Clinical trial NCT02735291: Study Evaluating the Efficacy and Safety With CAR-T for Recurrent or Refractory Acute Non T Lymphocyte Leukemia
Official title: Single Arm, Two Phase, Multicenter Trial to Evaluating the Efficacy and Safety of the CAR-T for Recurrent or Refractory Acute Non T Lymphocyte Leukemia
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Leukemia
Keywords: Leukemia CAR-T
Lead sponsor: Sinobioway Cell Therapy Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: The Second Hospital of Anhui Medical University
First posted: 2016-04-12
Enrollment: 72
Sex: ALL
Minimum age (years): 3
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This single-arm,multicenter Phase 2 trial will treat the patients who have recurrent or refractory acute non T lymphocyte leukemia with an infusion of the patient's own T cells that have been genetically modified to express a chimeric antigen receptor (CAR) that will bind to tumour cells that express the EPCAM protein on the cell surface.The study will determine if these modified T cells help the body's immune system eliminate tumour cells.The trial will also study the safety of treatment with CAR-T,how long CAR-T cells stay in the patient's body and the impact of this treatment on survival.

Detailed description: This is a single-arm,multicenter Phase 2 study to evaluate the efficacy and safety of the CAR-T for recurrent or refractory acute non T lymphocyte leukemia.The study will be conducted using a phase I/II design.The study will have the following sequential phases:Part A (screening leukapheresis, cell product preparation, and cytoreductive chemotherapy) and Part B (treatment and follow-up). The follow-up period for each participant is approximately 35 months after the final CAR-T infusion. The total duration of the study is expected to be approximately 3 years. A total of 24 patients may be enrolled over a period of 3 years.

Eligibility criteria: Inclusion Criteria:

1. All patients with acute non T lymphocytic leukemia after conventional treatment is invalid or recurrence of refractory, and by flow cytometry or pathological immunohistochemical examination, confirm the leukemia cells express can intervene molecular targets;
2. Age 3 to 75 years old, both male and female;
3. Is expected to survive more than 3 months;
4. Physical condition is good: 0-2 score ECOG score;
5. General requirements peripheral blood as basic normal (i.e., white blood cells \>= 4.0 x10\^9 / L, hemoglobin \> 100 g/L, the platelet count\>= 50 x10\^9 / L ), progress faster, in patients with special severe fully inform the patient related risk and gain understanding and consent, peripheral blood cell index can be extended to white blood cells \>= 2.0 x10\^9 / L, hemoglobin \> 60 g/L, the platelet count \>= 30 x10\^9 / L. But blood T lymphocytes in peripheral blood count must \>=0.2 x10\^9 / L;
6. No obvious abnormal heart, liver and kidney function (namely basic normal ecg; kidney function: Cr \<=2.0 x ULN (Upper limit of normal value ); liver function: Alt/aspartate aminotransferase acuities \<=2.5 x ULN, BIL\<= 2.0 x ULN) , no large wounds that haven't healed on the body;
7. Into groups to participate in voluntarily, good adherence, can cooperate test observation, childbearing age women must be 7 days before starting treatment expert pregnancy test and the results were negative, and signed a written informed consent form.

Exclusion Criteria:

1. Various types of T lymphocyte leukemia, etc;
2. Organ failure, such as heart: Class III and IV; liver: to Child grading of liver function grade C; kidney: kidney failure and uremia stage; lung: symptoms of severe respiratory failure; brain: disorder of consciousness;
3. Existing serious acute infection, uncontrollable, or have fester sex and chronic infection, wound in delay no more.
4. Patients with significant graft versus host disease (GVHD) after organ transplant history or allogeneic hematopoietic stem cell transplantation;
5. Systemic autoimmune diseases or immunodeficiency disease, patients with allergic constitution.
6. Coagulation abnormalities and severe thrombosis;
7. Pregnancy and lactation women;
8. Any other chronic disease patients who have been treated with immune agents or hormone therapy;
9. Patients who have participated in other clinical trials or other clinical trials in the past 30 days
10. The Investigator believe the patients should not participate in this experiment.

Primary outcomes:
measure: The overall efficiency
timeFrame: 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Hefei
country: China
facility: The Second Hospital of Anhui Medical University
state: Anhui
city: Nanjing
country: China
facility: No. 454 Hospital of People'S Liberation Army
state: Jiangsu
Study officials:
affiliation: Chief physician
name: Zhimin Zhai, Professor
role: PRINCIPAL_INVESTIGATOR
affiliation: Chief physician
name: Hui Liao, Doctor
role: STUDY_DIRECTOR
References:
citation: An F, Wang H, Liu Z, Wu F, Zhang J, Tao Q, Li Y, Shen Y, Ruan Y, Zhang Q, Pan Y, Zhu W, Qin H, Wang Y, Fu Y, Feng Z, Zhai Z. Influence of patient characteristics on chimeric antigen receptor T cell therapy in B-cell acute lymphoblastic leukemia. Nat Commun. 2020 Nov 23;11(1):5928. doi: 10.1038/s41467-020-19774-x.