Uproleselan Study

Uproleselan Study

A Phase III Randomized, Double-Blind Trial to Evaluate the Efficacy of Uproleselan Administered with Chemotherapy versus Chemotherapy Alone in Patients with Relapsed/Refractory Acute Myeloid Leukemia

Disease:

Acute Myeloid Leukaemia (AML)

Summary:

The bone marrow is like a factory, producing all types of blood cells. This process begins with immature stem cells in the bone marrow, and results in production of mature red cells, platelets and different types of white cells.
Acute myeloid leukaemia (AML) is an aggressive cancer of the bone marrow. In AML, there is an overproduction of immature white cells, known as blasts. These immature cells do not function properly to prevent and fight infection, and can result in production of inadequate numbers of red cells and platelets, which in turn may lead to anaemia, bleeding and bruising.
A molecule called E-selectin, with a variety of functions, is expressed in the circulatory system during periods of inflammation and stress and is permanently expressed in the bone marrow. In people with AML, blast cells have potential to bind to E-selectin within the bone marrow, and may encourage resistance of the leukaemia to chemotherapy. The presence of E-selectin in the circulation can also contribute to some chemotherapy side effects (eg. sore mouth, known as mucositis).

Uproleselan (GMI-1271) is a drug which works as an E-selectin inhibitor. It prevents binding of the AML blast cells to E-selectin, and inhibits the activity of E-selectin in the circulation. It thereby potentially increases sensitivity and response of the AML to chemotherapy treatment, and potentially reduces some chemotherapy side effects. The safety of Uproleselan has already been established in early phase clinical trials.

The purpose of this study is investigate whether the addition of Uproleselan to standard chemotherapy regimens improves outcomes for patients with AML that has not responded to previous treatment, or that has returned following previous successful treatment. Patients on the study will be randomised once enrolled on the trial to receive either standard chemotherapy treatment, or to receive chemotherapy plus the addition of Uproleselan.

Eligibility:

Include, but not limited to, the following:

Include, but not limited to, the following;
- A confirmed diagnosis of AML.
- Must not have a subtype of AML known as acute promyelocytic leukaemia (APML) or biphenotypic leukaemia.
- Must not have had a prior form of chronic leukaemia known as chronic myeloid leukaemia (CML).
- AML in the following settings
- 1) Patients with a relapse of AML having been previously successfully treated, including patients who have had one stem cell transplant (specified circumstances), OR
- 2) Patients with AML that has not responded to previous treatment (specified circumstances)
- No involvement with AML of the brain, spinal cord or cerebrospinal fluid.
- No serious health conditions that make intensive chemotherapy unsuitable.
- No symptoms of advanced heart failure
- Other diagnosis of cancer within the last two years, except for some local skin cancers requiring a minor surgical procedure for curative treatment, and selected curatively treated other cancers.

Contact person:

Louise Hay

Email louise.hay@health.wa.gov.au

Phone +61 (0)8 6383 3207

Principal Investigator:

Dr Gavin Cull

Sponsor:

GlycoMimetics Incorporated

Phase:

III

Protocol Number:

GMI-1271-301

Trial Registration Number:

NCT03616470

Clinical Trials.gov

ANZ Clinical Trial Registry