Allogeneic Hematopoietic Stem Cell Transplantation With Briquilimab-Based Conditioning in Participants With GATA2 Deficiency

PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

7

Participants

Timeline

Start Date

November 29, 2023

Primary Completion Date

October 16, 2025

Study Completion Date

July 31, 2028

Conditions
GATA2Immunodeficiency
Interventions
DRUG

Mycophenolate Mofetil

15 mg/kg IV three times per day starting on day +5 until approximately day +30 (+/- 2 days)

DRUG

Tacrolimus

0.02 mg/kg IV daily starting on day +5

DRUG

Post-Transplant Cyclophosphamide

50 mg/kg IV daily on days +3 and +4

RADIATION

Total Body Irradiation

200cGy on day -1

PROCEDURE

Hematopoietic Cell Transplant

stem cell transplant on day 0

DRUG

Briquilimab

Single 0.6 mg/kg IV infusion administered between days -13 and day -10

DRUG

Cyclophosphamide

14.5 mg/kg IV daily on days -6 and -5; for 7/8 Unrelated or Haploidentical Donor, prior to transplant.

DRUG

Fludarabine

30 mg/m2 IV over 30 minutes daily. For 8/8 Matched Related or Unrelated Donor, fludarabine dose will be on days -4, -3, and -2. For 7/8 Unrelated or Haploidentical Donor, fludarabine dose will be on days -6, -5, -4, -3, and -2.

Trial Locations (1)

20892

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Cancer Institute (NCI)

NIH

NCT05907746 - Allogeneic Hematopoietic Stem Cell Transplantation With Briquilimab-Based Conditioning in Participants With GATA2 Deficiency | Biotech Hunter | Biotech Hunter