New Dual-Target CAR T-Cell therapy aims to beat relapsed childhood cancers
NCT ID NCT05442515
First seen Jun 27, 2026 · Last updated Jul 31, 2026 · Updated 6 times
Summary
This study tests a new type of CAR T-cell therapy that targets two proteins, CD19 and CD22, on cancer cells. It is for children and young adults aged 3 to 39 with B-cell cancers like leukemia or lymphoma that have not been cured by standard treatments. Participants receive their own genetically modified immune cells after chemotherapy. The goal is to see if this therapy is safe and effective.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- CD19/CD22 CAR T-cells
- What this could lead to
- If successful, this could offer a new treatment option for children and young adults with B-cell cancers that have come back after standard therapy.
- What could go wrong
- This is an early-phase trial, so the treatment may not work for everyone. There are risks of serious side effects like cytokine release syndrome and nervous system problems.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 130 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Dec 2022
- Expected to finish
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Jul 2029
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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3 to 39 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
* INCLUSION CRITERIA: * Diagnosis * Participant must: * Have pathology confirmed B cell ALL (not isolated to the testis or CNS), CML with ALL transformation, or high-grade lymphoma (e.g., Burkitt's lymphoma, B-lymphoblastic lymphoma, diffuse large B-cell lymphoma, inclusive of low-grade lymphoma that has transformed to high grade disease); and * Have relapsed or been refractory after at least one standard chemotherapy regimen and at least one salvage treatment. Participants with Philadelphia chromosome + ALL must have failed prior tyrosine kinase inhibitor; and * Be ineligible for allogeneic stem cell transplant (SCT), have refused SCT, or have recurred after SCT; and * Be unable to access (in a timely manner), ineligible for, or have relapsed/failed after or not responded to a commercially available CD19 CAR T-cell construct; and * Have evidence of at least minimal residual disease or PET-avid disease (lymphoma) at the time of enrollment. * CD22/CD19 expression * Cohorts A1b, B1b, C2b * CD19 must be detected on \>15% of the malignant cells by immunohistochemistry or \> 80% by flow cytometry. * CD22 positivity must be confirmed. * Cohorts D1b, 2 B-ALL * CD19 or CD22 positivity must be confirmed * Age \>= 3 years of age and \<=39 years of age at time of enrollment. * Clinical Performance status: Participants \>= 16 years of age: Karnofsky \>= 50%; Participants \< 16 years of age: Lansky scale \>= 50%. * Participants must have adequate organ and marrow function as defined below: * leukocytes \>= 750/mcL\* * platelets \>= 50,000/mcL\* * total bilirubin \<=2 X ULN (except in the case of participants with documented Gilbert's disease \> 3x ULN) * AST(SGOT)/ALT(SGPT) \<=10 X institutional upper limit of normal * creatinine \<= the maximum for age listed in the table below OR * measured creatinine clearance \>=60 mL/min/1.73 m\^2 for participants with creatinine levels above the max listed below per age. * Age (Years) \<= 5 / Maximum Serum Creatinine (mg/dL) \<= 0.8 * Age (Years) 6 to \<= 10 / Maximum Serum Creatinine (mg/dL) \<= 1.0 * Age (Years) \>10 / Maximum Serum Creatinine (mg/dL) \<= 1.2 * a participant will not be excluded because of pancytopenia \>= Grade 3 if it is due to underlying bone marrow involvement by leukemia * Central nervous system (CNS) Status * Participants with leukemia with CNS 1 and 2 disease are eligible in the absence of exclusion criteria * Participants of child-bearing or child-fathering potential must be willing to practice effective birth control from the time of enrollment until 12 months following completion of study treatment for women and for 4 months following completion of study treatment for men. * Participants who are breastfeeding or plan to breastfeed must agree to discontinue/postpone breastfeeding while on study therapy and until 1 month after the administration of CAR. * Cardiac function: Left ventricular ejection fraction \>= 45% or fractional shortening \>=28% * Pulmonary Function * Baseline oxygen saturation \>92% on room air at rest * Ability of participant or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document. * Ability and willingness of participant or Legally Authorized Representative (LAR) to co- enroll on 15-C-0028: Follow-up Evaluation for Gene-Therapy Related Delayed Adverse Events after Participation in Pediatric Oncology Branch Clinical Trials. EXCLUSION CRITERIA: Participants meeting any of the following criteria are not eligible for participation in the study: * Participants with CNS3 disease, progressing neurologic signs\* of CNS disease, radiologically detected active CNS lymphoma (\*resolving manifestation or persistent and/or irreversible findings from prior CNS involvement (e.g., blindness) is not exclusionary) * Hyperleukocytosis (\>= 50,000 blasts/microL) * Positive serum or urine beta-HCG pregnancy test performed at screening. * Participants will be excluded based on prior therapy if they fail to meet following washout criteria: * Therapy: Systemic Chemotherapy, anti-neoplastic agents, antibody- based therapies * Washout\*: \>=2 weeks * Exceptions: 6 weeks for clofarabine or nitrosoureas; No washout for prior intrathecal chemotherapy, steroid therapy, hydroxyurea (no dose increases within prior 2 weeks) or ALL maintenance-type chemotherapy (vincristine, 6-mercaptopurine, oral methotrexate, or a tyrosine kinase inhibitor for participants with Ph+ ALL) provided there is recovery from any acute toxic effects * Therapy: Radiation * Washout\*: \>=3 weeks * Exceptions: No time restriction with radiation therapy if the volume of bone marrow treated is less than 10% and the participant has measurable/evaluable disease outside the radiation window * Therapy: Allogeneic Stem Cell Transplant * Washout\*: \>= 100 days since SCT; \>= 30 days since completion of immunosuppression; \>= 6 weeks since donor lymphocyte infusion (DLI) * Exceptions: Cannot have evidence of active graft-versus-host disease (GVHD) requiring systemic immunosuppression * Therapy: CAR T-Cell Therapy or other Adoptive Cell Therapy * Washout\*: \> 30 days post infusion * Washout: Time between therapy and apheresis * Positive HIV antibodies consistent with active HIV. * Positive hepatitis C antibodies or positive Hepatitis B surface antigen (HbsAG) indicative of current/active HCV/HBV. * Active second malignancy other than in situ carcinoma of the cervix, unless the tumor was treated with curative intent at least two years previously and participant is in remission. * History of severe, immediate hypersensitivity reaction attributed to compounds of similar chemical or biologic composition to any agents used in study or in the manufacturing of the cells. * Uncontrolled, symptomatic, intercurrent illness or social situations that would limit compliance with study requirements or in the opinion of the PI would pose an unacceptable risk to the participant.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
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- Tweaking donor cells may shield older transplant patients from a dangerous complication
- The immune cells that could make stem cell transplants work better
- New drug BL-M08D1 joins standard therapy in fight against aggressive lymphoma