A Clinical Study Evaluating the Safety and Efficacy of BinD19 Treatment in Childhood R/R ALL and Lymphoma Subjects

A Clinical Study Evaluating the Safety and Efficacy of BinD19 Treatment in Relapse or Refractory Childhood Acute Lymphoblastic Leukemia and Lymphoma Subjects

This is a single arm, open-label, uni-center, phase I/II study to determine the safety and efficacy of an experimental therapy called BinD19 cells in childhood patients with B-cell acute lymphoblastic leukemia or lymphoma, who are chemo-refractory, relapsed after allogeneic SCT, or are otherwise ineligible for allogeneic stem cell transplant.

Study Overview

• Status: Active, not recruiting
• Conditions: Relapsed B-cell Acute Lymphoblastic Leukemia, Childhood; Refractory B-cell Acute Lymphoblastic Leukemia, Childhood; Relapsed/Refractory B-cell Lymphoma, Childhood
• Intervention / Treatment: Biological: BinD19

• Study Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China, 201102
      • Children's Hospital of Fudan University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Male and female subjects with CD19+ B cell malignancies in patients with no available curative treatment options (such as autologous or allogeneic SCT) who have limited prognosis (several months to < 2 year survival) with currently available therapies will be enrolled.
• CD19+ leukemia or lymphoma
• Not eligible for allogeneic SCT because of age, comorbid disease, or lack of available family member or unrelated donor
• Follicular lymphoma, previously identified as CD19+:
• Disease responding or stable after most recent therapy (chemotherapy, MoAb, etc)
• ECOG result is 0, 1 or 2.
• With normal heart, liver and kidney functions.
• Negative serum DNA for EBV and CMV; negative antigen for HBV; negative serum antibody for HCV, HIV and syphilis.
• Negative in pregnancy test (female subject only).

Exclusion Criteria:

• ECOG result is 3, 4 or 5.
• Pregnant or lactating female
• Uncontrolled active infection
• Active hepatitis B or hepatitis C infection
• Concurrent use of systemic steroids. Recent or current use of inhaled steroids is not exclusionary
• Previously treatment with any gene therapy products
• HIV infection
• Enrolled to other clinical study in the last 4 weeks.
• Subjects with systemic auto-immune disease or immunodeficiency.
• Subjects with CNS diseases.
• Subjects with secondary tumors.
• Subjects with tumor infiltration in liver, brain or GI tract.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BinD19; BinD19 (autologous T cells transduced with CD19 TCR-ζ/4-1BB vector) administered as an IV infusion on days 0, 1 and 2 in the absence of disease progression or unacceptable toxicity. Minimum/ maximum dose: 1x10^6/kg / 1x10^7/kg administered to childhood patients with R/R B cell Acute Lymphoblastic Leukemia (ALL) or Lymphoma.	Biological: BinD19; Autologous T cells purified from the peripheral blood mononuclear cells of subjects, transduced with TCR- ζ/4-1BB lentiviral vector, expanded in vitro and then frozen for future administration.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Adverse Events [Safety and Feasibility]	To evaluate the safety and feasibility of BinD19 CAR-T cells in patients with refractory /relapsed B-cell acute lymphoblastic leukemia or lymphoma.	Study treatment until Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Response [Efficacy]	Efficacy assessments for ALL were performed based on bone marrow and blood morphologic criteria and physical examination findings. Efficacy assessments for Lymphoma were based on tumor load, B cell number and immunoglobulins.	5 years

Collaborators and Investigators

• Sponsor: Shenzhen BinDeBio Ltd.
• Collaborators: Children's Hospital of Fudan University
• Investigators: Study Director: ZHONG HUA YANG, Shenzhen BinDeBio Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2016
• Primary Completion (Anticipated): May 30, 2021
• Study Completion (Anticipated): December 30, 2021

Study Registration Dates

• First Submitted: August 25, 2017
• First Submitted That Met QC Criteria: August 25, 2017
• First Posted (Actual): August 29, 2017

Study Record Updates

• Last Update Posted (Actual): February 4, 2021
• Last Update Submitted That Met QC Criteria: February 3, 2021
• Last Verified: February 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Lymphoma; Leukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Lymphoid
• Other Study ID Numbers: 2017FDEK-BinD19

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No