Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

PHASE2RecruitingINTERVENTIONAL
Enrollment

27

Participants

Timeline

Start Date

February 12, 2020

Primary Completion Date

December 31, 2027

Study Completion Date

December 31, 2028

Conditions
Primary Immune Deficiency DisorderImmune Deficiency DiseaseBone Marrow FailureShort Telomere LengthFanconi AnemiaNon Fanconi DNA-DSB Repair Disorder
Interventions
DRUG

Alemtuzumab

Preparative regimen

DRUG

Fludarabine

Preparative regimen

DRUG

Melphalan

Preparative regimen

RADIATION

Low Dose Total Body Irradiation

Preparative regimen

DRUG

Cyclophosphamide

GVHD prophylaxis

DRUG

Tacrolimus

GVHD prophylaxis

DRUG

Mycophenolate Mofetil

GVHD prophylaxis

Trial Locations (1)

21287

RECRUITING

Johns Hopkins University, Baltimore

Sponsors
All Listed Sponsors
collaborator

Maryland Stem Cell Research Fund

UNKNOWN

lead

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

OTHER

NCT04232085 - Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures | Biotech Hunter | Biotech Hunter