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

Status: Recruiting
Location: See all (25) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

BMT CTN 2207 will investigate the use of marrow transplantation for treatment of severe aplastic anemia that has not previously been treated.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 3
Maximum Age: 75
Healthy Volunteers: f
View:

• Age 3 years to 75 years

• 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)

• 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.

• Available donor as defined in the protocol.

• Participant and/or legal guardian must sign informed consent.

• Adequate organ function defined by institutional transplant standards or defined as below:

‣ 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.

⁃ Hepatic: Total bilirubin \< 2.0 mg/dL unless Gilbert's disease is present

⁃ 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.

⁃ 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 \[e.g., Utah, 4,200 feet above sea level, does not give supplemental oxygen unless below 90%\]).

• Karnofsky or Lansky performance status ≥ 60%.

• Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence.

Locations
United States
Alabama
University of Alabama at Birmingham
RECRUITING
Birmingham
California
City of Hope
RECRUITING
Duarte
University of California, Los Angeles
NOT_YET_RECRUITING
Los Angeles
Stanford University
RECRUITING
Stanford
Florida
Moffitt Cancer Center
RECRUITING
Tampa
Georgia
Blood and Marrow Transplant Center at Northside Hospital
RECRUITING
Atlanta
Emory Winship Cancer Institute
NOT_YET_RECRUITING
Atlanta
Kansas
University of Kansas Medical Center
RECRUITING
Westwood
Massachusetts
Dana Farber Cancer Institute
RECRUITING
Boston
Massachusetts General Hospital
RECRUITING
Boston
Maryland
Johns Hopkins University
RECRUITING
Baltimore
Michigan
Karmanos Cancer Institute
RECRUITING
Detroit
Minnesota
Mayo Clinic
NOT_YET_RECRUITING
Rochester
Missouri
Washington University School of Medicine, Barnes-Jewish Hospital
RECRUITING
St Louis
North Carolina
UNC Chapel Hill
RECRUITING
Chapel Hill
Levine Cancer Institute
RECRUITING
Charlotte
Duke University Health System
RECRUITING
Durham
New York
Memorial Sloan Kettering Cancer Center
RECRUITING
New York
Ohio
The Ohio State University
RECRUITING
Columbus
Oklahoma
University of Oklahoma
NOT_YET_RECRUITING
Oklahoma City
Pennsylvania
University of Pennsylvania
RECRUITING
Philadelphia
Tennessee
Vanderbilt University
RECRUITING
Nashville
Utah
Huntsman Cancer Institute
NOT_YET_RECRUITING
Salt Lake City
Washington
Fred Hutchinson Cancer Center
RECRUITING
Seattle
Wisconsin
Medical College of Wisconsin
RECRUITING
Milwaukee
Contact Information
Primary
Jennifer Romeril
bmtctn2207@emmes.com
301-251-1161
Time Frame
Start Date: 2025-05-12
Estimated Completion Date: 2029-02-01
Participants
Target number of participants: 60
Treatments
Active_comparator: Haploidentical transplantation
Patients receiving bone marrow transplanted from a haploidentical related donor will be included in this arm.
Active_comparator: Unrelated donor transplantation
Patients receiving bone marrow transplanted from an unrelated donor will be included in this arm.
Sponsors
Leads: Medical College of Wisconsin
Collaborators: Sanofi, National Cancer Institute (NCI), National Heart, Lung, and Blood Institute (NHLBI)

This content was sourced from clinicaltrials.gov