Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
AgeUp to 30
SponsorChildren's Hospital of Philadelphia

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Signed informed consent

Lansky or Karnofsky performance ≥60

Hematologic and Organ Function per current institutional SOP.

Disqualifiers

Uncontrolled bacterial, viral or fungal infections

Suitable, fully HLA matched sibling donor

Donor unable to donate peripheral stem cells

Pregnant participants

Trial design

Design model

Single group

Treatments tested in this trial

  • Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)

    Biological/Vaccine

    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.

Treatment groups

100 Participants
are divided into 1 treatment group
Group A: Expanded access to CliniMACs device for T cell depletionExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Overall Survival

Number of participants who remain alive.

Time frame
1 year post transplant

Secondary outcomes

1

Graft versus Host Disease

Number of participants who had grades II-IV acute graft versus host disease and/or limited or extensive chronic gvhd

Time frame
1 year post transplant
2

Graft Failure

Number of participants who experienced primary or secondary graft failure or autologous reconstitution.

Time frame
1 year post transplant

Other outcomes

Sponsors and contacts

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