Clinical trial NCT03752541: Efficacy and Safety Evaluation of BCMA-UCART
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
Study type: INTERVENTIONAL
Overall status: SUSPENDED
Phase: NA
Conditions: Multiple Myeloma
Lead sponsor: Bioray Laboratories
Sponsor class: INDUSTRY
Collaborators: name: Shanghai Tongji Hospital, Tongji University School of Medicine
name: Second Xiangya Hospital of Central South University
First posted: 2018-11-26
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NA
Intervention model: SEQUENTIAL
Intervention model description: 0.5-1\*10\~6/KgBW, 1-2\*10\~6/KgBW,2-3\*10\~6/KgBW
Primary purpose: TREATMENT

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

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.

Eligibility criteria: 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

Primary outcomes:
measure: Objective response rate (ORR)
timeFrame: Up to 90 days after T cell infusion
Secondary outcomes:
measure: Incidence and Severity of Adverse Events as a Measure of Safety and Tolerability
timeFrame: Up to 35 days after T cell infusion
measure: Duration of persistence of BCMA-UCART
timeFrame: Baseline up to 1 year
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Shanghai Tongji Hospital
state: Shanghai
Study officials:
affiliation: Shanghai Tongji Hospital, Tongji University School of Medicine
name: Aibin Liang, PhD
role: PRINCIPAL_INVESTIGATOR
References: