Active — Not RecruitingPhase 1

A Pediatric Trial of Genetically Modified Autologous T Cells Directed Against CD19 for Relapsed CD19+ Acute Lymphoblastic Leukemia

United States6 participantsStarted 2012-03-25

Plain-language summary

Patients with relapsed leukemia often develop resistance to chemotherapy. For this reason, we are attempting to use a patient's own T cells, which can be genetically modified to expresses a chimeric antigen receptor(CAR). The CAR enables the T cell to recognize and kill the leukemic cells though the recognition of CD19, a protein expressed on the surface of the majority of pediatric ALL. This is a phase I study designed to determine the maximum tolerated dose of the CAR+ T cells and define the toxicity of the treatment. As a secondary aim, we will be looking at the efficacy of the T cells on eradicating the patient's leukemic cells.

Who can participate

Age range

1 Year – 26 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * CD19+ Leukemia in 1st marrow relapse with MRD at the end of 1st month of re-induction * CD19+ Leukemia in 2nd or greater relapse * CD19+ Leukemia with indication for HCT, but has contraindication * Age between 1 and 26 years of age * Karnofsky of \>50 or Lansky \>50 * Life Expectancy \>12 weeks * Able to tolerate a blood draw of 4-6mL/kg * Recovered from acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy * absolute lymphocyte count of \>/=750 cell/mm3 or \>/=500 is \>20kg * creatinine clearance or radioisotope GFR \>/= 70mL/min/1.73m2 OR normal serum creatinine based on age/gender * total bilirubin \</= 1.5x upper limit normal OR direct bilirubin \</= 1.5mg/dl * ALT \</= 3x upper limit normal * corrected QTc \<450msec of ECG * Shortening Fraction \>28% by ECHO or Ejection Fraction \>50% by MUGA * Documented negative HIV, Hep B and Hep C * Agree to long-term follow up for up to 15 years if they receive T cell infusion Exclusion Criteria: * Philadelphia Positive Leukemia * Prior Allogeneic Stem Cell Transplant * CNS 2 or 3 * prior cellular immunotherapy with chimeric antigen receptor modified T cells * fully humanized antibodies within three half lives * systemic corticosteroids within 7 days of enrollment * requires supplemental oxygen or has a chest X-ray with an infectious process * CNS pathology (seizure disorder, paresis, aphasia, cerebrovascular ischemia/hemorrhage, severe brain injuries, dementia, cerebellar disease, or…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Since this is a Phase 1 trial primarily focused on measuring adverse events and safety rather than proving that the treatment works, what kinds of side effects have been seen so far in the children who have already received these genetically modified T cells, and how were those side effects managed?
2This trial targets CD19 on leukemia cells using the child's own genetically modified T cells — would my child's leukemia cells need to be confirmed as CD19-positive before we could even consider this, and how is that tested?
3Since the trial is no longer actively recruiting, what would it actually take for my child to be considered for enrollment, and is there any realistic path in?
4Given that this is a relapsed ALL trial, how does this CAR-T cell approach compare to other options available right now for a child whose leukemia has come back, including standard salvage chemotherapy or other approved CAR-T therapies?
5Because this is a Phase 1 study, we know more about risks than we do about long-term benefit — based on what's been observed so far, do you think the potential benefit outweighs the unknowns for my child's specific situation?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Participant with Adverse Events

Timeframe: 42 days

Trial details

NCT IDNCT01683279

SponsorSeattle Children's Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2015-01-07

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