NCT02356653 Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC
| NCT ID | NCT02356653 |
| Status | Recruiting |
| Phase | EARLY_Phase 1 |
| Sponsor | Children's Hospital of Philadelphia |
| Condition | Leukemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 100 participants |
| Start Date | 2013-12 |
| Primary Completion | 2027-01 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 100 participants in total. It began in 2013-12 with a primary completion date of 2027-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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
Inclusion 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: 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 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 Exclusion Criteria: 1. Uncontrolled bacterial, viral or fungal infections 2. Suitable, fully HLA matched sibling donor 3. Donor unable to donate peripheral stem cells 4. Pregnant participants
Contact & Investigator
Timothy Olson, MD, PhD
PRINCIPAL INVESTIGATOR
Children's Hospital of Philadelphia
Frequently Asked Questions
Who can join the NCT02356653 clinical trial?
This trial is open to participants of all sexes, up to 30 Years, studying Leukemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT02356653 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT02356653 currently recruiting?
Yes, NCT02356653 is actively recruiting participants. Contact the research team at cttsbmtintake@chop.edu for enrollment information.
Where is the NCT02356653 trial being conducted?
This trial is being conducted at Philadelphia, United States.
Who is sponsoring the NCT02356653 clinical trial?
NCT02356653 is sponsored by Children's Hospital of Philadelphia. The principal investigator is Timothy Olson, MD, PhD at Children's Hospital of Philadelphia. The trial plans to enroll 100 participants.
Related Trials
Related Intelligence Guides
In-depth guides covering this condition's trials, eligibility, and what to expect.