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

• ClinicalTrials.gov Identifier: NCT02162511
• Recruitment Status: Unknown
• First Posted: June 12, 2014
• Last Update Posted: May 21, 2015
• Sponsor: Rajni Agarwal
• Information provided by (Responsible Party): Rajni Agarwal, Stanford University

Tracking Information

• First Submitted Date: June 10, 2014
• First Posted Date: June 12, 2014
• Last Update Posted Date: May 21, 2015
• Study Start Date: May 2014
• Estimated Primary Completion Date: December 2019 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: June 11, 2014): Incidence of severe (Grade III/IV) acute graft versus host disease [ Time Frame: Day 100 post transplant ]
• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Malignant Diseases
• Non-malignant Diseases

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

Study Arms

• Experimental: ARM A Malignant TBI
  Malignant diseases Conditioning including total body irradiation and chemotherapy
  Intervention: Device: CliniMACS CD34+ cell enrichment and T-cell depletion
• Experimental: ARM B Malignant Non-TBI
  Malignant diseases chemotherapy based conditioning
  Intervention: Device: CliniMACS CD34+ cell enrichment and T-cell depletion
• Experimental: ARM C Non-malignant
  Non-malignant diseases Chemotherapy based conditioning
  Intervention: Device: CliniMACS CD34+ cell enrichment and T-cell depletion

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: June 11, 2014): 30
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 2020
• Estimated Primary Completion Date: December 2019 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: up to 35 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02162511
• Other Study ID Numbers: 28663
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Rajni Agarwal, Stanford University
• Study Sponsor: Rajni Agarwal
• Collaborators: Not Provided

Investigators

• Principal Investigator: Rajni Agarwal, MD; Stanford University

• PRS Account: Stanford University
• Verification Date: May 2015