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

Who is this study for? Child to adult patients with Aplastic Anemia
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug, Radiation
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 1
Maximum Age: 75
Healthy Volunteers: f
View:

• 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

Locations
United States
Georgia
Blood and Marrow Transplant Group of Georgia
RECRUITING
Atlanta
Northside Hospital
RECRUITING
Atlanta
Contact Information
Primary
Melhem Solh, MD
msolh@bmtga.com
404-255-1930
Backup
Stacey Brown
stacey.brown@northside.com
404-851-8238
Time Frame
Start Date: 2016-09-09
Estimated Completion Date: 2026-08-31
Participants
Target number of participants: 20
Treatments
Experimental: Flu/Cy/TBI
Fludarabine, Cyclophosphamide, TBI followed by bone marrow transplantation. Post-transplant Cyclophosphamide will be on Days 3 \& 4.
Sponsors
Leads: Northside Hospital, Inc.

This content was sourced from clinicaltrials.gov

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