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

NCT02828592 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2026-04-20

No results posted yet for this 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.

Conditions

  • Severe Aplastic Anemia

Interventions

DRUG

Fludarabine

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

DRUG

Cyclophosphamide

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

RADIATION

Total Body Irradiation

300 cGy x1 dose (Day -1)

DRUG

Rabbit ATG

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

DRUG

Cyclophosphamide

Post-transplant: 50 mg/kg IV QD (Day +3 to +4)

Sponsors & Collaborators

  • Northside Hospital, Inc.

    lead OTHER

Principal Investigators

  • Melhem Solh, MD · Blood and Marrow Transplant Group of Georgia

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-09-09
Primary Completion
2027-08-31
Completion
2028-08-31

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 NCT02828592 on ClinicalTrials.gov