ExpMACs: Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC
Study Details
Study Description
Brief Summary
The goal 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 IRB approved protocols that utilize CliniMACs technology for T depletion.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Early Phase 1 |
Detailed Description
Only 25-30% of patients who may benefit from HSCT have a matched related donor. There is a higher rate of complications using cells from an unrelated or partially matched related donor. T cells within the donor cells may cause a complication called graft vs. host disease (GVHD). The goal of this study is to use the CliniMACs device to remove the T cells that cause GVHD, called T cell depletion.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Expanded access to CliniMACs device for T cell depletion access for patients who lack a fully HLA matched sibling, and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-open protocols that utilize CliniMACs technology for T depletion. Subjects will undergo transplant of stem cells with CD3+/CD19+ depletion. |
Device: Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
Processing of stem cells using the CliniMACs device to selectively deplete specific T cells to decrease risk of graft versus host disease when using donor stem cells which are not fully matched.
Other Names:
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Outcome Measures
Primary Outcome Measures
- Overall Survival [1 year]
Secondary Outcome Measures
- Graft versus Host Disease [1 year]
- Graft Failure [100 days]
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Patients who lack a fully HLA matched sibling and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT) but do not meet criteria for current open institutional protocols using ClinMACs device for CD3+/CD19+ depletion.
-
Patients with the following transplantable diseases:
Non-malignant diseases:
Metabolic storage diseases correctable by HSCT
Bone marrow failure syndromes
Immunodeficiencies/immune dysregulation syndromes
Malignant diseases:
Acute leukemias
Chronic leukemias
Lymphomas
Myelodyplastic syndrome
- Organ function criteria:
Lansky or Karnofsky performance ≥60
Serum creatinine ≤3xupper limit of normal for age
Hepatic: Transaminases ≤10xnormal
Cardiac shortening fraction ≥27%
Bilirubin <2.5x normal (unless elevation due to Gilberts disease)
-
No active untreated infection
-
Signed informed consent
-
No fully HLA matched sibling donor available
-
Females of childbearing potential must have negative pregnancy test
Exclusion Criteria:
-
Uncontrolled bacterial, viral or fungal infections
-
Fully HLA matched sibling donor
-
Donor unable to donate peripheral stem cells
-
Pregnant Females
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | United States | 19104 |
Sponsors and Collaborators
- Children's Hospital of Philadelphia
Investigators
- Principal Investigator: Nancy J Bunin, MD, Children's Hospital of Philadelphia
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 13-010286
- 12BT125