Menu
Recruiting NCT02356653

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

Early Phase I Interventional Leukemia Inborn Errors of Metabolism Bone Marrow Failure Syndromes Immunodeficiencies

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs).
Who it may be relevant to
Registry conditions: Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes, Immunodeficiencies. Basic parameters: up to 30 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

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.

Detailed description

Only 25-30% of patients who may benefit from HSCT have a matched related donor. There is a higher rate of complications using cells from an unrelated or partially matched related donor. T cells within the donor cells may cause a complication called graft vs. host disease (GVHD). The goal of this study is to use the CliniMACs device to remove the T cells that cause GVHD, called T cell depletion.

Interventions

  • Biological Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
    Processing of stem cells using the CliniMACs device to selectively deplete specific T cells to decrease risk of graft versus host disease when using donor stem cells which are not fully matched.

Primary outcome measures

  • Overall Survival [Time frame: 1 year post transplant]
Secondary outcome measures (2)
  • Graft versus Host Disease [Time frame: 1 year post transplant]
  • Graft Failure [Time frame: 1 year post transplant]

Eligibility criteria

Inclusion criteria

  • 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.
  • Patients with the following transplantable diseases:

Non-malignant diseases:

Metabolic storage diseases correctable by HSCT, Bone marrow failure syndromes, Immunodeficiencies/immune dysregulation syndromes/including HLH, Hemoglobinopathies correctable and requiring HSCT, and Other diseases treated with HSCT/Other non-malignant blood, metabolic, or immune disorders for which HSCT has been recommended

Malignant diseases:

Acute leukemias, Chronic leukemias, Lymphomas, Myelodyplastic syndrome

  • Signed informed consent
  • Lansky or Karnofsky performance ≥60
  • Hematologic and Organ Function per current institutional SOP.
  • Infectious Evaluation as per current institutional SOP.
  • Participants of childbearing potential must have a negative pregnancy test as per institutional SOP
  • In cases that are deemed clinical emergencies (primary or secondary graft failure, severe marrow suppression), the above status criteria will be waived.
  • 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

Exclusion criteria

  • Uncontrolled bacterial, viral or fungal infections
  • Suitable, fully HLA matched sibling donor
  • Donor unable to donate peripheral stem cells
  • Pregnant participants

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 1 center
  • Children's Hospital of Philadelphia — Philadelphia

Identifiers

NCT: NCT02356653 · 13-010286 · 12BT125

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗