New drug approach to prevent transplant rejection in leukemia patients

Plain-English translation of NCT04314219 on ClinicalTrials.gov ↗ · Source last updated March 2025 · Translation generated June 2026 · How we translate trials

Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.

What is this trial?

This trial compares two different medication approaches to prevent graft-versus-host disease (GVHD), a serious complication that can occur after a stem cell transplant. One group receives cyclophosphamide, a newer approach given shortly after transplant, while the other receives , the standard treatment. Both groups also receive tacrolimus, another medication to prevent rejection. The trial includes patients aged 14–65 with acute leukemia (AML or ALL) who are in remission and have a matched family member ready to donate stem cells.

Graft-versus-host disease is one of the biggest challenges after stem cell transplant and can cause serious, long-lasting complications. This trial is testing whether the newer medication approach can prevent this disease more effectively or with fewer side effects than the standard treatment.

Do you qualify?

You likely qualify if…

you have been diagnosed with acute leukemia (AML or ALL) and are currently in remission
you are between 14 and 65 years old
you have a fully matched family member (brother, sister, or parent) who is willing and able to donate stem cells
your heart, lungs, liver, and kidneys are functioning well enough to tolerate a transplant
you are healthy enough for the intensive chemotherapy that comes before the transplant (Karnofsky score of 70 or higher)

You likely don't qualify if…

you have an active, uncontrolled infection (bacterial, viral, or fungal)
you are pregnant, breastfeeding, or unwilling to use reliable birth control for one year after transplant
you have untreated or uncontrolled HIV, HTLV, hepatitis B, or hepatitis C
you have a history of cancer within the last 5 years (except non-melanoma skin cancer)
you have active or uncontrolled autoimmune disease

What participation looks like

If you qualify, you will be randomly assigned to receive either the new medication approach (cyclophosphamide with tacrolimus) or the standard treatment ( with tacrolimus). Both groups will first receive intensive chemotherapy to prepare your body, followed by the stem cell transplant from your matched donor. After transplant, you will receive your assigned medications and have frequent hospital visits and blood tests to monitor for complications and how well the transplant is working. This process typically involves a hospital stay of several weeks, followed by ongoing outpatient monitoring for many months.

AI-generated summary from trial data · Jun 18, 2026 · Not medical advice

Trial locations(1 site)

Saudi Arabia

King Faisal Specialist Hospital & Research Center, Riyadh