Skip to content
Triangle logo TriangleGet started
← Study library

NCT03792633 · CITED IN SOURCE DOCUMENTS

Study of huCART19 for Very High-Risk (VHR) Subsets of Pediatric B-ALL

An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.

Phase
PHASE2
Status at capture
COMPLETED
Registry last update
2025-06-05

This is a phase 2 study to evaluate humanized CD19 redirected autologous T cells (or huCART19 cells) with CD19 expressing relapsed and refractory B-cell acute lymphoblastic leukemia. This study is targeting pediatric and young adult patients aged 3 months - 29 years with CD19+ B cell malignancies in newly diagnosed B-ALL patients predicted to have an exceedingly poor outcome with conventional chemotherapy, in high-risk first relapse, or and in second or greater relapse in this phase 2 trial. In addition, a second cohort will test the efficacy of huCART19 in patients with poor response to prior B cell directed engineered cell therapy.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

→

baseline

Baseline characteristics

→

outcome · POSTED

Number of Subjects With Event-Free Survival in Patients With Newly Diagnosed VHR B-ALL or High-risk Relapse of B-ALL 1 Year After Treatment.

→

outcome · POSTED

Number of Subjects With Event-Free Survival in Patients With Poor Response to Prior B Cell Directed Engineered Cell Therapy 1 Year After Treatment.

→

outcome · POSTED

Overall Remission Rate

→

outcome · POSTED

Overall Remission Rate (Cohort B)

→

outcome · POSTED

Percentage of Subjects With Relapse-free Survival

→

adverse events

Adverse events

→

more info

Limitations, agreements, and source contact

→
Who could take part
eligibility Criteria
Inclusion Criteria: 1. Signed informed consent form must be obtained. 2. Relapsed or refractory B-cell ALL: a. Cohort A: Patients with newly diagnosed VHR B-ALL or high-risk relapse of B-ALL who meet one of the following criteria: i. Newly diagnosed NCI HR B-ALL with induction failure: M3 marrow (\>25% blasts) at end of induction OR ii. First marrow relapse of B-ALL at \< 36 months from diagnosis OR iii. 2nd or greater relapse OR iv. Any relapse after allogeneic HSCT and ≥ 4 months from SCT at enrollment OR v. Refractory disease defined as having not achieved an MRD-negative and/or CSF-negative CR after ≥ 2 chemotherapy regimens/cycles of frontline therapy or 1 cycle of reinduction therapy for patients in first relapse OR vi. Ineligible for allogeneic SCT because of: 1. Comorbid disease 2. Other contraindications to allogeneic SCT conditioning regimen 3. Lack of suitable donor 4. Prior SCT 5. Declines allogeneic SCT as the therapeutic option after documented discussion, with expected outcomes, about the role of SCT with a BMT physician not part of the study team b. Cohort B: Patients previously treated with B cell directed engineered cell therapy who meet one of the following criteria: i. partial response or no response to prior cell therapy ii. CD19+ relapse after prior cell therapy iii. demonstrated early (≤6 months from infusion) B cell recovery suggesting loss of engineered cells c. Patients with prior or current history of CNS3 disease will be eligible if CNS disease is responsive to therapy (at infusion, must meet criteria in Section 5.3 of the protocol) 3\. Documentation of CD19 tumor expression in bone marrow, peripheral blood, CSF, or tumor tissue by flow cytometry at relapse (or a recent sample in the case of refractory disease). If the patient has received CD19-directed therapy, then the flow cytometry should be obtained after this therapy to show CD19 expression. 4\. Adequate organ function defined as: 1. A serum creatinine based on age/gender 2. ALT≤ 500 U/L 3. Bilirubin ≤2.0 mg/dl 4. Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea, \< Grade 3 hypoxia; DLCO ≥ 40% (corrected for anemia) if PFTs are clinically appropriate as determined by the treating investigator 5. Left Ventricular Shortening Fraction (LVSF) ≥ 28% or Ejection Fraction (LVEF) ≥ 45% confirmed by ECHO, or adequate ventricular function documented by a scan or a cardiologist. 5\. Age 3 months to 29 years. 6. Adequate performance status (Lansky or Karnofsky score ≥50). 7. Subjects of reproductive potential must agree to use acceptable birth control methods. Exclusion Criteria: 1. Active hepatitis B or active hepatitis C. 2. HIV Infection. 3. Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy. 4. Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well. 5. CNS3 disease that is progressive on therapy, or with CNS parenchymal lesions that might increase the risk of CNS toxicity. 6. Pregnant or nursing (lactating) women. 7. Uncontrolled active infection. 8. Active medical disorder that, in the opinion of the investigator, would substantially increase the risk of uncontrollable CRS or neurotoxicity.
healthy Volunteers
false
maximum Age
29 Years
minimum Age
3 Months
sex
ALL
std Ages
  1. CHILD
  2. ADULT
Treatment arms and interventions
arm Groups
  1. intervention Names
    1. Biological: huCART19
    label
    Newly Diagnosed VHR B-ALL or High-Risk Relapse of B
    type
    EXPERIMENTAL
  2. intervention Names
    1. Biological: huCART19
    label
    Poor Response to Prior B Cell Directed Engineered cell therapy
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Newly Diagnosed VHR B-ALL or High-Risk Relapse of B
    2. Poor Response to Prior B Cell Directed Engineered cell therapy
    description
    IV injection
    name
    huCART19
    type
    BIOLOGICAL
Study design
allocation
NON_RANDOMIZED
intervention Model
PARALLEL
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
106
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. description
    Time from infusion to the first of event or censoring Events = Relapse, No Response, or Death; Censoring =Initiation of New Anticancer Therapy, Last Day of Follow-up/EOS; whichever occurs first.
    measure
    Number of Subjects With Event-Free Survival in Patients With Newly Diagnosed VHR B-ALL or High-risk Relapse of B-ALL 1 Year After Treatment.
    time Frame
    1 year
  2. description
    Time from infusion to the first of event or censoring Events = Relapse, No Response (including CR/CRi without B Cell Aplasia), or Death; Censoring =Initiation of New Anticancer Therapy, Last Day of Follow-up/EOS; whichever occurs first.
    measure
    Number of Subjects With Event-Free Survival in Patients With Poor Response to Prior B Cell Directed Engineered Cell Therapy 1 Year After Treatment.
    time Frame
    1 year
secondary Outcomes
  1. description
    Overall remission rate as determined by the response at day 28, computed as the number of subjects with CR or Cri, in patients with newly diagnosed VHR B-ALL or high-risk relapse of B-ALL.
    measure
    Overall Remission Rate
    time Frame
    28 Days
  2. description
    Number of subjects with CR/CRi with B Cell Aplasia at Day 28
    measure
    Overall Remission Rate (Cohort B)
    time Frame
    28 Days
  3. description
    Time from first response of CR/CRi to the first of event or censoring, in responders. Cohort A: Events = Relapse or Death; Censoring = Initiation of New Anticancer Therapy, Last Day of Follow-up/EOS; whichever occurs first. Cohort B: When defining responders, CR/CRi without B Cell Aplasia is considered no response. Events = Relapse or Death; Censoring = Initiation of New Anticancer Therapy, Last Day of Follow-up/EOS; whichever occurs first.
    measure
    Percentage of Subjects With Relapse-free Survival
    time Frame
    1 year
Full study description
brief Summary
This is a phase 2 study to evaluate humanized CD19 redirected autologous T cells (or huCART19 cells) with CD19 expressing relapsed and refractory B-cell acute lymphoblastic leukemia. This study is targeting pediatric and young adult patients aged 3 months - 29 years with CD19+ B cell malignancies in newly diagnosed B-ALL patients predicted to have an exceedingly poor outcome with conventional chemotherapy, in high-risk first relapse, or and in second or greater relapse in this phase 2 trial. In addition, a second cohort will test the efficacy of huCART19 in patients with poor response to prior B cell directed engineered cell therapy.
Source references
Source notices and limitations
    Discovery and provenance
    1. release id
      ctgov-registry-7ad944f6deaf1f5cd6122126
      edge id
      nct-edge-b5834a11f03a2a2ad960
      requested nct id
      NCT03792633
      primary nct id
      NCT03792633
      source kind
      nci_explicit_reference_or_link
      source record id
      Source null
      source file sha256
      8416f6a719c496873bd76f9837e64f45de187c289bc5ec0bf994c3e18f233983
      payload sha256
      85229a3795d9a793182bb75a31bac38ca602d4e3b9f7595a8892c5cb978fe3ed
      payload
      clinical indication matching status
      not_inferred
      end exclusive
      true
      id
      nct-edge-b5834a11f03a2a2ad960
      link basis
      explicit_NCT_identifier_in_acquired_source
      nct id
      NCT03792633
      occurrences
      1. context exact
        .01% are classified as very high risk and are eligible for a chimeric antigen receptor (CAR) T-cell clinical trial in first remission (<a href="/clinicaltrials/NCT03792633">NCT03792633</a>).</p><p id="_2188" tabindex="-1">Patients with B-ALL and Down syndrome are classified into risk groups similar to other children, but Down syn
        end
        420414
        exact text
        NCT03792633
        start
        420403
      2. context exact
        sified as very high risk and are eligible for a chimeric antigen receptor (CAR) T-cell clinical trial in first remission (<a href="/clinicaltrials/NCT03792633">NCT03792633</a>).</p><p id="_2188" tabindex="-1">Patients with B-ALL and Down syndrome are classified into risk groups similar to other children, but Down syndrome patient
        end
        420427
        exact text
        NCT03792633
        start
        420416
      offset unit
      unicode_code_points
      source file
      /tmp/cancer-full-research/nci/raw/346ccc02fe964bb078.html
      source file sha256
      8416f6a719c496873bd76f9837e64f45de187c289bc5ec0bf994c3e18f233983
      source json pointer
      Source null
      source kind
      nci_explicit_reference_or_link
      source record id
      Source null
      source retrieved at
      2026-09-09T23:21:53.377184+00:00
      source string basis
      UTF-8_text_with_universal_newline_translation
      source string sha256
      d68ab4be374e916e0de9c7632347ccef2167b36c83a023ce62cc9bb0575973b9
      source title
      Childhood Acute Lymphoblastic Leukemia Treatment (PDQ®)–Health Professional Version
      source update dates
      1. context
        Updated: April 21, 2025
        datetime
        2025-04-21T12:00:00Z
        display
        April 21, 2025

    Preserved source evidence · Not medical advice