Phase 2
N=2
Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases
Bone Marrow Failure Syndromes · Immunodeficiencies · Immune Dysregulation Syndromes
Bottom Line
View on ClinicalTrials.gov: NCT02277639 ↗Enrolled (actual)
2
Serious AEs
0.0%
Results posted
Apr 2018
Primary outcome: Primary: Number of Participants With Engraftment — 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- CliniMACs device (Device)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Children's Hospital of Philadelphia
- Primary completion
- Mar 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Engraftment |
2 | — |
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 donors. There are two conditioning regimens depending upon patient diagnosis and age.
Eligibility Criteria
Inclusion Criteria
- 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 Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT02277639). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.