Clinical Trial

Disease: Relapsed or Refractory Multiple Myeloma, MM, (NCT03752541)

Disease info:

Multiple myeloma is a cancer that develops in the bone marrow, the spongy tissue found in the centre of most bones. Multiple myeloma is characterised by abnormalities in plasma cells, a type of white blood cell. In myeloma, these abnormal cells multiply uncontrollably, increasing from about one percent of cells in the bone marrow to the majority of bone marrow cells. The abnormal cells form tumours within the bone, causing bone pain and an increased risk of fractures.

Relapsed myeloma refers to when a patient had active treatment that their disease responded to, went off treatment and then the disease came back. 

Refractory myeloma is a disease that is progressing despite active treatment.

 

Frequency:
Multiple myeloma occurs in approximately 4 per 100,000 people per year; there are currently about 100,000 affected individuals in the United States.
Official title:
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
Who:

Principal Investigator: Aibin Liang, PhD.  Shanghai Tongji Hospital, Tongji University School of Medicine

Sponsor:

Bioray Laboratories - Bangyao Biotechnology Co., Ltd. 

Partners:

Shanghai Tongji Hospital, Tongji University School of Medicine

Second Xiangya Hospital of Central South University

Locations:

China, Shanghai

Shanghai Tongji Hospital, Shanghai, Shanghai, China, 200065

Study start:
Nov. 1, 2019
Enrollment:
20 participants
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene knock-out
Gene:
T Cell Receptor Alpha Constant (TRAC), Human Leukocyte Antigen Class I (HLA-I)
Delivery method:
- Ex-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/record/NCT03752541
Safety updates:

(24-05-2021) LoA Update: Shanghai Bioray Laboratory’s gene therapy BCMA-UCART for multiple myeloma plunges 12 points in FDA regulatory potential following updated Phase I completion date

Other links:

LinkMultiple Myeloma Disease Information and FrequencyLinkThe Role of Gene Editing Within CAR T-Cell Therapy Development - Interview with Professor Waseem QasimLinkApplications and explorations of CRISPR-Cas9 in CAR T-cell therapyLinkClinical Trial Update: Gene-edited CAR T Therapies

IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Suspended

Description

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

A conditioning therapy with cyclophosphamide and fludarabine will be conducted for subjects before CART therapy.

Last updated: Apr. 20, 2024
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