RAD001 + PKC412 for Acute Myeloid Leukemia

Recruiting in Palo Alto (17 mi)

+2 other locations

Overseen byRichard Stone, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Richard Stone, MD

No Placebo Group

Trial Summary

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.

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

I can take care of myself but cannot do heavy physical work.

INR < 1.3 (or < 3 on anticoagulants)

I don't expect to need treatment to reduce my blood cell count within a month, except for hydroxyurea.

+6 more

Exclusion Criteria

I have been diagnosed with acute promyelocytic leukemia.

I had a bone marrow or stem cell transplant less than 2 months ago.

I do not have severe health or mental conditions that could stop me from completing the study.

+14 more

Participant Groups

The study tests RAD001 combined with PKC412 to find the safest high dose against certain blood cancers. RAD001 targets a protein pathway that's overactive in cancer cells while PKC412 blocks an enzyme linked to AML cell growth.

1Treatment groups

Experimental Treatment

Group I: Only one arm on this study.Experimental Treatment2 Interventions