The PIMMs study is an early clinical trial that is investigating the effects of a specific drug, ivosidenib, in patients who have had unexplained low blood counts for at least six months.
Unexplained low blood counts can be caused by conditions such as vitamin deficiencies or age-associated anemia or be a side effect of the use of some medications. It’s often diagnosed after a routine blood test. However, when doctors can’t determine the cause of low blood counts, genetic testing is usually the next step. Such testing may reveal a genetic mutation in the blood cells.
Low blood cell counts coupled with a genetic mutation in the blood is called Clonal Cytopenia of Undetermined Significance (CCUS). Individuals with CCUS have a high risk of developing a leukemia or myelodysplastic syndrome (MDS) within the next 4-5 years. The PIMMs Clinical Trial focuses specifically on CCUS patients with a genetic mutation in the gene IDH1, specifically the R132 mutation.
What is an IDH1 Gene Mutation?
A specific type of gene called IDH1 has been linked to the development of cancer. These genes make an enzyme called isocitrate dehydrogenase 1, which helps the body break down fats and convert it to energy. Mutations in the IDH1 gene can lead to uncontrolled cell growth, putting a person at risk for cancer. IDH1 gene mutations have been found in several cancers, including acute myeloid leukemia, chondrosarcoma (a type of bone cancer), gliomas (brain and spinal tumors), and lymphomas.
What is Ivosidenib?
In recent years, a class of drugs known as IDH1 inhibitors has been shown to stop or slow the growth of cancer cells in patients that carry the IDH1 gene mutation and who have already developed cancer. Ivosidenib (brand name Tibsovo®) is in this class of drugs.
In 2018, the U.S. Food and Drug Administration approved ivosidenib for adult patients with relapsed or refractory AML who also have an IDH1 mutation. A year later, the FDA broadened its use to include newly diagnosed IDH1-mutant AML patients age 75+ who are unable to undergo other cancer treatments such as chemotherapy. The drug then was approved to treat relapsed locally advanced or metastatic cholangiocarcinoma.
In 2022, the FDA approved a combination therapy of ivosidenib and another drug, azacitidine, to treat newly diagnosed IDH1-mutant AML patients.
What is the PIMMs trial investigating?
It is known that patients with CCUS have up to an 80-percent chance that the condition will progress to MDS/AML. The PIMMs trial is investigating whether ivosidenib can be given to patients diagnosed with CCUS to improve cytopenias. If results are positive, long-term research may investigate whether ivosidenib can be used as a preventive medication to prevent or lower the risk of cancer development.
Who Can Enroll in the PIMMs trial?
Patients who are diagnosed with clonal cytopenia of undetermined significance (CCUS) who also have the IDH1 R132 gene mutation.
How Many People Will Participate in the PIMMs trial?
In this first investigational trial, an estimated 15 people will be enrolled.
How Can I Participate?
We have onsite and remote options to participate in this study.
- You can work directly with oncologists at a participating site for the PIMMs clinical trial; OR
- We will coordinate with your local oncologist and local labs so that you can participate no matter where you live.
Enrollment in the PIMMs clinical trial is voluntary and there is no financial compensation for participating.
How to Enroll