RAD001 + PKC412 for Acute Myeloid Leukemia
Trial Summary
Do I have to stop taking my current medications for the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, you cannot participate if you are on chronic treatment with systemic steroids or another immunosuppressive agent.
What data supports the effectiveness of the drug PKC412 for treating acute myeloid leukemia?
PKC412, also known as midostaurin, has shown effectiveness in treating acute myeloid leukemia (AML) with FLT3 mutations, as it can inhibit the growth of leukemic cells and improve overall survival when combined with standard chemotherapy. It has been approved for use in AML patients with these specific mutations.12345
Is the combination of RAD001 and PKC412 safe for humans?
PKC412, also known as Midostaurin, has been studied for its effects on acute myeloid leukemia (AML) cells, particularly those with specific mutations. It can cause cell death in certain leukemia cells, but its safety profile in humans, especially in combination with other drugs like RAD001, is not fully detailed in the available studies. Further clinical trials are needed to better understand its safety in humans.13467
How does the drug combination of RAD001 and PKC412 differ from other treatments for acute myeloid leukemia?
The combination of RAD001 and PKC412 is unique because it targets the FLT3 mutation, which is common in acute myeloid leukemia (AML) and associated with poor prognosis. PKC412 (Midostaurin) is a kinase inhibitor that has shown effectiveness in treating FLT3-mutated AML, and combining it with RAD001 may enhance its therapeutic effects, potentially overcoming resistance seen with PKC412 alone.12357
What is the purpose of this trial?
The purpose of this research study is to determine the safety of the combination of RAD001 and PKC412 as a cancer treatment, and to establish the highest dose of RAD001 that can be given in conjunction with PKC412. These drugs have been used in other research trials for individuals with solid and hematology malignancies. Past research on PKC412 shows that it blocks the abnormal functioning of an enzyme called FLT3. FLT3 is found in your cells in either a normal (wild type) or genetically changed form and plays a role in the survival and growth of AML cells. RAD001 is an inhibitor of a central growth pathway that involves the protein MTOR. The MTOR pathway is overactive in cancer cells, causing the cells to grow abnormally. By inhibiting the abnormal growth activity of the MTOR pathway, RAD001 slows down and possibly stops the growth of cancer cells.
Research Team
Richard Stone, MD
Principal Investigator
Dana-Farber Cancer Institute
Eligibility Criteria
This trial is for adults with relapsed or refractory AML, MDS, or CMML who can't have standard therapy. They should be in relatively stable health (ECOG ≤2), not pregnant, using double barrier contraception if of childbearing potential, and without recent transplants or other cancers within 5 years.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive RAD001 and PKC412 in a dose-escalation study to determine the maximum tolerated dose
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- PKC412
- RAD001
Find a Clinic Near You
Who Is Running the Clinical Trial?
Richard Stone, MD
Lead Sponsor
Novartis
Industry Sponsor
Vasant Narasimhan
Novartis
Chief Executive Officer since 2018
MD from Harvard Medical School, Bachelor's in Biological Sciences from University of Chicago, Master's in Public Policy from John F. Kennedy School of Government
Shreeram Aradhye
Novartis
Chief Medical Officer since 2022
MD from Yale University, MSc in Clinical Epidemiology from University of Pennsylvania
Beth Israel Deaconess Medical Center
Collaborator
Massachusetts General Hospital
Collaborator
Brigham and Women's Hospital
Collaborator