Alpha/Beta T-cell Depleted Blood-forming Stem Cell Transplant From Related or Unrelated Donors for Blood Diseases in Children and Young Adults

ConditionBlood Disease
Trial statusNot yet recruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age3-25
SponsorUniversity of Wisconsin, Madison

About this trial

This study is being done to see if the investigators can take peripheral blood stem cells from either an adult family member or a closely matched unrelated donor, and run them through a special lab instrument to remove alpha/beta T cells and B cells and then give them to the patient to treat disease. This is an experimental way of doing a hematopoietic stem cell transplant (HSCT). The investigators want to see if the new stem cells will grow without bad graft vs. host disease (GVHD). This treatment approach is experimental in the United States.

Eligibility criteria

Qualifiers

No Human leukocyte antigen (HLA) identical sibling available AND

NO HLA matched unrelated donor available OR urgent need of HSCT precludes time necessary to search for suitable HLA matched unrelated donor AND

Haploidentical donor OR closely matched unrelated donor available and willing to undergo mobilization and apheresis

If subject has genetically confirmed inherited bone marrow failure, related donor must be evaluated for this disorder and testing must be negative.

Disqualifiers

Pregnant

HIV infection

Uncontrolled, serious active infection at screening

Significant serious intercurrent illnesses

Trial design

Treatments tested in this trial

  • TCRαβ+/CD19+ depleted Hematopoietic stem cell (HSC) graft
  • CliniMACS® System

Treatment groups

12 Participants
are divided into 1 treatment group

Locations

This trial has no locations