UCD19 CarT in Treatment of Pediatric B-ALL and B-NHL
Phase I/II Dose Escalation and Preliminary Efficacy of CD19 Directed CAR-T Cells Generated Using the Miltenyi CliniMACs Prodigy System (UCD19 CAR-T) in Pediatric Patients With Relapsed and/or Refractory B-Cell Acute Lymphoblastic Leukemia (B-ALL) and B-Cell Non-Hodgkin Lymphoma (B-NHL)
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 31 Days to 30 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests a new CAR-T treatment made locally. CAR-T uses immune cells to target cancer cells with a marker called CD19. It is for children and young adults with certain B-cell blood cancers: acute lymphoblastic leukemia (B-ALL) or non-Hodgkin lymphoma (B-NHL).
This phase I/II trial will investigate a new CD19 directed CAR-T therapy manufactured locally with the goals to expedite infusion to wider patient inclusion that includes those who were previously excluded, such as pediatric patients with B-cell NHL and patients in primary relapse.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be 31 days to 30 years old and have B-ALL or B-NHL with the CD19 marker.
- For group one: cancer has returned at least twice, returned after a donor bone marrow transplant, or not responded to standard treatment. People who qualify for a transplant but cannot have one or decline it may also qualify.
- For group two: B-ALL has returned for the first time, with certain high-risk gene changes, return only in the brain or spinal cord area that calls for radiation, Down syndrome, cancer cells left after repeat starting chemotherapy, or age 18 or older. Newly diagnosed B-ALL with small amounts of cancer left after the consolidation phase of chemotherapy may also qualify.
- Be able to have leukapheresis, a procedure that collects white blood cells, or have suitable cells already collected and stored.
- Have a score of at least 50% on a scale of ability to do daily activities. Be unable to get, or have declined, a CD19 CAR-T treatment already on the market.
- Give signed consent through the patient, parent, guardian, or legal representative, as appropriate. Children older than 7 may also be asked to agree.
- Agree to study procedures, visits, and long-term follow-up.
- Not be pregnant or breastfeeding. If able to become pregnant or father a child, agree to highly effective birth control from the first CAR-T treatment until 12 months after the last treatment.
Who can't join
- Cancer is growing quickly, with no suitable treatment to control it while waiting for CAR-T.
- Have active graft-versus-host disease, when donor cells attack the body.
- Have an uncontrolled, life-threatening infection that makes study treatment unsafe.
- Have serious heart problems, low blood oxygen, or poor kidney function based on study tests.
- Have very high liver blood test results, unless these are thought to be caused by the cancer.
- Are pregnant, plan to become pregnant, or will not use birth control, including not having sex, during the study.
- Have HIV, active hepatitis B, or active hepatitis C.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: * Meets clinical criteria for leukapheresis or has a leukapheresis product previously collected and stored per recommended guidelines. * Provision of signed and dated consent form from parent or guardian (patients \<18), the patient themselves (\>18), or legally authorized representative (patient \>18 who lack decision-making capacity); Pediatric patients will be included in age-appropriate discussions and assent will be obtained for those \> 7 years of age, when appropriate, according to institutional standards. * Willingness to participate in long term follow up study. * Stated willingness to comply with all study procedures and be available for the duration of the study. * Males OR non-pregnant, non-breastfeeding females. o Patients of child-bearing potential or capable of fathering a child must agree to use highly effective contraception from the time of initial CAR T cell administration though 12 months following the final administration of investigational product. * Aged 31 days to 30 years (inclusive) at time of consent and enrollment. * Acute Lymphoblastic Leukemia (ALL) OR Non-Hodgkin Lymphoma (NHL) of B-cell origin that: * Has confirmed expression of CD19 by flow cytometry, immunohistochemistry (IHC), or both. Cohort One Criteria: * Meets any one of the following conditions: * Relapsed two or more times * Relapsed at any time after allogeneic BMT * Refractory to standard therapy as determined by the treating physician * Meets criteria for BMT but is ineligible as determined by the treating physician Patient and/or parents declining BMT options and would prefer CAR-T Therapy. * Non-Hodgkin Lymphoma includes all of the following: * Diffuse large B-cell lymphoma (DLBCL) * Burkitt Lymphoma * Intermediate lymphoma between Burkitt and DLBCL * Primary Mediastinal B-cell Lymphoma (PMBL) * Follicular lymphoma * High grade B cell lymphoma * Transformed lymphoma Cohort Two Criteria: * B-ALL in first relapse with any one of the following conditions: * High-risk genomic alterations at initial diagnosis such as KMT2A gene rearrangement, t(17;19), hypodiploidy, Ph-like mutations, BCR-ABL1 fusion (Ph+ ALL), iAMP21, and TP53 inactivating mutation/deletion. * Isolated CNS relapse such that cranial radiation would be indicated as a component of standard salvage therapy. * Down syndrome. * Minimal residual disease (MRD) positivity of \> 0.01% by FACS or \> 0 clonal sequences by NGS in bone marrow post re-induction chemotherapy. * Age 18 years or older. OR Newly diagnosed with persistent MRD ≥ 0.01% by flow cytometry in bone marrow at end of consolidation. * Performance score (Lansky or Karnofsky) of 50% or better; * Unable to or declined to receive commercially available CD19 CAR-T Therapy. Exclusion Criteria: * Evidence of rapidly progressive disease without adequate salvage/bridging regimens as determined by the investigator. * Active Graft-versus-Host Disease (GvHD). * Active, uncontrolled, life-threatening infection that at the determination of the treating physician would preclude safe leukapheresis or tolerance of LD chemotherapy, cell infusion, or cytokine release syndrome. * Evidence of severe organ dysfunction as defined by: * Myocardial dysfunction: Ejection fraction ≤ 40% or shortening fraction ≤ 28%, evidence of physiologically significant pericardial effusion as determined by an echocardiogram (ECHO), and clinically significant electrocardiogram (ECG) findings. * Baseline oxygen saturation of ≤ 90% on room air * Transaminases \> 10x upper limit of normal (ULN) or bilirubin \>2x the ULN, unless thought to be related to primary disease * Estimated Cr clearance \<60 mL/min/1.73 m2 (if nuclear medicine GFR or other more specific testing exceeds this level than it can supersede the estimated clearance) * Post-pubertal females that are pregnant, planning to become pregnant, or unwilling to use birth control (includes abstinence) for the study duration. * Known HIV infection, or active Hepatitis B or active Hepatitis C infection.
Study sites (1)
CO
- Children's Hospital Colorado · Aurora, CO 80045Vanessa Fabrizio, MD, MS · Study contact · 720-777-6860 · BMT@childrenscolorado.orgVanessa Fabrizio, MD, MS · Principal investigatorMore trials in Aurora