A phase 1 study to assess the safety of short-course treatment with venetoclax prior to non-myeloablative stem cell transplantation for patients with haematological malignancies.

ACTRN 12622000308796

Brief Summary

The purpose of this study is to investigate the safety of oral venetoclax treatment prior to fludarabine and cyclophosphamide non-myeloablative conditioning allogeneic stem cell transplantation for patients with haematological malignancies.

Intervention/Treatment

• Drug: Venetoclax
• Drug: Fludarabine
• Drug: Cyclophosphamide
• Allogeneic stem cell transplantation (alloSCT).

Inclusion Criteria

Patients are eligible for inclusion if all of the following criteria are met:

1. Age greater than or equal to 18 years.
2. Planned to undergo allogeneic stem cell transplantation for one of the following haematological malignancies: acutenew, recent, comes with an urgent or significant sense, is sudden, sharp leukaemiacancer of blood and/or blood forming tissues (including myeloid and/or lymphoid lineage or biphenotypic), myelodysplastic syndrome, chronica long-lasting disease that changes slowly over time lymphocytic leukaemia (CLL), B-cell non-Hodgkin lymphomacancers of the lymphatic system (NHL) and plasma cell myeloma.
3. Physician preference for a non-myeloablative conditioning regimen.
4. Available 10/10 HLA-matched related or unrelated haematopoietic stem cell donor.
5. Transplantation to be performed from a peripheral bloodthe red bodily fluid that transports oxygen and other nutrients around the body stem cell source.
6. Adequate renal and hepatic function at screeningtesting for cancer or conditions that can lead to cancer before symptoms appear, also known as cancer screening as follows:
   • Calculated creatinine clearance >50ml/min as measured by Cockroft Gault formula.
   • AST and ALT less than or equal to 3.0 x ULN.
   • Bilirubin less than or equal to 1.5 x ULN (except patients with Gilbert’s Syndrome).
7. Able to tolerate oral medications.
8. Disease status at the time of transplantation as follows:
   • Acute leukaemia in complete morphologic remissiona reduction or absence of symptoms in disease, can be partial or complete.
   • Myelodysplastic syndrome with less than 10% bone marrowsoft, spongy tissue found in bones that makes blood cells blasts.
   • CLL in complete remission (CR), partial response (PR) or PR with lymphocytosis.
   • NHL in CR or PR.
   • Plasma cell myeloma in CR, very good partial response (VGPR) or PR within 3 months of prior autologous stem cell transplantation as part of a tandem auto-allo transplant approach.
9. ECOG performance status 0-1.