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

Conditions

• Severe Aplastic Anemia

Eligibility Criteria

Sex

ALL

Age

3 Years - 75 Years

Healthy Volunteers

Not accepted

Interventions

• Haploidentical donor bone marrow transplant — DRUG
  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.
• Unrelated donor bone marrow transplant — DRUG
  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.

Study Details

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

Key Dates

Start date

May 12, 2025

Status verified

Feb 2026

Primary completion

Aug 1, 2028

Completion

Feb 1, 2029

Study Design

Enrollment

60 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• 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.

Primary Outcome Measure

Graft versus host disease (GVHD)-free failure-free survival (GFFS) at One year [ Time Frame: 1 year after initiation of conditioning ]

Central Contacts

• Jennifer Romeril
  301-251-1161
  [email protected]

Locations (25)

Find similar trials in Birmingham, AL

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