Blood Samples to Identify Biomarkers in Patients Treated With Cyclophosphamide After Donor Stem Cell Transplant

Blood Samples to Identify Biomarkers for Post-Transplant Cyclophosphamide

This trial uses blood samples to understand how patients' bodies process and respond to a drug called cyclophosphamide given after a donor stem cell transplant. Identifying biomarkers (molecules that can indicate normal or abnormal processes) may help researchers develop a blood test that can be used to predict how well patients will process and respond to cyclophosphamide.

Study Overview

• Status: Active, not recruiting
• Conditions: Malignant Neoplasm; Allogeneic Hematopoietic Stem Cell Transplant Recipient; Donor
• Intervention / Treatment: Other: Biospecimen Collection-Blood; Other: Biospecimen Collection-Stool

Detailed Description

"Ancillary-Correlative" study type

PRIMARY OBJECTIVES:

I. To determine whether endogenous metabolomics compounds obtained before cyclophosphamide administration can predict the ratio of 4hydroxycyclophosphamide to cyclophosphamide area under the curve (4HCY/CY AUC).

II. To evaluate mathematic models of CY and mycophenolic acid (MPA) pharmacokinetics and develop mathematic models including these pharmacokinetics, -omics data and clinical outcomes.

SECONDARY OBJECTIVES:

I. To assess if recipients' metabolomics and pharmacokinetics (e.g., CY and its metabolites AUCs) are associated with acute graft versus host disease (GVHD) and other clinical outcomes.

II. To assess the association of the intestinal microbiome with the plasma metabolome, acute GVHD and other clinical outcomes.

III. To obtain donor blood samples at one time pre-transplant and assess if donors' metabolomics are associated with acute GVHD and other clinical outcomes.

EXPLORATORY OBJECTIVE:

I. Donor and recipient germline deoxyribonucleic acid (DNA) isolation and genomic analysis.

OUTLINE: Participants are assigned to 1 of 2 arms.

ARM I (PATIENTS RECEIVING A HAPLOIDENTICAL TRANSPLANT): Patients undergo collection of blood prior to transplant, on day 0, days 3-7, day 14, and day 21. Patients also undergo collection of saliva prior to transplant and collection of stool prior to and post-transplant. Donors undergo collection of blood and saliva within 8 weeks prior to donation.

• Study Type: Observational
• Enrollment (Actual): 72

Contacts and Locations

Study Locations

• United States
  • California
    • Duarte, California, United States, 91010
      • City of Hope Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

Patients of the City of Hope Comprehensive Cancer Center and donors.

Description

Inclusion Criteria:

• Scheduled to undergo allogeneic hematopoietic cell transplant (HCT) with a haploidentical donor.
• Allogeneic HCT scheduled to treat any underlying disease. Patients with nonmalignant diseases or cancer are eligible
• Scheduled to receive post-transplant cyclophosphamide (any dose, any number of doses, any dosing frequency) as part of their post-graft immunosuppression or GVHD prophylaxis. Patients enrolled on treatment protocols that include post transplant cyclophosphamide (PTCy) but do not include mycophenolate mofetil (MMF) or tacrolimus can participate

• Willingness to:

  • Provide blood
  • Permit medical record review

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Arm I (biospecimen collection); Patients undergo collection of blood prior to transplant, on day 0, days 3-7, day 14, and day 21. Patients also undergo collection of saliva prior to transplant and collection of stool prior to and post-transplant. Donors undergo collection of blood and saliva within 8 weeks prior to donation.	Other: Biospecimen Collection-Blood; Undergo collection of blood; Other: Biospecimen Collection-Stool; Undergo collection of stool

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mathematical models of cyclophosphamide (CY) and mycophenolic acid pharmacokinetics (PK) with t cell effects.	Will determine whether readily available patient characteristics influence CY PK using population (pop)PK modeling. Will validate our existing popPK model of CY, 4HCY, and carboxyethylphosphoramide mustard.	Up to day 21

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Acute GVHD	Cumulative incidence estimates will be calculated with appropriate accounting for competing risks. Adjusted hazard ratios will be estimated via Cox regression models.	Up to 4 years

Collaborators and Investigators

• Sponsor: City of Hope Medical Center
• Collaborators: National Cancer Institute (NCI)
• Investigators: Principal Investigator: Jeannine S McCune, City of Hope Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): November 13, 2018
• Primary Completion (Estimated): April 24, 2027
• Study Completion (Estimated): April 24, 2027

Study Registration Dates

• First Submitted: November 8, 2019
• First Submitted That Met QC Criteria: November 8, 2019
• First Posted (Actual): November 13, 2019

Study Record Updates

• Last Update Posted (Actual): July 28, 2026
• Last Update Submitted That Met QC Criteria: July 27, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms
• Other Study ID Numbers: 18358 (Other Identifier: City of Hope Medical Center); NCI-2019-07376 (Registry Identifier: CTRP (Clinical Trial Reporting Program)); U01CA239373 (U.S. NIH Grant/Contract)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No