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Active, Not Recruiting

NCT Number: NCT04160390

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Allogeneic Hematopoietic Stem Cell Transplant Recipient Donor Malignant Neoplasm Neoplasms

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

City of Hope Medical Center

Duarte, California, 91010, United States

About this study

"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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Biospecimen Collection-Blood

Other

Undergo collection of blood

Biospecimen Collection-Stool

Other

Undergo collection of stool

Primary outcomes

  1. Mathematical models of cyclophosphamide (CY) and mycophenolic acid pharmacokinetics (PK) with t cell effects.

    Time frame: Up to day 21

    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.

Secondary outcomes

  1. Acute GVHD

    Time frame: Up to 4 years

    Cumulative incidence estimates will be calculated with appropriate accounting for competing risks. Adjusted hazard ratios will be estimated via Cox regression models.

Sponsors and collaborators

Lead sponsor

City of Hope Medical Center

Other

Collaborators

  • National Cancer Institute (NCI)

Registry information

Official study title

Blood Samples to Identify Biomarkers for Post-Transplant Cyclophosphamide

Important dates

Study start
2018
Primary completion
2027
Study completion
2027
First posted
Nov 13, 2019
Registry last updated
Jul 28, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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