Clinical trial NCT02162511: CD34+ Cell Enriched and T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Mismatched Related Donors or Borderline Organ Function
Official title: An Expanded Access Study Using the CliniMACS System to Offer Therapeutic Manipulated Grafts That Are CD34 Cell Enriched and T Cell Depleted for Allogeneic Stem Cell Recipients With Mismatched Related Donors or Borderline Organ Function
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Malignant Diseases
Non-malignant Diseases
Keywords: Haplocompatible
Stem cell transplant
Borderline organ function
Alternative donor stem cell transplant
Lead sponsor: Stanford University
Sponsor class: OTHER
First posted: 2014-06-12
Enrollment: 3
Sex: ALL
Maximum age (years): 35
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Number of Patients With Severe (Grade III/IV) Acute Graft vs Host Disease (GVHD)
timeFrame: Day +100
Secondary outcomes:
measure: Number of Participants With Graft Failure
timeFrame: Up to Day +42 after stem cell transplant
measure: Length of Time to Engraftment
timeFrame: up to +1 year post-transplant
measure: Chimerism of Donor Cells
timeFrame: Day +100 post-transplant
measure: Immune Recovery (CD4)
timeFrame: up to +1 year post-transplant
measure: Number of Participants With Immune Recovery (CD4 >200) by Year 1
timeFrame: up to +1 year post-transplant
measure: Immune Recovery Shown as Phytohemagglutin (PHA)
timeFrame: 6 months and 1 year post-transplant
measure: Number of Patients With Post-transplant Lymphoproliferative Disease (PTLD)
timeFrame: up to +1 year post-transplant
measure: Number of Patients With Severe Toxicities
timeFrame: up to +1 year post-transplant
measure: Number of Participants Experiencing Post-transplant Infections
timeFrame: up to +1 year post-transplant
measure: Transplant-related Mortality (TRM)
timeFrame: at Day +100 and +1 year post-transplant
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford Children's Hospital
state: California
Study officials:
affiliation: Stanford University
name: Rajni Agarwal, MD
role: PRINCIPAL_INVESTIGATOR
References: