FORMA

FORMA

A Phase 1/2 study evaluating the safety, efficacy, pharmacokinetics and pharmacodynamics of FT-2102 with or without azacitidine or cytarabine in patients with AML or MDS with an IDH-1 mutation.

Disease:

Acute Myeloid Leukaemia (AML) and high risk Myelodysplasia (MDS).

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.

Myelodysplasia (MDS) is a type of cancer of bone marrow cells. In MDS, the stem cells are damaged, which results in the production of abnormal, clonal blood cells. This may lead to inadequate production of any blood cell type, and may result in anaemia, bleeding, bruising or increased infections.

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.

MDS can evolve into AML. The prognosis of MDS, including the risk of progression to AML, is calculated using a risk score which takes into account blood counts, number of blast cells seen on bone marrow testing, and genetic changes within the abnormal bone marrow cells. There are also a range of molecular markers that may be mutated in abnormal cells in MDS or AML and may affect prognosis. One such marker is IDH-1. This is expressed in around 10% of AML and 3% of MDS.

New drugs that target a range of molecular mutations are being developed for use in MDS and AML – including drugs that target mutated IDH-1 (IDH-1 inhibitors).

The aim of this study is to understand the safety and activity of FT2102 (an IDH-1 inhibitor) as a single agent, and in combination with accepted standard treatments for AML and MDS – Azacytadine or Cytarabine, in people with MDS or AML and who have been show to have the IDH-1 mutation.

Eligibility:

Include, but not limited to, the following:

Inclusion Criteria:
1. Relapsed or refractory pathologically proven acute myeloid leukemia (AML) or intermediate-very high risk Myelodysplastic Syndrome (MDS) as defined by WHO criteria or Revised International Prognostic Scoring System (IPSS-R)
2. Patients must have documented IDH1-R132 gene-mutated disease as evaluated by the site
3. Good performance status
4. Good kidney and liver function

Exclusion Criteria:
1. Patients with symptomatic central nervous system (CNS) metastases or other tumor location (such as spinal cord compression, other compressive mass, uncontrolled painful lesion, bone fracture, etc.) necessitating an urgent therapeutic intervention, palliative care, surgery or radiation therapy
2. Congestive heart failure (New York Heart Association Class III or IV) or unstable angina pectoris. Previous history of myocardial infarction within 1 year prior to study entry, uncontrolled hypertension or uncontrolled arrhythmias
3. Pulmonary disease (e.g. COPD, asthma, etc) that is not controlled (moderate to severe symptoms) with current medication
4. Active, uncontrolled bacterial, viral, or fungal infections, requiring systemic therapy

Contact person:

Louise Hay
Email louise.hay@health.wa.gov.au
Phone 08 6383 3207


Email
Phone

Principal Investigator:

Dr Carolyn Grove

6457 7600

Sponsor:

Forma Therapeutics Inc.

Phase:

1/2

Protocol Number:

2102-HEM-101

Trial Registration Number:

NCT02719574

Clinical Trials.gov

ANZ Clinical Trial Registry