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

PHASE2RecruitingINTERVENTIONAL
Enrollment

60

Participants

Timeline

Start Date

May 12, 2025

Primary Completion Date

August 1, 2028

Study Completion Date

February 1, 2029

Conditions
Severe Aplastic Anemia
Interventions
DRUG

Haploidentical donor bone marrow transplant

"Drugs:~1. Antithymocyte Globulin (ATG) dose will be 0.5 mg/kg IV on Day -9 over 6 hours and 2 mg/kg IV on Days -8 and -7 over 4 hours.~2. Fludarabine dose will be 30 mg/m\^2 IV daily for 5 days from Day -6 to Day -2.~3. Cyclophosphamide dose will be 14.5 mg/kg IV daily for 2 days (Day -6 to Day -5) prior to transplantation and 50 mg/kg IV daily for 2 days (Day +3 to Day +4) after transplantation.~4. Tacrolimus should be started on Day +5 and administered to maintain a level of 10-15 ng/mL.~5. Mycophenolate mofetil (MMF) dose will be 15 mg/kg PO three times a day (TID) up to 1 gm TID (or IV equivalent) starting on Day +5 through Day +35.~6. G-CSF will be given IV or SQ starting on Day +5 at 5 mcg/kg/day until ANC is \> 1500 for 3 days.~Radiation:~1\. Total Body Irradiation (TBI): will be given as a single dose of 400 cGy on Day -1.~Procedure:~1\. HSCT: Eligible patients will receive a haploidentical donor bone marrow transplant."

DRUG

Unrelated donor bone marrow transplant

"Drugs:~1. Antithymocyte Globulin (ATG) dose will be 0.5 mg/kg IV on Day -9 over 6 hours and 2 mg/kg IV on Days -8 and -7 over 4 hours.~2. Fludarabine dose will be 30 mg/m\^2 IV daily for 5 days from Day -6 to Day -2.~3. Cyclophosphamide dose will be 14.5 mg/kg IV daily for 2 days (Day -6 to Day -5) prior to transplantation and 50 mg/kg IV daily for 2 days (Day +3 to Day +4) after transplantation.~4. Tacrolimus should be started on Day +5 and administered to maintain a level of 10-15 ng/mL.~5. Mycophenolate mofetil (MMF) dose will be 15 mg/kg PO three times a day (TID) up to 1 gm TID (or IV equivalent) starting on Day +5 through Day +35.~6. G-CSF will be given IV or SQ starting on Day +5 at 5 mcg/kg/day until ANC is \> 1500 for 3 days.~Radiation:~1\. Total Body Irradiation (TBI): will be given as a single dose of 400 cGy on Day -1.~Procedure:~1\. HSCT: Eligible patients will receive an unrelated donor bone marrow transplant."

Trial Locations (25)

10021

RECRUITING

Memorial Sloan Kettering Cancer Center, New York

19104

RECRUITING

University of Pennsylvania, Philadelphia

21218

RECRUITING

Johns Hopkins University, Baltimore

27599

RECRUITING

UNC Chapel Hill, Chapel Hill

27705

RECRUITING

Duke University Health System, Durham

28204

RECRUITING

Levine Cancer Institute, Charlotte

30322

NOT_YET_RECRUITING

Emory Winship Cancer Institute, Atlanta

30342

RECRUITING

Blood and Marrow Transplant Center at Northside Hospital, Atlanta

33612

RECRUITING

Moffitt Cancer Center, Tampa

35294

RECRUITING

University of Alabama at Birmingham, Birmingham

37235

RECRUITING

Vanderbilt University, Nashville

43210

RECRUITING

The Ohio State University, Columbus

48201

RECRUITING

Karmanos Cancer Institute, Detroit

53226

RECRUITING

Medical College of Wisconsin, Milwaukee

55905

NOT_YET_RECRUITING

Mayo Clinic, Rochester

63110

RECRUITING

Washington University School of Medicine, Barnes-Jewish Hospital, St Louis

66205

RECRUITING

University of Kansas Medical Center, Westwood

73117

NOT_YET_RECRUITING

University of Oklahoma, Oklahoma City

84112

NOT_YET_RECRUITING

Huntsman Cancer Institute, Salt Lake City

90095

NOT_YET_RECRUITING

University of California, Los Angeles, Los Angeles

91010

RECRUITING

City of Hope, Duarte

94305

RECRUITING

Stanford University, Stanford

98109

RECRUITING

Fred Hutchinson Cancer Center, Seattle

02114

RECRUITING

Massachusetts General Hospital, Boston

02215

RECRUITING

Dana Farber Cancer Institute, Boston

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

collaborator

National Cancer Institute (NCI)

NIH

collaborator

Sanofi

INDUSTRY

lead

Medical College of Wisconsin

OTHER

NCT06517641 - Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia | Biotech Hunter | Biotech Hunter