Phase I/II Trial to Determine 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

Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug, Procedure, Radiation
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Healthy Volunteers: t
View:

⁃ Donor

• Related (age \>=12) and unrelated (age \>=18) donors deemed eligible (i.e., evaluated at NIH, COH, and FHCC in accordance with existing institutional Standard Policies and Procedures or evaluated per the standards required by the IRB of the National Marrow Donor Program or applicable registry), and willing to donate research samples will be included.

• Ability of participant or parent/legal guardian to understand and the willingness to sign a written informed consent document.

Locations
United States
California
City of Hope
NOT_YET_RECRUITING
Duarte
Maryland
National Institutes of Health Clinical Center
RECRUITING
Bethesda
Contact Information
Primary
Amy H Chai
amy.chai@nih.gov
(240) 858-3755
Backup
Christopher G Kanakry, M.D.
christopher.kanakry@nih.gov
(240) 760-6171
Time Frame
Start Date: 2022-11-18
Estimated Completion Date: 2027-07-02
Participants
Target number of participants: 260
Treatments
No_intervention: Donors (Haplo HCT)
Research on collected samples
No_intervention: Donors (Matched HCT)
Research on collected samples
Experimental: Phase I Dose De-escalation (Haplo HCT)
PTCy at de-escalating doses to assess for safety and determine Phase II dose
Experimental: Phase I Dose De-escalation (Matched HCT)
PTCy at de-escalating doses to assess for safety and determine Phase II dose
Experimental: Phase I Pilot for Comparative Data (Haplo HCT)
Standard PTCy 50 mg/kgday on days +3 and +4
Experimental: Phase I Pilot for Comparative Data (Matched HCT)
Standard PTCy 50 mg/kg/day on days +3 and +4
Experimental: Phase II Efficacy (Haplo HCT)
PTCy at shortest duration, safe dose (from Phase I)
Experimental: Phase II Efficacy (Matched HCT)
PTCy at shortest duration, safe dose (from Phase I)
Sponsors
Leads: National Cancer Institute (NCI)

This content was sourced from clinicaltrials.gov

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