Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

NARecruitingINTERVENTIONAL
Enrollment

30

Participants

Timeline

Start Date

September 4, 2012

Primary Completion Date

December 31, 2025

Study Completion Date

December 31, 2026

Conditions
SCIDOmenn's SyndromeReticular DysgenesisWiskott-Aldrich SyndromeBare Lymphocyte SyndromeCommon Variable ImmunodeficiencyChronic Granulomatous DiseaseCD40 Ligand DeficiencyHyper IgM SyndromeX-linked Lymphoproliferative DiseaseHemophagocytic LymphohistiocytosisGriscelli SyndromeChediak-Higashi SyndromeLangerhan's Cell Histiocytosis
Interventions
DRUG

Alemtuzumab 0.3 mg

0.3 mg/kg intravenously (IV) on days -12 through -10

DRUG

Cyclophosphamide

cyclophosphamide 50 mg/kg IV on days -9 through -6

DRUG

Busulfan

busulfan 0.8 or 1.1 mg/kg IV on days -5 through -2

BIOLOGICAL

Stem Cell Transplantation

"Unrelated donor bone marrow will be collected in the usual manner using established parameters determined by the National Marrow Donor Program. A minimum of 3 x 10\^8 nucleated cells/kg recipient weight will be collected with a goal of ≥ 5 x 10\^8 nucleated cells/kg recipient weight.~Umbilical cord blood selection will be per the current University of Minnesota Cord Blood Unit Selection algorithm. One or two units may be used to obtain the minimum cell dose. One of the UCB units selected for transplantation must contain ≥ 3.5 x 10\^7 nucleated cells/kg recipient weight based on cell numbers at time of cryopreservation, and the total combined cell dose of both units must be \> 5.0 x 10\^7 nucleated cells/kg."

DRUG

Fludarabine phosphate 40 mg

40 mg/m\^2 IV on days -5 through -2 (for children \< 6 months and/or \< 10 kg weight dose at 1.33 mg/kg)

DRUG

Melphalan

140 mg/m\^2 IV on day -3

DRUG

Alemtuzumab 0.2 mg

0.2 mg/kg intravenously (IV) on days -14 through -10

DRUG

Busulfan

busulfan 0.8 or 1.1 mg/kg IV on days -9 through -6

DRUG

Fludarabine phosphate 30 mg

fludarabine 30 mg/m\^2 IV on days -8 through -4

DRUG

MESNA

administered as per the standard institutional protocol.

Trial Locations (1)

55455

RECRUITING

Masonic Cancer Center, University of Minnesota, Minneapolis

All Listed Sponsors
lead

Masonic Cancer Center, University of Minnesota

OTHER