Recruiting
Phase 2

Bone Marrow Transplant

Sponsor:

Northside Hospital, Inc.

Code:

NCT02828592

Conditions

Severe Aplastic Anemia

Eligibility Criteria

Sex: All

Age: 1 - 70+

Healthy Volunteers: Not accepted

Interventions

Fludarabine

Cyclophosphamide

Total Body Irradiation

Rabbit ATG

Cyclophosphamide

Study Details

Brief 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

Study ID

NCT02828592

Start date

Sep 9, 2016

Status verified date

Apr, 2026

Completion date

Aug 31, 2028

Anticipated

Primary completion date

Aug 31, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 1 - 70+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

20 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Flu/Cy/TBI

Fludarabine, Cyclophosphamide, TBI followed by bone marrow transplantation. Post-transplant Cyclophosphamide will be on Days 3 \& 4.

Interventions

Fludarabine

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

Cyclophosphamide

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

Total Body Irradiation

300 cGy x1 dose (Day -1)

Rabbit ATG

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

Cyclophosphamide

Post-transplant: 50 mg/kg IV QD (Day +3 to +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 and Locations

Central contacts

Melhem Solh, MD

404-255-1930msolh@bmtga.com

Stacey Brown

404-851-8238stacey.brown@northside.com

Locations

Blood and Marrow Transplant Group of Georgia

Recruiting

Atlanta, Georgia, United States, 30342

Contacts

Melhem Solh, MD

404-255-1930msolh@bmtga.com

Northside Hospital

Recruiting

Atlanta, Georgia, United States, 30342

Contacts

Melhem Solh, MD

404-255-1930msolh@bmtga.com

Stacey Brown

404-851-8238stacey.brown@northside.com

Principal Investigator:

Melhem Solh, MD

More Information

Sponsor

Northside Hospital, Inc.

Last update posted

Apr 20, 2026

Last verified

Apr, 2026

Keywords

  • SAA

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Northside Hospital, Inc. on 2026-04-20.