Clinical trial NCT04571138: A Pediatric and Young Adult Trial of Genetically Modified T Cells Directed Against CD22 for Relapsed/Refractory Leukemia or Lymphoma
Official title: Pediatric and Young Adult Leukemia Adoptive Therapy (PLAT)-07: A Phase 1/2 Study of CD22-Specific CAR T Cells for CD22+ Leukemia or Lymphoma
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Leukemia
Lymphoma
Lead sponsor: Seattle Children's Hospital
Sponsor class: OTHER
First posted: 2020-09-30
Enrollment: 42
Sex: ALL
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Patients with relapsed or refractory leukemia or lymphoma are often refractory to further chemotherapy. In this study, the investigators will attempt to use T cells obtained directly from the patient, which can be genetically engineered to express a chimeric antigen receptor (CAR). The CAR used in this study can recognize CD22, a protein expressed on the surface of leukemia and lymphoma cells. The phase 1 part of this study will determine the safety and appropriate dose level of these CAR T cells, and the phase 2 part of the study will determine how effective this CAR T cell therapy is. Both patients who have never had prior CAR T cell therapy and those who have had prior CAR T cell therapy may be eligible to participate in this study.


Eligibility criteria: Inclusion Criteria:

* Male and female subjects aged ≤ 30 years. First 2 enrolled subjects: age ≥ 18 and ≤ 30 years
* Evidence of refractory or recurrent CD22+ leukemia or lymphoma
* Able to tolerate apheresis, or subject with sufficient existing apheresis product or T cells for manufacturing investigational product.
* Life expectancy ≥ 8 weeks
* Lansky or Karnofsky, as applicable, score ≥ 50
* Recovered from acute toxic effects of all prior chemotherapy, immunotherapy, and radiotherapy, if the subject does not have a previously obtained apheresis product that is acceptable and available for manufacturing of CAR T cells
* ≥ 7 days post last chemotherapy and biologic therapy, with the exception of intrathecal chemotherapy and maintenance chemotherapy
* ≥ 7 days post last corticosteroid therapy
* ≥ 3 days post Tyrosine Kinase Inhibitor (TKI) use
* ≥ 1 day post hydroxyurea
* 30 days post most recent CAR T cell infusion
* Adequate organ function
* Adequate laboratory values, including absolute lymphocyte count ≥ 100 cells/uL
* Subjects of childbearing or child-fathering potential must agree to use highly effective contraception from consent through 12 months following infusion of investigational product on trial
* Subject and/or legally authorized representative has signed the informed consent form for this study

Exclusion Criteria:

* Presence of active malignancy other than disease under study
* History of symptomatic CNS pathology or ongoing symptomatic CNS pathology
* CNS involvement of leukemia or lymphoma that is symptomatic and in the opinion of the investigator, cannot be controlled during the interval between enrollment and CAR T cell infusion
* Subjects with uniform expression of CD19 on their malignant cells who are eligible but have not attempted CD19 directed CAR T cell therapy
* For subjects having had a previous stem cell transplant: presence of active GVHD, or receiving immunosuppressive therapy for treatment or prevention of GVHD within 4 weeks prior to enrollment
* Presence of active severe infection,
* Presence of primary immunodeficiency syndrome
* Subject has received prior virotherapy
* Pregnant or breastfeeding
* Subject and/or legally authorized representative unwilling to provide consent/assent for participation in the 15-year follow-up period, required if CAR T cell therapy is administered
* Presence of any condition that, in the opinion of the investigator, would prohibit the subject from undergoing treatment under this protocol

Primary outcomes:
measure: The adverse events associated with CAR T cell product infusions will be assessed
timeFrame: 28 days post-infusion
measure: The ability to successfully manufacture SCRI-CAR22v2
timeFrame: 28 days
measure: The leukemia response to SCRI-CAR22v2 in subjects with relapsed or refractory CD22+ leukemia will be assessed
timeFrame: 28 days post-infusion
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Washington
country: United States
facility: Children's National Hospital
state: District of Columbia
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
Study officials:
affiliation: Seattle Children's Hospital
name: Corinne Summers, MD
role: STUDY_CHAIR
References: