NCT04276870 Orphan Indications for CD19 Redirected Autologous T Cells
| NCT ID | NCT04276870 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Stephan Grupp MD PhD |
| Condition | Pediatric and Young Adult Patientswith Hypodiploid or t(17;19) B-ALL |
| Study Type | INTERVENTIONAL |
| Enrollment | 133 participants |
| Start Date | 2020-03-12 |
| Primary Completion | 2028-03-10 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 133 participants in total. It began in 2020-03-12 with a primary completion date of 2028-03-10.
⚠ 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
This is an open-label, four-cohort, phase 2 study to determine the efficacy of CART19 in pediatric and young adult patientswith hypodiploid (Cohort A) or t(17;19) B-ALL (Cohort B), infants with very high risk KMT2A B-ALL (Cohort C), and in patients with central nervous system (CNS) relapse who did not receive cranial radiation (XRT) or bone marrow transplantation (BMT) (Cohort D).
Eligibility Criteria
Inclusion Criteria: 1. Signed informed consent form must be obtained prior to any study procedure. 2. Male and female patients with documented CD19+ B-ALL a.Cohort A \& B: Patients, regardless their response to initial or relapsed B ALL therapy, with the following characteristics: i.Cohort A: Subjects with confirmation of a hypodiploid karyotype (chromosome number fewer than 45) ii.Cohort B: Subjects with cytogenetic confirmation of the chromosomal translocation t(17;19) (Cohort B) b.Cohort C: Infants w/ newly diagnosed KMT2A rearranged B-ALL classified as very high risk by the following criteria: i.Age \< 3 months at diagnosis ii.Age \< 6 months and WBC \> 300,000x109/L at diagnosis or a poor prednisone response in induction iii.MRD positive \> 0.01 (or PCR \> 104) after 2 courses of standard infant ALL therapy. c.Cohort D: Subjects in a first or greater CNS relapse, prior to therapy with cranial XRT or HSCT for the current relapse 3. Documentation of CD19 tumor expression in bone marrow, peripheral blood, CSF, or tumor tissue. 4. Age 0 to 29 years 5. Adequate organ function defined as: 1. A serum creatinine based on age/gender as follows: Maximum Serum Creatinine (mg/dL) Age Male Female 0 to \< 2 years 0.6 0.6 2 to \< 6 years 0.8 0.8 6 to \< 10 years 1.0 1.0 10 to \< 13 years 1.2 1.2 13 to \< 16 years 1.5 1.4 ≥ 16 years 1.7 1.4 2. Adequate liver function: i.ALT≤ 5 x ULN; ALT ii.Total bilirubin ≤ 3 x ULN iii.ALT and/or bilirubin results that exceed this range are acceptable if, in the opinion of the physician-investigator (or as confirmed by liver biopsy), the abnormalities are directly related to ALL infiltration of the liver. c.Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and \< Grade 3 hypoxia; DLCO ≥ 40% (corrected for anemia) if PFTs are clinically appropriate as determined by the physician-investigator. d.Left Ventricular Shortening Fraction (LVSF) ≥ 28%, or Left Ventricular Ejection Fraction (LVEF) ≥ 45% by echocardiogram. In cases where quanitative assessment of LVSF/LVEF is not possible, a statement by the cardiologist that the ECHO shows qualititatively normal ventricular function wll suffice. 6. Adequate performance status defined as Lansky or Karnofsky score ≥ 50 7. Subjects of reproductive potential must agree to use acceptable birth control methods Exclusion Criteria: 1. For subjects with a CNS relapse, prior cranial XRT or BMT for the current relapse is an exclusion. 2. Active hepatitis B or active hepatitis C. 3. HIV Infection. 4. Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy. 5. Concurrent use of systemic steroids 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 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. 6. CNS3 disease that is progressive on therapy, or with CNS parenchymal lesions that might increase the risk of CNS toxicity. 7. Pregnant or nursing (lactating) women. 8. Uncontrolled active infection.
Contact & Investigator
Stephan Grupp, MD, PhD
STUDY DIRECTOR
Children's Hospital of Philadelphia
Frequently Asked Questions
Who can join the NCT04276870 clinical trial?
This trial is open to participants of all sexes, aged 0 Years or older, up to 29 Years, studying Pediatric and Young Adult Patientswith Hypodiploid or t(17;19) B-ALL. 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 NCT04276870 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT04276870 currently recruiting?
Yes, NCT04276870 is actively recruiting participants. Contact the research team at CARTNurseNavigator@chop.edu for enrollment information.
Where is the NCT04276870 trial being conducted?
This trial is being conducted at Philadelphia, United States.
Who is sponsoring the NCT04276870 clinical trial?
NCT04276870 is sponsored by Stephan Grupp MD PhD. The principal investigator is Stephan Grupp, MD, PhD at Children's Hospital of Philadelphia. The trial plans to enroll 133 participants.