Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia (NCT06517641)

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

1. Age 3 years to 75 years
2. Confirmed diagnosis of acquired SAA defined as: a. Bone marrow cellularity \< 25% or variable marrow cellularity but with \< 30% residual hematopoietic cells deemed HYPOcellular for age AND b. Two (2) out of 3 of the following (in peripheral blood). i. Neutrophils \< 0.5 x109/L ii. Platelets \< 20 x109/L iii. Reticulocyte count \< 20 x109/L (\< 60 x 109/L using an automated analysis)
3. No suitable fully matched related donor as per Investigator's discretion (6/6 match for HLA A and B at intermediate or high-resolution and DRB1 at high-resolution using deoxyribonucleic acid \[DNA\]-based typing) available.
4. Available donor as defined in the protocol.
5. Participant and/or legal guardian must sign informed consent.
6. Adequate organ function defined by institutional transplant standards or defined as below:
7. Cardiac: Left ventricular ejection fraction (LVEF) at rest \> 40% with no clinical signs of cardiac failure. For participants aged \< 13 years, shortening fraction (SF) ≥ 26% by echocardiogram or multigated acquisition (MUGA) may be substituted for LVEF.
8. Hepatic: Total bilirubin \< 2.0 mg/dL unless Gilbert's disease is present
9. Renal: For participants \> 13.0 years of age at the time of enrollment: estimated creatinine clearance (CrCl) \> 60 mL/minute (per institutional standard). For participants \< 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 m2, then renal function must be measured by 24-hour creatinine clearance or nuclear GFR, and must be \> 50 mL/min/1.73 m2.
10. Pulmonary: i. For participants \> 13.0 years of age: Diffusing capacity of the lung for carbon monoxide (DLCO, corrected/adjusted for hemoglobin \[Hb\]) \> 50%, or Spirometry with forced expiratory volume 1 (FEV1) \> 50% predicted (without administration of bronchodilator) and forced vital capacity (FVC) \> 50% predicted. ii. For participants \< 13.0 years of age unable to perform pulmonary function tests (PFTs) 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.

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (25 Sites)

• University of Alabama at Birmingham — Birmingham, Alabama
• City of Hope — Duarte, California
• University of California, Los Angeles — Los Angeles, California
• Stanford University — Stanford, California
• Moffitt Cancer Center — Tampa, Florida
• Emory Winship Cancer Institute — Atlanta, Georgia
• Blood and Marrow Transplant Center at Northside Hospital — Atlanta, Georgia
• University of Kansas Medical Center — Westwood, Kansas
• Johns Hopkins University — Baltimore, Maryland
• Massachusetts General Hospital — Boston, Massachusetts
• Dana Farber Cancer Institute — Boston, Massachusetts
• Karmanos Cancer Institute — Detroit, Michigan
• Mayo Clinic — Rochester, Minnesota
• Washington University School of Medicine, Barnes-Jewish Hospital — St Louis, Missouri
• Memorial Sloan Kettering Cancer Center — New York, New York
• UNC Chapel Hill — Chapel Hill, North Carolina
• Levine Cancer Institute — Charlotte, North Carolina
• Duke University Health System — Durham, North Carolina
• The Ohio State University — Columbus, Ohio
• University of Oklahoma — Oklahoma City, Oklahoma
• University of Pennsylvania — Philadelphia, Pennsylvania
• Vanderbilt University — Nashville, Tennessee
• Huntsman Cancer Institute — Salt Lake City, Utah
• Fred Hutchinson Cancer Center — Seattle, Washington
• Medical College of Wisconsin — Milwaukee, Wisconsin

💰 Cost, Insurance & Patient Rights

• 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
• Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
• Voluntary Participation: You may withdraw at any time without affecting your standard medical care.