CD34+ Cell Enriched and T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Mismatched Related Donors or Borderline Organ Function
Study Details
Study Description
Brief Summary
The purpose of this protocol is to provide access to the CliniMACS® System to hematopoietic cell transplant (HSCT) patients who do not have a matched related donor. The CliniMACS system is currently approved for use in patients who have AML, and a genetically matched sibling donor. Through this protocol, the investigators will be able to offer potentially life-saving transplants to patients who have genetically mis-matched donor, who have no other options for treatment.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: ARM A Malignant TBI Malignant diseases Conditioning including total body irradiation and chemotherapy |
Device: CliniMACS CD34+ cell enrichment and T-cell depletion
|
| Experimental: ARM B Malignant Non-TBI Malignant diseases chemotherapy based conditioning |
Device: CliniMACS CD34+ cell enrichment and T-cell depletion
|
| Experimental: ARM C Non-malignant Non-malignant diseases Chemotherapy based conditioning |
Device: CliniMACS CD34+ cell enrichment and T-cell depletion
|
Outcome Measures
Primary Outcome Measures
- Incidence of severe (Grade III/IV) acute graft versus host disease [Day 100 post transplant]
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Participant age is 0 (newborn) to 35 years-old.
-
Participant has a disorder affecting the hematopoietic system that are inherited, acquired, or a result from the myeloablative treatment that can benefit from alternative stem cell transplantation according to standard practice guidelines for including patients for transplant.
-
Participant's medical screening clears s/he for allogeneic transplantation as per current institutional SOP based on standards of foundation for accreditation of cellular therapy and stem cell transplantation (FACT);
-
Participant must lack a healthy, HLA-identical related or unrelated donor unless s/he has a borderline organ function that will preclude the recipient from receiving a curative therapy due to the need of post-HSCT immunosuppressive therapy.
-
Participant must have a matched or mismatched-related donor who is:
-
Able to receive granulocyte colony-stimulating factor (G-CSF) and undergo apheresis either through placement of catheters in antecubital veins or a temporary central venous catheter OR agrees on a bone marrow harvest;
-
Healthy as per donor selection screening (following current SOP based on standards of foundation for accreditation of cellular therapy and stem cell transplantation - FACT);
-
Willing to participate and sign consent.
-
Participant or Legal Authorized Representative is able to sign informed consent (and signed assent, if applicable) for transplant.
Exclusion Criteria:
-
Participant does not qualify for an allogeneic transplant due to medical screening, underlying disease, or lack of alternative donors.
-
Any condition that compromises compliance with the procedures of this protocol, as judged by the principal investigator.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Stanford Children's Hospital | Palo Alto | California | United States | 94305 |
Sponsors and Collaborators
- Rajni Agarwal
Investigators
- Principal Investigator: Rajni Agarwal, MD, Stanford University
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 28663