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

NCT02162511 | Completed Results DMC FDA Device

• 1 other identifier: 28663
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

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.

Conditions

Malignant Diseases; Non-malignant Diseases

Keywords

Haplocompatible; Stem cell transplant; Borderline organ function; Alternative donor stem cell transplant

Outcome Measures

Primary Outcomes (1)

• Number of Patients With Severe (Grade III/IV) Acute Graft vs Host Disease (GVHD)

  GVHD is a condition that occurs when donor bone marrow or stem cells attack the recipient.

  Day +100

Secondary Outcomes (10)

• Number of Participants With Graft Failure

  Up to Day +42 after stem cell transplant

• Length of Time to Engraftment

  up to +1 year post-transplant

• Chimerism of Donor Cells

  Day +100 post-transplant

• Immune Recovery (CD4)

  up to +1 year post-transplant

• Number of Participants With Immune Recovery (CD4 >200) by Year 1

  up to +1 year post-transplant

Study Arms (3)

ARM A Malignant TBI

EXPERIMENTAL

Malignant diseases Conditioning including total body irradiation and chemotherapy

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

ARM B Malignant Non-TBI

EXPERIMENTAL

Malignant diseases chemotherapy based conditioning

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

ARM C Non-malignant

EXPERIMENTAL

Non-malignant diseases Chemotherapy based conditioning

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

Interventions

CliniMACS CD34+ cell enrichment and T-cell depletion DEVICE

ARM A Malignant TBI; ARM B Malignant Non-TBI; ARM C Non-malignant

Eligibility Criteria

• Age: Up to 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Rajni Agarwal: lead

Study Sites (1)

Stanford Children's Hospital

Palo Alto, California, 94305, United States

Location

Related Links

• Stanford Stem Cell Transplantation website

Limitations and Caveats

This study did not meet its planned enrollment. Statistical power was not achieved for any analysis.

Results Point of Contact

• Title: Erica Goodwin
• Organization: Stanford University

egood55@stanford.edu

253-508-3556

Study Officials

• Rajni Agarwal, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: June 10, 2014
• First Posted: June 12, 2014
• Study Start: May 1, 2014
• Primary Completion: July 1, 2019
• Study Completion: March 1, 2023
• Last Updated: June 26, 2023
• Results First Posted: June 26, 2023

Record last verified: 2023-05

Locations

US (1)