Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity

PHASE2RecruitingINTERVENTIONAL
Enrollment

66

Participants

Timeline

Start Date

September 22, 2020

Primary Completion Date

October 1, 2026

Study Completion Date

November 30, 2027

Conditions
Lymphoproliferative DisordersAutoimmune LymphoproliferativeImmune System DiseasesCommon Variable ImmunodeficiencyPrimary T-cell Immunodeficiency Disorders
Interventions
DRUG

Busulfan test dose

0.8 mg/kg IV infusion over 2 hours

DRUG

Fludarabine

40 mg/m2 IV infusion over 30 min once daily for 4 days

DRUG

Busulfan

"AUC Targeted Dose based on busulfan test dose PKs, IV infusion over 3 hours once daily (3.2 mg/kg IV per day will be the default dose) per the below time frame: For 10/10 Matched Related and Unrelated Donor Recipients For the High Intensity Arm, the busulfan dose will be given for 4 days (-6, -5, -4, and -3).~For Intermediate Intensity Arm, the busulfan dose will be given for 3 days (-6,-5, and -4).~For Low Intensity Arm, the busulfan dose will be given for 2 days on days (-6 and -5). 9/10 HLA Matched Related or Unrelated Donor Recipients For the High Intensity Arm, the busulfan dose will be given for 3 days (-6,~-5, and -4). For the the Intermediate Intensity Arm, the busulfan dose will be given for 2 days (-6 and -5).~For the Low Intensity Arm, the busulfan dose will be given for 1 day on day (-6)."

DRUG

Alemtuzumab

Alemtuzumab will be given if there is evidence of immune dysregulation 10 mg/m2 SC divided over three days (-14, -13, and -12)

RADIATION

Total body Irradiation

200 cGy Transplant Day -1 (Only for 9/10 HLA Matched Related or Unrelated Donor Recipients )

PROCEDURE

Allogeneic HSCT

Stem cell transplant

DRUG

Tacrolimus (Tacro)

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

DRUG

Mycophenolate mofetil (MMF)

Mycophenolate mofetil 15 mg/kg IV over 2 hours three times a day starting on day +5 will continue until Approximately+35 (+/- two days)

DRUG

Cyclophosphamide (Cytoxan)

Cyclophosphamide: 50 mg/kg IV once daily over 2 hours on days +3 and +4, dosed according to ideal body weight

Trial Locations (1)

20892

RECRUITING

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Cancer Institute (NCI)

NIH