N/A
N=3
CD34+ Cell Enriched and T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Mismatched Related Donors or Borderline Organ Function
Malignant Diseases · Non-malignant Diseases
Bottom Line
View on ClinicalTrials.gov: NCT02162511 ↗Enrolled (actual)
3
Serious AEs
0.0%
Results posted
Jun 2023
Primary outcome: Primary: Number of Patients With Severe (Grade III/IV) Acute Graft vs Host Disease (GVHD) — 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- CliniMACS CD34+ cell enrichment and T-cell depletion (Device)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Rajni Agarwal
- Primary completion
- Jul 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients With Severe (Grade III/IV) Acute Graft vs Host Disease (GVHD) |
— | — |
| SECONDARY Number of Participants With Graft Failure |
2 | — |
| SECONDARY Length of Time to Engraftment |
10 | — |
| SECONDARY Chimerism of Donor Cells |
95; 70; 100; 99; 98; 99 | — |
| SECONDARY Immune Recovery (CD4) |
148 | — |
| SECONDARY Number of Participants With Immune Recovery (CD4 >200) by Year 1 |
— | — |
| SECONDARY Immune Recovery Shown as Phytohemagglutin (PHA) |
33736; 110809 | — |
| SECONDARY Number of Patients With Post-transplant Lymphoproliferative Disease (PTLD) |
— | — |
| SECONDARY Number of Patients With Severe Toxicities |
2 | — |
| SECONDARY Number of Participants Experiencing Post-transplant Infections |
1; 1; 1; 2; 1; 1 | — |
| SECONDARY Transplant-related Mortality (TRM) |
1; 1 | — |
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.
Data sourced from ClinicalTrials.gov (NCT02162511). 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.