Efficacy and Safety Evaluation of BCMA-UCART

Efficacy and Safety Evaluation of BCMA-UCART（Allogeneic Engineered T-cells Expressing Anti-BCMA Chimeric Antigen Receptor）in the Treatment of Relapsed or Refractory Multiple Myeloma

This trial aims to evaluate the safety and efficacy of BCMA-UCART in treating patients with relapsed or refractory multiple myeloma.

Study Overview

• Status: Suspended
• Conditions: Multiple Myeloma
• Intervention / Treatment: Biological: BCMA-UCART

Detailed Description

BCMA(B-Cell maturation antigen) is a tumor antigen of multiple myeloma. Using a genetic engineering strategy to assemble an anti-BCMA CAR(chimeric antigen receptor) in T cells will help these CART cells to recognize and kill BCMA-expressing MM tumor cells. BCMA-UCART is a kind of allogeneic CART, originating from T cells of health donors. This open-label, dose-escalation study aims to evaluate the safety and anti-tumor efficacy of BCMA-UCART in treating relapsed or refractory multiple myeloma.

• Study Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Not Applicable

Contacts and Locations

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China, 200065
      • Shanghai Tongji Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 61 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Have the capacity to give informed consent;
• Confirmed diagnosis of active MM as defined by NCCN and IMWG criteria;
• Have a diagnosis of BCMA+ multiple myeloma (MM), (≥ 5% BCMA+ in CD138+ plasma cells by flow cytometry obtained within 45 days of study enrollment);
• Refractory and relapsed MM patients after > 2 cycles of induction therapy,or,have relapsed or treatment refractory disease following autologous stem cell transplant (ASCT);
• ECOG score=0-2.

• Subjects according with any of the following options:

  • Age≥50;
  • Failure with separation of T cells during autologous CART processing; or,
  • Failure with expansion of autologous CART; or,
  • The proportion of T cells in PBMC <10%; or,
  • Won't benefit from autologous CART therapy because of disease progress.

Exclusion Criteria:

• Pregnant or nursing women; Women of reproductive potential must have a negative serum pregnancy test performed within 48 hours of starting conditioning chemotherapy
• Active infection, HIV infection, syphilis serology reaction positive;
• Active hepatitis B, hepatitis C at the time of screening
• Significant hepatic dysfunction as following, SGOT(serum glutamic-oxaloacetic transaminase)> 5 x upper limit of normal; bilirubin > 3.0 mg/dL;
• Lymphotoxic chemotherapeutic agents within 2 weeks of leukapheresis
• serious mental disorder;
• With severe cardiac, liver, renal insufficiency, diabetes and other diseases;
• Participate in other clinical research in the past three months; previously treatment with any gene therapy products
• Contraindication to cyclophosphamide or fludarabine chemotherapy

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BCMA-UCART; Each subject will accept one of the following dosages of BCMA-UCART cells intravenously (IV) on day 0: 0.5-1*10~6/KgBW, 1-2*10~6/KgBW,2-3*10~6/KgBW.	Biological: BCMA-UCART; A conditioning therapy with cyclophosphamide and fludarabine will be conducted for subjects before CART therapy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective response rate (ORR)	Proportion of patients in whom a response among complete response or partial response, as defined by International Myeloma Working group(IMWG) response criteria , will be observed.	Up to 90 days after T cell infusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence and Severity of Adverse Events as a Measure of Safety and Tolerability	Adverse events assessed according to NCI-CTCAE v4.03 criteria	Up to 35 days after T cell infusion
Duration of persistence of BCMA-UCART	Detect the duration of BCMA-UCART after injection using FACS or Q-PCR	Baseline up to 1 year

Collaborators and Investigators

• Sponsor: Bioray Laboratories
• Collaborators: Second Xiangya Hospital of Central South University; Shanghai Tongji Hospital, Tongji University School of Medicine
• Investigators: Principal Investigator: Aibin Liang, PhD, Shanghai Tongji Hospital, Tongji University School of Medicine

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2019
• Primary Completion (Anticipated): March 20, 2023
• Study Completion (Anticipated): November 20, 2023

Study Registration Dates

• First Submitted: November 21, 2018
• First Submitted That Met QC Criteria: November 21, 2018
• First Posted (Actual): November 26, 2018

Study Record Updates

• Last Update Posted (Actual): May 20, 2022
• Last Update Submitted That Met QC Criteria: May 15, 2022
• Last Verified: May 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Immunoproliferative Disorders; Hematologic Diseases; Hemorrhagic Disorders; Hemostatic Disorders; Paraproteinemias; Blood Protein Disorders; Multiple Myeloma; Neoplasms, Plasma Cell
• Other Study ID Numbers: 2018-CAR-00CH3

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