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Phase I

A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime

  • Study HIC#:2000021396
  • Last Updated:12/22/2022

The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient for engraftment of donor hematopoietic cells. It is also to determine major toxicities from these conditioning regimens, within the first 100 days after transplantation.

  • Start Date06/22/2018
  • End Date12/31/2026

Trial Purpose and Description

Primary Outcome Measures :

  1. Donor engraftment as measured by chimerism [ Time Frame: 100 days post-transplant ]
  2. Major toxicities as graded by the CTC v4 [ Time Frame: 100 days post-transplant ]

Secondary Outcome Measures :

  1. Time to neutrophil and platelet engraftment as measured by complete blood counts [ Time Frame: Notapplicable ]
  2. Incidence of acute graft-versus-host disease as measured by protocol grading scale [ Time Frame: 100 days post-transplant ]
  3. Incidence of chronic graft-versus-host disease as measured by protocol grading scale [ Time Frame: 2 years post-transplant ]
  4. Long-term donor engraftment by donor chimerism [ Time Frame: 2 years post-transplant ]
  5. Immune reconstitution by laboratory evaluations [ Time Frame: 1 year post-transplant ]
  6. Overall and disease free survival [ Time Frame: 2 years post-transplant ]

Eligibility Criteria

Inclusion Criteria:

Stratum 1: Patient must have a hemoglobinopathy receiving a matched related transplant with bone marrow or peripheral blood stem cells.

Stratum 2: Patient must have thalassemia receiving an unrelated donor transplant with bone marrow or umbilical cord blood stem cells.

Stratum 3: Patient must have a hemoglobinopathy receiving a minimally mismatched unrelated donor transplant with bone marrow or single or double umbilical cord blood product.

Stratum 4: Patient must have a non-malignant disorder (excluding hemoglobinopathy) receiving a minor mismatched unrelated donor transplant with bone marrow or single or double umbilical cord blood product.

All strata:

  • Recipient age < 21 years
  • Lansky/Karnofsky >/= 40
  • Adequate pulmonary, renal, liver, and other organ function as defined in protocol
  • Negative pregnancy test
  • Adequate total nucleated cell or CD34+ dose of product as defined in protocol
  • If sickle cell, Hemoglobin S <45%

Exclusion Criteria:

  • HIV positive
  • Invasive infection
  • Pregnancy/lactating