New transplant approach shows promise for rare blood disorder

NCT ID NCT02918292

Disease control Sponsor: Medical College of Wisconsin Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a stem cell transplant from a half-matched family donor for people with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. The goal was to see if this approach could improve survival one year after the transplant. The trial involved 32 participants and used a special drug regimen to help the body accept the new cells.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

32 people

The number who actually took part.

Started

Jul 2017

Finished

Aug 2021

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Patient is \< 75 years of age at time of enrollment. 2. Confirmed diagnosis of SAA, either from initial diagnosis or follow-up assessments, defined as: 1. Bone marrow cellularity \< 25% or marrow cellularity \< 50% but with \< 30% residual hematopoietic cells. 2. Two out of three of the following (in peripheral blood): Neutrophils \< 0.5 x10\^9/L, Platelets \< 20 x10\^9/L, or Reticulocyte count \< 20 x10\^9/L (\<60 x 10\^9/L using an automated analysis) 3. No suitable fully matched related sibling donor (6/6 match for human leukocyte antigen (HLA)-A and B at intermediate or high resolution and DRB1 at high resolution using DNA-based typing) available. 4. Failed at least one trial of immunosuppressive therapy (IST) by being refractory or having relapsed. IST could have included ATG based regimens, calcineurin inhibitors and/or other higher dose therapy directed at the treatment of primary SAA. 5. Available relative of the patient who is a haploidentical match, including biological parents, siblings or half siblings, children, uncles/aunts, first cousins, etc. Eligible haploidentical donors will have 2-4 mismatches if HLA-A, -B, -C, and -DRB1 typing is used; 2-5 mismatches if HLA-A, -B, -C, -DRB1, and -DQB1 typing is used; and 2-6 mismatches if HLA-A, -B, -C, -DRB1, -DQB1, and -DPB1 typing is used. A unidirectional mismatch in either the graft versus host or host versus graft direction is considered a mismatch. The donor and recipient must demonstrate that they are a full haplotype match by being identical at a minimum of one allele (at high resolution DNA-based typing) at the following genetic loci: HLA-A, -B, -C, and DRB1 if 8 allele typing is used; HLA-A, -B, -C, -DRB1, and -DQB1 if 10 allele typing is used; and HLA-A, -B, -C, -DRB1-, DQB1, and -DPB1 is 12 allele typing is used by the local center. See Section 2.4 for additional information. 6. Patient and/or legal guardian must sign informed consent for HSCT. 7. The haplo donor and/or legal guardian must be able to sign informed consent documents. 8. The potential haplo donor must be willing and able to donate bone marrow. 9. The weight of the haplo donor must be ≥ 20 kg. 10. Adequate organ function defined as: 1. Cardiac: Left ventricular ejection fraction (LVEF) at rest ≥ 40%. For patients aged \< 13 years, shortening fraction (SF) ≥ 26% by echocardiogram or Multi Gated Acquisition Scan (MUGA) may be substituted for LVEF. 2. Hepatic: Total bilirubin \< 3.0 x the upper limit of normal (ULN) for age (patients who have been diagnosed with Gilbert's Disease are allowed to exceed this limit) and alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \< 5.0 x ULN for age. 3. Renal: For patients \> 13.0 years of age at the time of enrollment: estimated creatinine clearance \> 50 mL/minute (using the Cockcroft-Gault formula and actual body weight). For patients \< 13.0 years of age at enrollment: Glomerular Filtration Rate (GFR) estimated by the updated Schwartz formula ≥ 90 mL/min/1.73 m2. If the estimated GFR is \< 90 mL/min/1.73 m\^2, then renal function must be measured by 24-hour creatinine clearance or nuclear GFR, and must be \> 50 mL/min/1.73 m\^2. 4. Pulmonary: For patients \> 13.0 years of age: Diffusing capacity of the lung for carbon monoxide (DLCO) (corrected/adjusted for hemoglobin) \> 40% and forced expiratory volume in one second (FEV1) \> 50% predicted (without administration of bronchodilator) and forced vital capacity (FVC) \> 50% predicted. For patients \< 13.0 years of age unable to perform pulmonary function tests (PFT) due to age or developmental ability: (1) no evidence of dyspnea at rest and (2) no need for supplemental oxygen and (3) O2 saturation \> 92% on room air at sea level (with lower levels allowed at higher elevations per established center standard of care (e.g., Utah, 4,200 feet above sea level, does not give supplemental oxygen unless below 90%)). 11. Karnofsky or Lansky performance status ≥ 60%. 12. Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence. Exclusion Criteria: 1. Inherited bone marrow failure syndromes such as Fanconi anemia must be ruled out according to center standard. 2. Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre-MDS) or MDS on marrow examination (e.g. Monosomy 7). 3. Presence of anti-donor HLA antibodies (positive anti-donor HLA antibody is defined as a positive cross-match test of any titer by complement-dependent cytotoxicity or flow cytometric testing or the presence of anti-donor HLA antibody to the high expression loci HLA-A, B, C, DRB1, or DPB1 with mean fluorescence intensity (MFI) \> 1000 by solid phase immunoassay). 4. Prior allogeneic stem cell transplant. 5. Prior solid organ transplant. 6. Known life-threatening reaction (i.e., anaphylaxis) to Thymoglobulin® that would prohibit use for the patient as this study requires use of the Thymoglobulin® preparation of ATG. 7. Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as currently taking medication and with progression or no clinical improvement on adequate medical treatment. 8. Seropositive for the human immunodeficiency virus (HIV). 9. Active Hepatitis B or C determined by a detectable viral load of HBV or HCV. 10. Female patients who are pregnant (per institutional practice) or breast-feeding. 11. Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ. Cancer treated with curative intent \> 5 years previously will be allowed. Cancer treated with curative intent ≤ 5 years previously will not be allowed unless approved by the Protocol Chairs and/or Protocol Officer. 12. Alemtuzumab or ATG within 2 weeks of enrollment.

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Conditions

The condition(s) this trial relates to.

acquired aplastic anemia aplastic anemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Severe aplastic anemia

Contacts and locations

Locations

All Children's Hospital

St. Petersburg, Florida, 33701, United States
Baylor College of Medicine

Houston, Texas, 77030, United States
Children's Hospital and Research Center Oakland

Oakland, California, 94618, United States
Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital of Wisconsin/Midwest Children's Cancer

Milwaukee, Wisconsin, 53211, United States
Children's Mercy Hospital and Clinics

Kansas City, Missouri, 64108, United States
Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States
City of Hope National Medical Center

Duarte, California, 91010, United States
Duke University Medical Center

Durham, North Carolina, 27705, United States
Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States
H. Lee Moffitt Cancer Center

Tampa, Florida, 33624, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Indiana University Medical Center/ Riley Hospital for Children

Indianapolis, Indiana, 46202, United States
Johns Hopkins Unversity

Baltimore, Maryland, 21231, United States
Karmanos Cancer Institute/Children's Hospital of Michigan

Detroit, Michigan, 48201, United States
Miami Children's Hospital

Miami, Florida, 33155, United States
Northside Hospital

Atlanta, Georgia, 30342, United States
Oregon Health and Science University

Portland, Oregon, 97239-3098, United States
Penn State College of Medicine/The Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States
Roswell Park Cancer Institute

Buffalo, New York, 14263, United States
Stanford Hospital and Clinics

Stanford, California, 94305, United States
Texas Transplant Institute

San Antonio, Texas, 78229, United States
University of Florida College of Medicine

Gainesville, Florida, 32610, United States
University of Louisville/Kosair Children's Hospital

Louisville, Kentucky, 40202, United States
University of Michigan Medical Center

Ann Arbor, Michigan, 48105, United States
University of Pennsylvania Cancer Center

Philadelphia, Pennsylvania, 19104, United States
University of Texas/MD Anderson Cancer Center

Houston, Texas, 77030, United States
Washington University/Barnes Jewish Hospital

St Louis, Missouri, 63110, United States

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