Clinical trial NCT02924753: The Safety and Efficacy of CART-19 Cells in B-cell Acute Lymphoblastic Leukemia (B-ALL).
Official title: The Safety and Efficacy of CART-19 Cells in Relapse and Refractory Patients With CD19+ B-cell Acute Lymphoblastic Leukemia.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: B-cell Acute Lymphoblastic Leukemia
Lead sponsor: Henan Cancer Hospital
Sponsor class: OTHER_GOV
Collaborators: name: The Beijing Pregene Science and Technology Company, Ltd.
First posted: 2016-10-05
Enrollment: 20
Sex: ALL
Minimum age (years): 4
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a study for patients who have been previously treated for B-ALL. The purpose of this study is to determine the safety and feasibility of CART-19 cells to the patients with relapsed and refractory CD19+ B-ALL.

Detailed description: Subjects will be staged and the suitability of their T cells for CART-19 manufacturing will be determined at entry phase,. Subjects will be collected large numbers of peripheral blood mononuclear cells (PBMC) for CART-19 manufacturing. The T cells will be purified from the PBMC, transduced with CART-19 lentiviral vector, expanded in vitro and then administered to subjects.

Subjects will have blood tests to assess safety and efficacy, and persistence of the CART-19 cells at regular intervals through four weeks after their last infusion of the study. Following the 6 months of intensive follow-up, subjects will be evaluated quarterly for two years with a physical examination, blood tests, bone marrow aspirate, minimal residual disease (MRD) and persistence of CART-19. Following this evaluation, subjects will be evaluated health problems every year for an additional thirteen years.

Eligibility criteria: Inclusion Criteria:

1. 4 years to 70 years, expected survival \> 3 months
2. CD19 positive B-cell acute lymphoblastic leukemia
3. Karnofsky Performance Status (KPS) \>70
4. Relapsed after allogeneic or autologous stem cell transplantation (SCT);
5. Cardiac function: 1-2 levels; Liver: TBIL≤3 Upper Limit of Normal (ULN)，aspartate aminotransferase (AST) ≤2.5 ULN，ALT ≤2.5 ULN; kidney: Cr≤1.25 ULN; bone marrow: White Blood Cell (WBC) ≥ 3.0×109/L, Hb ≥90 g/L, Platelet (PLT) ≥ 80×109/L）
6. No serious allergic constitution
7. No other serous diseases that conflicts with the clinical program
8. No other cancer history
9. No serious mental disorder
10. Informed consent is signed by a subject or his lineal relation.

Exclusion Criteria:

1. Pregnant or lactating women; (female participants of reproductive potential must have a negative serum or urine pregnancy test)
2. Uncontrolled active infection, HIV infection, syphilis serology reaction positive
3. Active hepatitis B or hepatitis C infection
4. Recent or current use of glucocorticoid or other immunosuppressor
5. With severe cardiac, liver, renal insufficiency, diabetes and other diseases
6. Transaminase \>2.5 ULN, Bilirubin \>3 ULN，Creatinine\>1.25 ULN
7. Participate in other clinical research in the past three months; previously treatment with any gene therapy products
8. Researchers think of that does not fit to participate in the study, or other cases that affect the clinical trial results

Primary outcomes:
measure: safety as assessed by the occurrence of study related adverse events.
timeFrame: 6 months
Secondary outcomes:
measure: efficacy
timeFrame: 2 years
measure: duration of CART-19
timeFrame: 2 years
Other outcomes:
Locations:
city: Zhengzhou
country: China
facility: Henan Cancer Hospital
state: Henan
Study officials:
affiliation: Henan Cancer Hospital
name: Yongping Song
role: STUDY_DIRECTOR
References: