Fact CheckerSign in

NCT02356653RecruitingEarly phase 1

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

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

Source: ClinicalTrials.gov · Last updated 20 Feb 2026

Brief summary (registry, in English)

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.

Status (registry)
Recruiting
Phase (registry)
Early phase 1
Study type
Interventional
Enrollment
100 (estimated)
First submitted
22 Oct 2014
Start date
Dec 2013
Primary completion date
Jan 2027
Lead sponsor
Children's Hospital of Philadelphia
NCT number
NCT02356653

What you commit to

  • Study type: Interventional · Early phase 1
  • Planned participants: 100
  • From Dec 2013 to the primary completion date, Jan 2027
  • 1 site in 1 country
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Other illness

  • Uncontrolled bacterial, viral or fungal infections

Other

  • Suitable, fully HLA matched sibling donor
  • Donor unable to donate peripheral stem cells
  • Pregnant participants

Who the study is looking for

  • 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

Age in the registry: - to 30 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (1)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
Children's Hospital of Philadelphia
Coordinator of this site? Confirm its status		PhiladelphiaUnited StatesRecruiting

This page is information, not medical advice. Discuss any trial with your doctor.