Status:
COMPLETED
Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases
Lead Sponsor:
Children's Hospital of Philadelphia
Conditions:
Bone Marrow Failure Syndromes
Immunodeficiencies
Eligibility:
All Genders
Up to 22 years
Phase:
PHASE2
Brief Summary
This is a Phase II trial to determine the ability of a reduced intensity conditioning regimen to allow successful engraftment with CD3+ /CD19+ depleted peripheral stem cell grafts from mismatched dono...
Detailed Description
This study will allow transplantation using a reduced intensity conditioning regimen for children with non-malignant diseases who lack a matched related or unrelated donor. Donors will be unrelated or...
Eligibility Criteria
Inclusion
- Bone marrow failure syndromes for which SCT is indicated, including severe aplastic anemia refractory to non transplant therapies congenital neutropenia, congenital thrombocytopenia, congenital red cell aplasia
- Immunodeficiencies for which allogeneic hematopoietic stem cell transplant is indicated, including severe combined immunodeficiencies, Wiskott-Aldrich syndrome, IPEX syndrome, X-linked lymphoproliferative disease
- Immune dysregulation syndromes, including refractory or recurrent hemophagocytic lymphohistiocytosis, HLH with genetic mutations, refractory multisystemic Langerhans cell histiocytosis, other MAS refractory to standard therapy
- Organ function clearance
Exclusion
- Uncontrolled bacterial, viral or fungal infections
- HLA matched related or unrelated donor able to donate mobilized peripheral stem cells.
- Fanconi's syndrome, dyskeratosis congenita or other chromosomal fragility syndromes
- Pregnant Females
Key Trial Info
Start Date :
November 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2016
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT02277639
Start Date
November 1 2011
End Date
March 1 2016
Last Update
April 11 2018
Active Locations (1)
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1
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104