Actively Recruiting

Phase 1
Phase 2
Age: 12Years - 120Years
All Genders
Healthy Volunteers
NCT05436418

The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation

Led by National Cancer Institute (NCI) · Updated on 2026-04-27

260

Participants Needed

2

Research Sites

292 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Background: Blood cancers (such as leukemias or lymphomas) often do not respond to standard treatments. A transplant of blood stem cells from a healthy donor can help people with these cancers. Sometimes these transplants cause serious side effects, including a common immunologic problem called graft-versus-host disease. A drug called cyclophosphamide given early after the transplant (post-transplantation cyclophosphamide, PTCy) can reduce these complications. But sometimes this drug has its own negative effects. Furthermore, studies in mice suggest that an intermediate, rather than very high, dose of this drug may best protect against graft-versus-host disease. Objective: To find out if a lower dose of PTCy is more helpful for people who undergo blood stem cell transplants. Eligibility: People aged 18 and older who have a blood cancer and are eligible for a transplant of blood stem cells from another person. Healthy donors are also needed but must be related to the individual needing the transplant. Design: Participants will undergo screening. Transplant recipients will have imaging scans and tests of their heart and lung function. They will be assessed for the status of their cancer, including bone marrow taken from their pelvis and possibly also scans and/or fluid drawn from the spine depending on the disease type. Donors will be screened for general health. They will give several tubes of blood. They will give an oral swab and saliva and stool samples for research. Recipients will be in the hospital at least 4 to 6 weeks. They will have a temporary catheter inserted into a vein in the chest or neck. Medications will be given and blood will be drawn through the catheter. The transplanted stem cells will be given through the catheter. Participants will receive medications both before and after the transplant. Participants will return to the clinic at least once a week for 3 months after leaving the hospital. Follow-up visits will continue periodically for 5 years.

CONDITIONS

Official Title

The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation

Who Can Participate

Age: 12Years - 120Years
All Genders
Healthy Volunteers

Eligibility Criteria

Eligible

You may qualify if you...

  • Have a confirmed blood cancer suitable for allogeneic stem cell transplant including specific leukemias, lymphomas, myelodysplastic syndromes, myelofibrosis, chronic leukemias, multiple myeloma, or related conditions
  • Age 50 years or older, or age 18-49 with additional health risk factors making myeloablative conditioning unsuitable
  • Have at least one suitable related or unrelated donor with proper HLA matching
  • Karnofsky performance score of 70 or above
  • Adequate organ function including cardiac ejection fraction \u2265 45%, lung function tests \u2265 50% predicted, kidney function clearance \u2265 60 ml/min, bilirubin \u2264 2 times upper limit, and liver enzymes \u2264 3 times upper limit
  • Agree to use effective contraception before and for one year after transplant if able to father or bear children
  • Willing and able to provide informed consent
Not Eligible

You will not qualify if you...

  • Receiving other investigational treatments within 2 weeks prior to conditioning
  • Currently nursing
  • Active non-blood cancers that are metastatic, relapsed, refractory, or recently treated with curative intent (excluding non-melanoma skin cancers)
  • Allergic reactions to similar drugs used in the study
  • Uncontrolled serious illnesses that would make transplant unsafe
  • For donors, no specific exclusion criteria listed

AI-Screening

AI-Powered Screening

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Trial Site Locations

Total: 2 locations

1

City of Hope

Duarte, California, United States, 91010

Not Yet Recruiting

2

National Institutes of Health Clinical Center

Bethesda, Maryland, United States, 20892

Actively Recruiting

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Research Team

A

Amy H Chai

CONTACT

C

Christopher G Kanakry, M.D.

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

8

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