Recruiting
Phase 2

Cyclophosphamide vs. Cyclophosphamide

Sponsor:

University of Nebraska

Code:

NCT06799195

Conditions

Hematological Malignancies

Graft-versus-Host Disease (GVHD)

Eligibility Criteria

Sex: All

Age: 60+

Healthy Volunteers: Not accepted

Interventions

Attenuated-dose Cyclophosphamide

High-dose Cyclophosphamide

Sirolimus

Mycophenolate Mofetil (MMF)

Study Details

Brief summary:

This study will compare post-transplant health-related quality of life following the use of standard versus attenuated dose of post-transplant cyclophosphamide in addition to two-drug graft-versus-host disease (GVHD) prophylaxis among recipients of allogeneic hematopoietic stem cell transplant.

Conditions

Hematological Malignancies

Graft-versus-Host Disease (GVHD)

Study ID

NCT06799195

Start date

Jun 23, 2025

Status verified date

Aug, 2025

Completion date

Nov, 2031

Anticipated

Primary completion date

Apr, 2031

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 60+

Healthy Volunteers: Not accepted

Inclusion criteria:

  • Adults aged 60 years or older
  • Diagnosis of a hematological malignancy or other serious hematological disorder that requires an allogeneic hematopoietic cell transplantation
  • Planned to receive any reduced-intensity conditioning regimen (any graft source is acceptable) and availability of human leukocyte antigen (HLA)-matched donor at HLA loci A, B, C, and HLA-DR beta chain antigen (DRB1)
  • Karnofsky Performance Status (KPS) of 70% or higher.

Exclusion criteria:

  • Previous history of one or more prior allogeneic stem cell transplants (i.e., second or third allogeneic transplant)
  • Planned use of high doses of cyclophosphamide (e.g., a total cyclophosphamide dose of approximately 50 mg/kg or more) as part of the conditioning regimen prior to allogeneic stem cell transplant. A lower dose of cyclophosphamide (e.g., fludarabine, cyclophosphamide, and low-dose total body irradiation regimen that uses 2 doses of cyclophosphamide at 14.5 mg/kg) is acceptable.
  • Known diagnosis of liver cirrhosis or other advanced liver disease that may impact cyclophosphamide metabolism.
  • Diagnosis of myelofibrosis
  • Creatinine clearance less than 40 mL/min/1.73 m², which may increase the risk of hemorrhagic cystitis with post-transplant cyclophosphamide (PTCy)
  • Systolic cardiac dysfunction with an ejection fraction of less than 45%.
  • Use of a haploidentical or mismatched donor.
  • Any other condition judged by the physician to increase the risk of toxicities associated with PTCy.

Study Design

Enrollment

126 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Attenuated-dose post-transplant cyclophosphamide (PTCy) Arm

Participants will receive attenuated-dose post-transplant cyclophosphamide (PTCy) at 25 mg/kg on days +3 and +4 after allogeneic hematopoietic stem cell transplantation. This is in addition to sirolimus and mycophenolate mofetil (MMF) for graft-versus-host disease (GVHD) prophylaxis.

active comparator: High-dose post-transplant cyclophosphamide (PTCy) Arm: Standard of Care

Participants will receive high-dose post-transplant cyclophosphamide (PTCy) at 50 mg/kg on days +3 and +4 after allogeneic hematopoietic stem cell transplantation. This is in addition to sirolimus and mycophenolate mofetil (MMF) for GVHD prophylaxis.

Interventions

Attenuated-dose Cyclophosphamide

Cyclophosphamide administered at an attenuated dose of 25 mg/kg on days +3 and +4 post-transplant for GVHD prophylaxis.

High-dose Cyclophosphamide

Cyclophosphamide administered at the standard high dose of 50 mg/kg on days +3 and +4 post-transplant for GVHD prophylaxis.

Sirolimus

Sirolimus is started on day +5 with a loading dose of 6 mg, followed by a maintenance dose of 2 mg daily, adjusted to target trough levels of 8-12 ng/mL. Sirolimus taper is recommended to start at day +90 and to be completed by day +180, provided there is no evidence of acute GVHD.

Mycophenolate Mofetil (MMF)

MMF is started on day +5 at a dose of 15 mg/kg per dose (maximum 1 g per dose) three times daily. MMF is generally discontinued by day +35 in the absence of GVHD.

Primary outcome measure

  • Change in Health-Related Quality of Life as Measured by Functional Assessment of Cancer Therapy-Bone Marrow Transplantation [ Time Frame: Baseline and 3 months post-transplant ]

Central Contacts and Locations

Central contacts

Taylor Johnson

402-559-4596taylora.johnson@unmc.edu

IIT OFFICE

402-559-4596iitoffice@unmc.edu

Locations

University of Nebraska Medical Center

Recruiting

Omaha, Nebraska, United States, 68198

Contacts

Taylor Johnson

402-559-4596taylora.johnson@unmc.edu

Principal Investigator:

Moataz Ellithi, MBChB

More Information

Sponsor

University of Nebraska

Last update posted

Dec 15, 2025

Last verified

Aug, 2025

Keywords

  • Allogeneic Hematopoietic Stem Cell Transplantation
  • GVHD Prophylaxis
  • Sirolimus
  • Mycophenolate Mofetil (MMF)
  • Health-Related Quality of Life (HRQoL)
  • Randomized Controlled Trial

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of Nebraska on 2025-12-15.