Sirolimus + Azacitidine for Myelodysplastic Syndrome

Recruiting in Palo Alto (17 mi)

+4 other locations

Overseen by Margaret Kasner, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Sidney Kimmel Cancer Center at Thomas Jefferson University

No Placebo Group

Prior Safety Data

Breakthrough Therapy

Trial Summary

What is the purpose of this trial?

This phase II trial studies how well sirolimus and azacitidine works in treating patients with high-risk myelodysplastic syndrome or recurrent acute myeloid leukemia. Sirolimus may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Sirolimus and azacitidine may kill more cancer cells.

Eligibility Criteria

Adults with high-risk myelodysplastic syndrome or recurrent acute myeloid leukemia not suitable for intensive chemo. Must be over 18, have a life expectancy of at least 4 weeks, able to take oral meds, and have acceptable organ function. Excludes those with severe diseases, active infections, HIV/AIDS, pregnant/breastfeeding women, or on certain drugs.

Inclusion Criteria

Patients must be ≥ 18 years old

Patients may have had a prior stem cell transplant (autologous or allogeneic), however they may not have active GvHD, nor be on any immunosuppression

Patients must have a diagnosis of one of the following:

Exclusion Criteria

Patients must not be receiving growth factors.

Patients with a current second malignancy requiring systemic therapy, other than non-melanoma skin cancers, are not eligible. If a patient has had a prior second malignancy that is not currently requiring active treatment, the patient will be considered eligible.

Patients with uncontrolled high blood pressure, unstable angina, symptomatic congestive heart failure, myocardial infarction within the past 6 months or serious uncontrolled cardiac arrhythmia are not eligible.

Participant Groups

The trial is testing the effectiveness of sirolimus (an inhibitor of cell growth) combined with azacitidine (a chemotherapy drug) in patients who either haven't responded well to other treatments or can't tolerate them.

Group I: MDS or AML with prior Azacitadine therapy Experimental Treatment 2 Interventions

Patients receive sirolimus PO on days 1-10 or 1-12 and azacitidine IV on days 4-8, 11, and 12 or days 4-10. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicity.

Group II: High risk Myleodysplastic Syndrome (MDS) Experimental Treatment 2 Interventions

Patients receive sirolimus PO on days 1-10 or 1-12 and azacitidine IV on days 4-8, 11, and 12 or days 4-10. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicity.

Group III: Acute Myeloid Leukemia (AML) Experimental Treatment 2 Interventions

Patients receive sirolimus PO on days 1-10 or 1-12 and azacitidine IV on days 4-8, 11, and 12 or days 4-10. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicity.

Azacitidine is already approved in European Union, United States, Canada, Japan, Australia for the following indications:

🇪🇺 Approved in European Union as Vidaza for:

• Acute myeloid leukemia
• Chronic myelomonocytic leukemia
• Myelodysplastic syndromes

🇺🇸 Approved in United States as Vidaza for:

• Myelodysplastic syndromes
• Chronic myelomonocytic leukemia

🇨🇦 Approved in Canada as Vidaza for:

• Myelodysplastic syndromes
• Acute myeloid leukemia

🇯🇵 Approved in Japan as Vidaza for:

• Myelodysplastic syndromes
• Acute myeloid leukemia

🇦🇺 Approved in Australia as Vidaza for:

• Myelodysplastic syndromes
• Acute myeloid leukemia

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Jefferson Health, Methodist Hospital Philadelphia, PA

Jefferson Health NJ Division (Kennedy Hospital) Sewell, NJ

Abington Hospital - Jefferson Health Abington, PA

Sidney Kimmel Cancer Center at Thomas Jefferson University Philadelphia, PA

More Trial Locations

Who Is Running the Clinical Trial?

Sidney Kimmel Cancer Center at Thomas Jefferson University Lead Sponsor

References