CD34+ Cell Enriched and T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Mismatched Related Donors or Borderline Organ Function

Rajni Agarwal

Status: Completed

Conditions: Malignant Diseases; Non-malignant Diseases

Treatments: Device: CliniMACS CD34+ cell enrichment and T-cell depletion

Study type: Interventional

Funder types: Other

Identifiers

NCT02162511

28663

Details and patient eligibility

About

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.

Enrollment: 3 patients

Sex: All

Ages: Under 35 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

3 participants in 3 patient groups

ARM A Malignant TBI

Experimental group

Description:

Malignant diseases Conditioning including total body irradiation and chemotherapy

Treatment:

Device: CliniMACS CD34+ cell enrichment and T-cell depletion

ARM B Malignant Non-TBI

Experimental group

Description:

Malignant diseases chemotherapy based conditioning

Treatment:

Device: CliniMACS CD34+ cell enrichment and T-cell depletion

ARM C Non-malignant

Experimental group

Description:

Non-malignant diseases Chemotherapy based conditioning

Treatment:

Device: CliniMACS CD34+ cell enrichment and T-cell depletion