Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations

PHASE2RecruitingINTERVENTIONAL
Enrollment

144

Participants

Timeline

Start Date

July 24, 2013

Primary Completion Date

December 31, 2027

Study Completion Date

December 31, 2028

Conditions
GATA2ImmunodeficiencyMDS
Interventions
PROCEDURE

Allogeneic HSCT

Stem cell transplant

DRUG

Busulfan Test dose

0.8 mg/kg IV infusion over 3 hours one time dose administered 5 to 14 days prior to start of preparative regimen (Days -11 to -20)

DRUG

Fludarabine (Fludara, Berlex Laboratories)

40 mg/m2 IV (in the vein) over 30 minutes (in the vein) once daily on Days -6, -5, -4, and -3 or 30 mg/m2 IV over 30 minutes (in the vein) once daily on Days -6, -5, -4, -3, and -2

DRUG

Busulfan (Busulfex)

3.2 mg/kg IV (in the vein) over 3 hours once daily on Days -6, -5, -4 and -3 (weight based dosing). If in Arm B and if poor or very poor risk clonal chromosomal abnormalities, busulfan will also be given on day -2.

DRUG

Cyclophosphamide (CTX, Cytoxan)

14.5 mg/kg IV (in the vein) infusion over 30 minutes once daily on days -6 and -5 (weight based dosing) or 50 mg/kg IV infusion over 2 hours on day -6 (weight based dosing). For post-transplant, 50/kg IV once daily x2 doses on days +3 and +4

PROCEDURE

Total Body Irradiation (TBI)

200 cGy on Day -1

DRUG

Mycophenolate mofetil (MMF)

15mg/kg IV over 2 hours BID starting on day +5 will continue until day +35 (+/- 2 days)

DRUG

Tacrolimus

0.02mg/kg IV continuous infusion over 24 hours starting on day +5 until day +180

BIOLOGICAL

Equine Anti-Thymocyte Globulin

(Deleted this intervention per amendment I): 30mg/kg IV (in the vein)once daily x 3 days on Days -6, -5, -4 (3 doses total)

Trial Locations (1)

20892

RECRUITING

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Cancer Institute (NCI)

NIH

NCT01861106 - Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations | Biotech Hunter | Biotech Hunter