About this trial

The primary objective of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor, and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia Institutional Review Board (IRB) approved protocols that utilize CliniMACs technology for T depletion.

Eligibility criteria

Qualifiers

Metabolic storage diseases correctable by HSCT

Bone marrow failure syndromes

Immunodeficiencies/immune dysregulation syndromes

Sickle cell disease or thalassemia

Disqualifiers

Uncontrolled bacterial, viral, or fungal infections

Fully HLA matched sibling donor (fully matched related donors including siblings may be included in special circumstances)

Donor unable to donate peripheral stem cells

Pregnant Females

Trial design

Treatments tested in this trial

  • Apha/beta T and CD19+ cell depletion using CliniMACS device

Treatment groups

No treatment groups listed

Locations

1
Children's Hospital of Philadelphia19104, PhiladelphiaPennsylvania, United States

Sponsors and collaborators

Children's Hospital of Philadelphia

Lead sponsor