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A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia

NCT05757310 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2026-01-05

No results posted yet for this study

Summary

This phase I trial evaluates the safety and feasibility of using a reduced-intensity regimen of cyclophosphamide, pentostatin, and anti-thymocyte globulin prior to a CD4+ T-cell depleted haploidentical hematopoietic cell transplant (haploHCT) for the treatment of patients with severe aplastic anemia that does not respond to treatment (refractory) or that has come back (recurrent). Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid. It may also lower the body's immune response. Pentostatin blocks a protein needed for cell growth. Anti-thymocyte globulin is an immunosuppressive drug can destroy immune cells known as T-cells. HaploHCT transfers blood-forming stem cells from a healthy partially-matched donor to a patient. Administering a regimen of cyclophosphamide, pentostatin, and anti-thymocyte globulin before haploHCT may help make room for the new, healthy cells and may reduce the risk of graft versus host disease.

Conditions

  • Recurrent Severe Aplastic Anemia
  • Refractory Severe Aplastic Anemia

Interventions

BIOLOGICAL

Anti-Thymocyte Globulin

Given IV

PROCEDURE

Biospecimen Collection

Undergo blood sample collection

PROCEDURE

Bone Marrow Aspirate

Undergo bone marrow aspirate

PROCEDURE

Bone Marrow Biopsy

Undergo bone marrow biopsy

DRUG

Cyclophosphamide

Given PO and IV

PROCEDURE

Haploidentical Hematopoietic Cell Transplantation

Undergo CD4+ T-cell depleted haploHCT

DRUG

Pentostatin

Given IV

OTHER

Questionnaire Administration

Ancillary studies

Sponsors & Collaborators

  • National Cancer Institute (NCI)

    collaborator NIH

  • City of Hope Medical Center

    lead OTHER

Principal Investigators

  • Ryotaro Nakamura · City of Hope Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
75 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2024-11-15
Primary Completion
2027-06-18
Completion
2027-06-18
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05757310 on ClinicalTrials.gov