A study to evaluate study drug (Etavopivat) for the treatment of anemia in patients with Myelodysplastic Syndromes (MDS)

Purpose of trial

Etavopivat is an investigational drug that is being studied by Forma Therapeutics, Inc for use in the treatment of MDS-induced anemia and/or to reduce the number of red blood cell transfusions needed to treat the anemia. “Investigational” means that the drug has not been approved by Health Canada. Health Canada has not approved the sale or use of etavopivat to treat anemia caused by MDS, although they have allowed its use in this clinical trial.

Myelodysplastic Syndromes (MDS) are rare bone marrow diseases that can often result in low blood cell counts.  Red blood cells (RBC) are a type of blood cell that is made in the bone marrow. They carry hemoglobin, an important protein that carries oxygen throughout the body. Low levels of hemoglobin can result in anemia, and may require red blood cell transfusions to treat it.

The study will examine whether the experimental medicine, etavopivat, may improve the anemia caused by MDS and/or reduce the number of red blood cell transfusions that you might need. Such changes could result in improved energy levels and reduce the risk of iron overload.

The main purpose of this study is to determine the effects, both positive and negative, of etavopivat in individuals with MDS. The Sponsor will work with the study doctor to find out the following information:
•        To assess the hemoglobin and transfusion improvement of participants
•        To assess the safety of etavopivat based on side effects, laboratory tests, vital signs, and other clinical measures in the study
•        To assess the effect of etavopivat on measures demonstrating clinical benefit 
•        To measure the amount of etavopivat in the bloodstream and how etavopivat affects its molecular targets in the body
•        To assess how etavopivat impacts quality of life

Eligibility

Key Inclusion (From ICF):
•        Are at least 18 years old at the time of signing this ICF 
•        Are able to understand and voluntarily sign an informed consent form prior to any study-related assessments/procedures being conducted
•        Are willing and able to follow the study visit schedule and other protocol requirements
•        Are willing to avoid pregnancy or fathering children from the start of the study to 90 days after the last dose of study drug
•        Are diagnosed with MDS of very low, low or intermediate risk disease
•        Are diagnosed with anemia or are red blood cell transfusion dependent
•        Are non-responsive or are unable to tolerate luspatercept or luspatercept is not recommended by your doctor

Key Exclusion (From ICF):
•        Have therapy-associated MDS (eg. t-MDS) that is known to result from chemical injury or treatment with chemotherapy and/or radiation for other diseases
•        Have known history of acute myeloid leukemia (AML)
•        Have anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
•        Are pregnant or breastfeeding
•        Are diagnosed with severe renal dysfunction or are on chronic dialysis
•        Have clinically significant and active bacterial, fungal, parasitic, or viral infection
•        Have a known positive status for HIV
•        Have active hepatitis B or C infection
•        Have liver dysfunction
•        Prior treatment with azacitidine or decitabine
•        Prior stem cell transplant