RECRUITING

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

Description

The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia IRB approved protocols that utilize CliniMACs technology for T depletion.

Conditions

LeukemiaInborn Errors of MetabolismBone Marrow Failure SyndromesImmunodeficienciesImmunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome
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Related Conditions:

LeukemiaInborn Errors of MetabolismBone Marrow Failure SyndromesImmunodeficienciesImmunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome

Study Overview

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Study Details

Study overview

The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia IRB approved protocols that utilize CliniMACs technology for T depletion.

Expanded Access Protocol Using CD3+/CD19+ Depleted Unrelated Donor or Related Donor Peripheral Stem Cells

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

Condition
Leukemia
Intervention / Treatment

-

Contacts and Locations

Philadelphia

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. Patients who lack a fully HLA matched sibling and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT) but are not deemed suitable candidates per their treating clinical team for current open institutional protocols using ClinMACs device for CD3+/CD19+ depletion.
  • 2. Patients with the following transplantable diseases:
  • 3. Signed informed consent
  • 4. Lansky or Karnofsky performance ≥60
  • 5. Hematologic and Organ Function per current institutional SOP.
  • 6. Infectious Evaluation as per current institutional SOP.
  • 7. Participants of childbearing potential must have a negative pregnancy test as per institutional SOP
  • 8. In cases that are deemed clinical emergencies (primary or secondary graft failure, severe marrow suppression), the above status criteria will be waived.
  • 9. Patients must have an identified living donor
  • * Donor selection will comply with 21 CFR 1271
  • * Unrelated donor that meets the matching criteria of the NMDP with allele matching at HLA -A, -B, -C, -DRB1, and -DQB1: Unrelated donors may be a 10/10 match, a 9/10 match, or an 8/10 match if one of the mismatches is at DQB1
  • * Related donor suitable for mobilization infectious disease criteria as per SOP, including HIV, HepB, HepC PCR negative.
  • * CHOP BMT procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. Our donor collection program is FACT accredited.
  • * Unrelated donor identified through the National Marrow Donor Program (NMDP) and fulfills the NMDP criteria for donation. Unrelated donor willing and able to undergo mobilization of peripheral stem cells and apheresis.
  • * The donors selected for this IND will either be unrelated donors identified through the National Marrow Donor Program (NMDP) or related donors. Regarding the unrelated donors; NMDP procedures for determining donor eligibility include donor screening and testing for relevant communicable disease agents and diseases
  • 1. Uncontrolled bacterial, viral or fungal infections
  • 2. Fully HLA matched sibling donor
  • 3. Donor unable to donate peripheral stem cells
  • 4. Pregnant participants

Ages Eligible for Study

to 30 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Children's Hospital of Philadelphia,

Timothy Olson, MD, PhD, PRINCIPAL_INVESTIGATOR, Children's Hospital of Philadelphia

Study Record Dates

2027-01