Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

Last updated: April 11, 2025
Sponsor: Northside Hospital, Inc.
Overall Status: Active - Recruiting

Phase

2

Condition

Aplastic Anemia

Anemia

Treatment

Fludarabine

Cyclophosphamide

Rabbit ATG

Clinical Study ID

NCT02828592
NSH 1158
  • Ages 1-75
  • All Genders

Study Summary

Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding. Patients with matched sibling donors for transplantation have a 80-90% chance of survival; however, a response rate with just immunosuppression for those patients lacking suitable HLA-matched related siblings is only 60%. With immunosuppression, only 1/3 of patients are cured, 1/3 are dependent on long term immunosuppression, and the other 1/3 relapse or develop a clonal disorder. Recent studies have shown that using a haploidentical donor for transplantation has good response rates and significantly lower rates of acute and chronic GVHD.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Availability of 3/6 - 5/6 matched (HLA-A, B, DR) related donor who must havenegative HLA cross-match in the host vs. graft direction

  • Age <= 65 years for previously treated and <= 75 years for previously treatedpatients

  • KPS >= 70%

  • Aplastic Anemia that meets the following criteria:

Peripheral Blood (must fulfill 2 of 3):

  • <500 PMN/mm3

  • <20,000 platelets

  • absolute reticulocyte count <40,000/microL

Bone Marrow (must be either):

  • markedly hypocellular (<25% of normal cellularity)

  • moderately hypocellular with 70% non-myeloid precursors and patient meets peripheralblood criteria above

Exclusion

Exclusion Criteria:

  • poor cardiac function (LVEF <40%)

  • poor pulmonary function (FEV1 & FVC <50% predicted)

  • poor liver function (bili >= 2mg/dL)

  • poor renal function (creatinine >= 2.0mg/dL or creatinine clearance <40mL/min)

  • prior allogeneic transplant

Study Design

Total Participants: 20
Treatment Group(s): 4
Primary Treatment: Fludarabine
Phase: 2
Study Start date:
September 09, 2016
Estimated Completion Date:
August 31, 2026

Study Description

Mismatched haploidentical donors will be identified for patients with severe aplastic anemia. These patients will undergo a preparative regimen of Fludarabine/Cyclophosphamide/TBI followed by haploidentical bone marrow transplantation. Post-transplant Cyclophosphamide will be administered on Days 3 & 4. Immunosuppression with Tacrolimus and MMF will begin on Day +5; MMF will be discontinued on Day +35 while Tacrolimus continues until Day +180. Investigators hypothesize that haploidentical transplantation with the above-mentioned preparative regimen will have a <30% graft failure rate. The one-sided exact Binomial test at 5% significance level will be used to test this hypothesis. The size of 20 patients provides the power of 92.5% for confirming the 30-day graft failure rate <30%.

Connect with a study center

  • Blood and Marrow Transplant Group of Georgia

    Atlanta, Georgia 30342
    United States

    Active - Recruiting

  • Northside Hospital

    Atlanta, Georgia 30342
    United States

    Active - Recruiting

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