---
title: Blood Samples to Identify Biomarkers in Patients Treated With Cyclophosphamide After Donor Stem Cell Transplant
nct_id: NCT04160390
overall_status: ACTIVE_NOT_RECRUITING
sponsor: City of Hope Medical Center
study_type: OBSERVATIONAL
primary_condition: Allogeneic Hematopoietic Stem Cell Transplant Recipient
countries: United States
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT04160390.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT04160390"
ct_last_update_post_date: 2025-07-28
last_seen_at: "2026-05-12T06:53:48.385Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# Blood Samples to Identify Biomarkers in Patients Treated With Cyclophosphamide After Donor Stem Cell Transplant

**Official Title:** Blood Samples to Identify Biomarkers for Post-Transplant Cyclophosphamide

**NCT ID:** [NCT04160390](https://clinicaltrials.gov/study/NCT04160390)

## Key Facts

- **Status:** ACTIVE_NOT_RECRUITING
- **Study Type:** OBSERVATIONAL
- **Target Enrollment:** 72
- **Lead Sponsor:** City of Hope Medical Center
- **Collaborators:** National Cancer Institute (NCI)
- **Conditions:** Allogeneic Hematopoietic Stem Cell Transplant Recipient, Donor, Malignant Neoplasm
- **Start Date:** 2018-11-13
- **Completion Date:** 2027-04-24
- **CT.gov Last Update:** 2025-07-28

## Brief Summary

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.

## 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.

## Eligibility

- **Minimum age:** 18 Years
- **Sex:** ALL
- **Healthy Volunteers:** Yes

```
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
```

## Arms

- **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.

## Interventions

- **Biospecimen Collection-Blood** (OTHER) — Undergo collection of blood
- **Biospecimen Collection-Stool** (OTHER) — Undergo collection of stool

## Primary Outcomes

- **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

- **Acute GVHD** _(time frame: Up to 4 years)_

## Locations (1)

- City of Hope Medical Center, Duarte, California, United States

## Recent Field Changes (last 30 days)

- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `sponsor.collaborators` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.city of hope medical center|duarte|california|united states` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT04160390.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT04160390*  
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