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NCT Number: NCT02828592

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

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.

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Key information

Conditions

Severe Aplastic Anemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Age range

1 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Blood and Marrow Transplant Group of Georgia, Atlanta, Georgia, United States

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About this study

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%.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Availability of 3/6 - 5/6 matched (HLA-A, B, DR) related donor who must have negative HLA cross-match in the host vs. graft direction
  • Age <= 65 years for previously treated and <= 75 years for previously treated patients
  • 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 peripheral blood criteria above

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

Treatment and study plan

Fludarabine

Drug

30 mg/m2 IV QD x 5 days (Days -6 to -2)

Cyclophosphamide

Drug

14.5 mg/kg/day IV x 2 doses (Days -6 & -5)

Total Body Irradiation

Radiation

300 cGy x1 dose (Day -1)

Other names: TBI

Rabbit ATG

Drug

1.5 mg/kg/day x 3 days (Days -3 to -1)

Primary outcomes

  1. Demonstrate sustained engraftment after T-cell replete HLA-mismatched haploidentical bone marrow transplantation by collecting chimerism tests monthly following transplant

    Time frame: 2 years

    Hypothesis is that following preparative regimen and bone marrow transplantation, the 30-day graft failure rate will be <30%.

Secondary outcomes

  1. Determine the incidence of regimen-related mortality at 100 days post transplantation by recording treatment-related adverse events

    Time frame: 2 years

  2. Determine the incidence of grade 2-4 and 3-4 acute graft versus host disease at 100 days post transplantation by assessing signs and symptoms of GVHD throughout post-transplant course

    Time frame: 2 years

  3. Determine incidence of chronic GVHD at 6 months and 1 year post transplantation by assessing signs and symptoms of GVHD throughout post-transplant course

    Time frame: 2 years

  4. Estimate overall survival at 100 days and 1 year post transplantation by collecting survival information at those time points

    Time frame: 2 years

Study contacts

Contact information is provided by the study sponsor or research team.

Melhem Solh, MD

CONTACT

[email protected]

404-255-1930

Stacey Brown

CONTACT

[email protected]

404-851-8238

Sponsors and collaborators

Lead sponsor

Northside Hospital, Inc.

Other

Registry information

Official study title

A Study of T-Cell Replete, HLA-Mismatched Haploidentical Bone Marrow Transplantation With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Lacking HLA-Matched Related Donor

Important dates

Study start
2016
Primary completion
2027
Study completion
2028
First posted
Jul 11, 2016
Registry last updated
Apr 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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