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

Part of paid clinical trials in Atlanta, Georgia.

Sponsor
Northside Hospital, Inc.
Study ID
NCT02828592
Phase
PHASE2
Status
Recruiting
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Conditions

  • Severe Aplastic Anemia

Eligibility Criteria

Sex
ALL
Age
1 Year - 75 Years
Healthy Volunteers
Not accepted

Interventions

  • 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)
  • Rabbit ATG — DRUG
    1.5 mg/kg/day x 3 days (Days -3 to -1)
  • Cyclophosphamide — DRUG
    Post-transplant: 50 mg/kg IV QD (Day +3 to +4)

Study Details

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.

Key Dates

Start date
Sep 9, 2016
Status verified
Apr 2026
Primary completion
Aug 31, 2027
Completion
Aug 31, 2028

Study Design

Enrollment
20 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Flu/Cy/TBI
    Fludarabine, Cyclophosphamide, TBI followed by bone marrow transplantation. Post-transplant Cyclophosphamide will be on Days 3 \& 4.

Primary Outcome Measure

Demonstrate sustained engraftment after T-cell replete HLA-mismatched haploidentical bone marrow transplantation by collecting chimerism tests monthly following transplant [ Time Frame: 2 years ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
Blood and Marrow Transplant Group of GeorgiaAtlantaGeorgia30342
Melhem Solh, MD
[email protected]
404-255-1930
Northside HospitalAtlantaGeorgia30342
Melhem Solh, MD
[email protected]
404-255-1930
Stacey Brown
[email protected]
404-851-8238
Scott Solomon, MD (SUB_INVESTIGATOR)
H. Kent Holland, MD (SUB_INVESTIGATOR)
Asad Bashey, MD, PhD (SUB_INVESTIGATOR)
Lawrence Morris, MD (SUB_INVESTIGATOR)
Melhem Solh, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Atlanta, GA

By research site
Blood and Marrow Transplant Group of Georgia· Atlanta, GANorthside Hospital· Atlanta, GA

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