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

Conditions

• Recurrent Severe Aplastic Anemia
• Refractory Severe Aplastic Anemia

Eligibility Criteria

Sex

ALL

Age

N/A - 75 Years

Healthy Volunteers

Accepted

Interventions

• Anti-Thymocyte Globulin — BIOLOGICAL
  Given IV
• Biospecimen Collection — PROCEDURE
  Undergo blood sample collection
• Bone Marrow Aspirate — PROCEDURE
  Undergo bone marrow aspirate
• Bone Marrow Biopsy — PROCEDURE
  Undergo bone marrow biopsy
• Cyclophosphamide — DRUG
  Given PO and IV
• Haploidentical Hematopoietic Cell Transplantation — PROCEDURE
  Undergo CD4+ T-cell depleted haploHCT
• Pentostatin — DRUG
  Given IV
• Questionnaire Administration — OTHER
  Ancillary studies

Study Details

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.

Key Dates

Start date

Nov 15, 2024

Status verified

Dec 2025

Primary completion

Jun 18, 2027

Completion

Jun 18, 2027

Study Design

Enrollment

6 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Treatment (conditioning, haploHCT)
  Patients receive cyclophosphamide PO and IV, pentostatin IV, anti-thymocyte globulin IV and undergo CD4+ T-cell depleted haploHCT on study. Patients also undergo bone marrow aspirate, bone marrow biopsy, and collection of blood samples at screening and follow-up.

Primary Outcome Measure

Incidence of adverse events [ Time Frame: From day -22 to day +100 or occurrence of unacceptable toxicity, whichever occurs first ]

Locations (1)

Find similar trials in Duarte, CA