Actively Recruiting
A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Haploidentical Related, Partially HLA-Mismatched, or Matched Unrelated Bone Marrow for Newly Diagnosed Patients With Severe Aplastic Anemia
Led by Medical College of Wisconsin · Updated on 2026-02-17
60
Participants Needed
25
Research Sites
26 weeks
Total Duration
On this page
Sponsors
M
Medical College of Wisconsin
Lead Sponsor
N
National Heart, Lung, and Blood Institute (NHLBI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating bone marrow transplantation as a first treatment for patients aged 3 to 75 years with severe aplastic anemia (SAA) who have not received prior therapy. SAA is a rare condition where the body stops producing enough blood cells, leading to low white blood cells, red blood cells, and platelets. This study aims to determine if transplantation from half-matched related donors or matched unrelated donors can restore normal blood cell production safely and effectively in untreated patients. Participants will receive a reduced-intensity conditioning regimen including fludarabine, cyclophosphamide, low-dose total body irradiation, and Thymoglobulin before receiving fresh bone marrow cells from either a haploidentical related donor or an unrelated donor. Graft-versus-host disease (GVHD) prevention will include post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil. The study includes two parallel groups based on donor type, each aiming to enroll 30 participants. Throughout the study, participants will be closely monitored for one year after transplant to assess survival without GVHD or treatment failure, recovery of blood cells, graft success, and incidence of acute and chronic GVHD. Assessments will include blood counts at multiple time points, evaluation of graft failure, and overall and failure-free survival. Participants will be followed regularly to track these outcomes and their health status during the year after transplantation.
CONDITIONS
Brief Title
Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 3 years to 75 years
- Confirmed diagnosis of acquired severe aplastic anemia with bone marrow cellularity less than 25% or hypoplastic marrow for age plus at least two of the following: neutrophils below 0.5 x109/L, platelets below 20 x109/L, or low reticulocyte count
- No suitable fully matched related donor available
- Available donor as defined by the study protocol
- Signed informed consent by participant or legal guardian
- Adequate organ function including cardiac, hepatic, renal, and pulmonary function per institutional transplant standards
- Karnofsky or Lansky performance status 60% or higher
- Females and males of childbearing potential agree to use two effective contraception methods or abstinence
You will not qualify if you...
- Inherited bone marrow failure syndromes such as Fanconi anemia or short telomere syndromes
- Clonal cytogenetic abnormalities consistent with pre-MDS or MDS
- Formal diagnosis of myelodysplastic syndrome
- Positive donor-specific HLA antibodies with high median fluorescence intensity
- Prior desensitization attempts for donor HLA antibodies
- Previous treatment for severe aplastic anemia including immunosuppressive therapy
- Prior allogeneic stem cell or solid organ transplant
- Life-threatening reaction to Thymoglobulin®
- Uncontrolled infections at enrollment
- Pregnancy or breastfeeding
- Prior malignancies except certain treated cancers more than five years ago
- Participants with controlled HIV, hepatitis B, or hepatitis C may be eligible if viral loads are undetectable and other criteria are met
- Prior infections must be controlled at enrollment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 9 days (Day -9 to Day -1)
Participants receive a preparative regimen including antithymocyte globulin, fludarabine, cyclophosphamide, and low dose total body irradiation before transplantation.
Daily visits for drug administration and radiation on Day -1
Duration - Approximately 35 days post-transplant
Participants receive a bone marrow transplant from either a haploidentical related donor or an unrelated donor, followed by graft-versus-host disease prophylaxis with post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil.
1 transplant procedure and daily visits for medication from Day +3 to Day +35
Duration - 1 year post-transplant
Participants are monitored for graft success, hematologic recovery, graft-versus-host disease, and overall health for one year after transplantation.
Regular follow-up visits including assessments at Day 28, Day 56, Day 100, Day 180, and Day 365 post-transplant
Trial Site Locations
Total: 25 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
Actively Recruiting
2
City of Hope
Duarte, California, United States, 91010
Actively Recruiting
3
University of California, Los Angeles
Los Angeles, California, United States, 90095
Actively Recruiting
4
Stanford University
Stanford, California, United States, 94305
Actively Recruiting
5
Moffitt Cancer Center
Tampa, Florida, United States, 33612
Actively Recruiting
6
Emory Winship Cancer Institute
Atlanta, Georgia, United States, 30322
Actively Recruiting
7
Blood and Marrow Transplant Center at Northside Hospital
Atlanta, Georgia, United States, 30342
Actively Recruiting
8
University of Kansas Medical Center
Westwood, Kansas, United States, 66205
Actively Recruiting
9
Johns Hopkins University
Baltimore, Maryland, United States, 21218
Actively Recruiting
10
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
11
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Actively Recruiting
12
Karmanos Cancer Institute
Detroit, Michigan, United States, 48201
Actively Recruiting
13
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
14
Washington University School of Medicine, Barnes-Jewish Hospital
St Louis, Missouri, United States, 63110
Actively Recruiting
15
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10021
Actively Recruiting
16
UNC Chapel Hill
Chapel Hill, North Carolina, United States, 27599
Actively Recruiting
17
Levine Cancer Institute
Charlotte, North Carolina, United States, 28204
Actively Recruiting
18
Duke University Health System
Durham, North Carolina, United States, 27705
Actively Recruiting
19
The Ohio State University
Columbus, Ohio, United States, 43210
Actively Recruiting
20
University of Oklahoma
Oklahoma City, Oklahoma, United States, 73117
Actively Recruiting
21
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
22
Vanderbilt University
Nashville, Tennessee, United States, 37235
Actively Recruiting
23
Huntsman Cancer Institute
Salt Lake City, Utah, United States, 84112
Actively Recruiting
24
Fred Hutchinson Cancer Center
Seattle, Washington, United States, 98109
Actively Recruiting
25
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
Research Team
J
Jennifer Romeril
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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