transplantation. Radiotherapy can prevent some of these relapses, and may improve the overall results of transplantation. Radiotherapy is often given only to bulky sites (i.e more than 5-10 cm in size) of lymphoma following transplantation, but the optimal dose and area of radiotherapy is not known. It is possible that treating all the areas of lymphoma (including non-bulky areas) may be more effective. In this research study, radiotherapy will be given to all the areas known to be affected by lymphoma (other than bone marrow). The aims of this study are to: (1) assess the ability of radiotherapy to reduce the risk of relapse following transplantation, and (2) carefully evaluate the side effects of adding radiotherapy to transplantation.

Primary Objective: Investigation of the safety and tolerability of Glivec in combination with induction chemotherapy for blast-phase chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia Secondary Objective(s): (1) Investigation of the efficacy of Glivec combined with induction chemotherapy in Ph+ acute leukaemias (2) Biochemical evaluation of Abl kinase suppression by Glivec in leukaemic blasts Patients aged 16-70 with CML in blast crisis or Ph+ ALL All patients will receive Glivec 600 mg orally once daily, beginning 7 days prior to induction chemotherapy. Subsequent therapy will be delivered on one of three arms: (1) CML in myeloid blast crisis : Chemotherapy will consist of induction with idarubicin and standard-dose cytarabine, followed by consolidation with 2 cycles of intermediate-dose cytarabine. Glivec will be administered without interruption in combination with the chemotherapy and then continued as a single agent until relapse or unacceptable toxicity. (2) CML in lymphoid blast crisis and relapsed Ph+ ALL: Chemotherapy will consist of 8 courses of hyper-CVAD and Glivec will be administered as in Arm 1. (3) De-novo Ph+ ALL: Chemotherapy will be given according to the French cooperative group ALL protocol (LALA 94). Glivec will be continued synchronously with induction, consolidation and maintenance chemotherapy, except for an interruption during cranial radiation.

Acute promyelocytic leukemia (APL) is a unique subtype of acute myeloid leukemia that is characterized by distinct clinical and laboratory abnormalities. It is associated with a striking risk of early death due to bleeding. Fortunately, the outcome for patients with APL has improved dramatically following the introduction of all-trans retinoic acid (ATRA) and its combination with chemotherapeutic drugs such as idarubicin. Patients with a white cell count > 10 x 109/L at diagnosis are at particularly increased risk of early death and of relapse (disease recurrence). Arsenic trioxide (ATO) has proved to be even more effective than ATRA as a single agent, and is now routinely used for the treatment of the 20%–30% of patients who relapse after initial treatment with ATRA and chemotherapy. ATO has a side-effect profile that is substantially different from both ATRA and chemotherapy. In the APML4 trial, members of the Australasian Leukaemia and Lymphoma Group (ALLG) combined the 3 most active drugs used for APL (ATRA, idarubicin, and ATO) in induction, and then consolidated the responses with 2 cycles of ATRA and ATO without chemotherapy. This strategy helped reduce the total amount of chemotherapy compared with many other protocols available, in the hope of reducing long-term side effects such as bone marrow damage and heart damage. Two years of maintenance was used as per the ALLG standard practice for APL. The results were very impressive. One hundred and twenty-four evaluable patients were enrolled between Nov 2004 and Sep 2009, and the outcomes were reported in 2 publications (Blood 2012, Lancet Haematology 2015). The early death rate was 3%, and 95% of patients achieved complete remission after induction with ATRA, ATO and idarubicin. All 112 patients who started consolidation achieved molecular remission (no detectable leukaemia by a very sensitive molecular assay). The long term results that were reported in Lancet Haematology showed that the relapse rate at 5 years was only 5%. Patients traditionally regarded as at very high risk of relapse had the same low relapse rate as patients who were in the standard risk category, and this was a major achievement of the APML4 treatment plan. Overall, 94% of patients were still alive at 5 years. When the results were compared with the ALLG’s previous APML3 trial (which did not include ATO), the results were substantially better with less relapses and better overall survival. Although side effects occurred, as in every clinical trial for leukaemia, the overall side effect profile was widely acknowledged as acceptable (ie. not excessive).