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Completed

NCT Number: NCT02162511

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

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.

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Key information

Age range

Up to 35 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Stanford Children's Hospital

Palo Alto, California, 94305, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

CliniMACS CD34+ cell enrichment and T-cell depletion

Device

Primary outcomes

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

    Time frame: Day +100

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

Secondary outcomes

  1. Number of Participants With Graft Failure

    Time frame: Up to Day +42 after stem cell transplant

    Failure of donor stem cells to make neutrophils

  2. Length of Time to Engraftment

    Time frame: up to +1 year post-transplant

    Absolute neutrophil count (ANC) >500 for 3 consecutive days and >80% donor cells in blood.

  3. Chimerism of Donor Cells

    Time frame: Day +100 post-transplant

    The percentage of donor cells for all evaluable (without disease progression) patients

  4. Immune Recovery (CD4)

    Time frame: up to +1 year post-transplant

    The time to CD4 count >100

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

    Time frame: up to +1 year post-transplant

  6. Immune Recovery Shown as Phytohemagglutin (PHA)

    Time frame: 6 months and 1 year post-transplant

    Immune recovery defined as achieving normal levels of PHA (53,000-200,000 CPM)

  7. Number of Patients With Post-transplant Lymphoproliferative Disease (PTLD)

    Time frame: up to +1 year post-transplant

    Post-transplant lymphoproliferative disorder (PTLD) is a well-known, life-threatening complication of organ transplantation, predominantly occurring after solid organ transplantation (SOT) and hematopoietic stem cell transplantation (HSCT).

  8. Number of Patients With Severe Toxicities

    Time frame: up to +1 year post-transplant

    Incidence of transplant-related toxicities

  9. Number of Participants Experiencing Post-transplant Infections

    Time frame: up to +1 year post-transplant

    Post-transplant infections will be described by incidence and type. Participants may have had more than one type of infection.

  10. Transplant-related Mortality (TRM)

    Time frame: at Day +100 and +1 year post-transplant

    Death related to transplant

Sponsors and collaborators

Lead sponsor

Rajni Agarwal

Other

Registry information

Official study 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

Important dates

Study start
2014
Primary completion
2019
Study completion
2023
First posted
Jun 12, 2014
Registry last updated
Jun 26, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.