Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

PHASE2RecruitingINTERVENTIONAL
Enrollment

56

Participants

Timeline

Start Date

February 19, 2019

Primary Completion Date

June 1, 2026

Study Completion Date

June 1, 2028

Conditions
Severe Aplastic Anemia (SAA)Hypo-Plastic Myelodysplastic Syndrome (MDS)Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
DRUG

Cyclophosphamide

This research protocol is therefore designed to evaluate the safety and effectiveness of using an unmanipulated G-CSF mobilized peripheral stem cell allograft from a haploidentical donor and post-transplant cyclophosphamide for patients with SAA, or SAA evolving to MDS, or PNH that has proven to be refractory to conventional immunosuppressive therapy (IST) in patients who lack an HLA-matched donor (sibling/ or matched unrelated donor.

OTHER

Peripheral Blood Stem Cells

This research protocol is therefore designed to evaluate the safety and effectiveness of using an unmanipulated G-CSF mobilized peripheral stem cell allograft from a haploidentical donor and post-transplant cyclophosphamide for patients with SAA, or SAA evolving to MDS, or PNH that has proven to be refractory to conventional immunosuppressive therapy (IST) in patients who lack an HLA-matched donor (sibling/ or matched unrelated donor.

Trial Locations (1)

20892

RECRUITING

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Heart, Lung, and Blood Institute (NHLBI)

NIH