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Study of T Cells Targeting CD19/BCMA (CART-19/BCMA) for High Risk Multiple Myeloma Followed With Auto-HSCT

Phase 1
Conditions
Safety and Efficacy
Interventions
Biological: anti-CD19 and anti-BCMA CAR
Drug: Immunomodulatory drugs
Registration Number
NCT03455972
Lead Sponsor
The First Affiliated Hospital of Soochow University
Brief Summary

CART therapy has showed good safety and efficacy in treatment of lymphoma and acute lymphoblastic leukemia. Researchers want to see if this helps people with high risk multiple myeloma after auto-HSCT.To test the safety and efficacy of giving targeting CD19 and BCMA T cells in treating high risk multiple myeloma followed with auto-HSCT.

Detailed Description

Adults ages 18-75 with high risk Multiple Myelomas (R-ISS III stage or with extramedullary infiltration or with del(17p), t(4;14), t(14;16), t(14;20), 1q21+ or disease progression during treatment).

Design:

Participants may be screened with:

Medical history Physical exam Blood and urine tests Heart tests Bone marrow sample Multiple scans and X-rays Participants will have apheresis. Blood is removed through a needle in an arm. T cells are removed. The rest of the blood is returned through a needle in the other arm.

The cells will be changed in a laboratory. Participants will get auto-HSCT. Hematopoietic reconstitution after auto-HSCT, participants will get the T cells through the IV within 3 days. Maintenance therapy with IMiDs was received after combined CAR T infusion.

After this, participants will stay in the hospital for at least 9 days and stay nearby for 2 weeks. Then they will have blood tests and see a doctor.

Participants will visit the clinic 1, 2, 3, 6, 9 and 12 months after the infusion, then every 3 months until disease progression. A bone marrow sample will be taken at the 3-month visit.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
15
Inclusion Criteria
  • Multiple myeloma patients eligible for auto-HSCT.
  • High risk multiple myeloma (R-ISS III stage or with extramedullary infiltration or with del(17p), t(4;14), t(14;16), t(14;20), 1q21+ or disease progression during treatment).
  • Expected survival ≥ 3 months.
  • Creatinine < 2.0 mg/dl.
  • Blood coagulation function: PT and APTT <2x normal.
  • Arterial blood oxygen saturation>92%.
  • ALT(alanine aminotransferase)/AST (aspartate aminotransferase)< 3x normal
  • Karnofsky scores ≥ 60 and ECOG score≤2.
  • Adequate venous access for apheresis, and no other contraindications for leukapheresis.
  • Patients should not take immunotherapy in three months prior to CART cells infusion.
  • Voluntary informed consent is given.
Exclusion Criteria
  • Pregnant or lactating women.
  • 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.
  • Any uncontrolled active medical disorder that would preclude participation as outlined.
  • HIV infection.
  • History of myocardial infarction and severe arrhythmia in half a year.
  • Any form of primary immunodeficiency (such as Severe Combined Immunodeficiency Disease).
  • Patients with fever of unknown origin (T>38℃).

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
anti-CD19 and anti-BCMA CARanti-CD19 and anti-BCMA CARParticipants will get auto-HSCT. Hematopoietic reconstitution after auto-HSCT, participants will get the anti-CD19 CAR T cells (on d0) and anti-BCMA CAR T cells as split-dose (40% on d1 and 60% on d2)
anti-CD19 and anti-BCMA CARImmunomodulatory drugsParticipants will get auto-HSCT. Hematopoietic reconstitution after auto-HSCT, participants will get the anti-CD19 CAR T cells (on d0) and anti-BCMA CAR T cells as split-dose (40% on d1 and 60% on d2)
Primary Outcome Measures
NameTimeMethod
OSMinimum of 2 years after first induction

Is defined as time from first induction date to time of death due to any cause

Incidence and severity of adverse eventsApproximately 3 years

Proportion of subjects with adverse events overall and by severity grade

PFSMinimum of 2 years after first induction

Is defined as time from first induction date to first documentation of PD, or death due to any cause, whichever occurs first.

Secondary Outcome Measures
NameTimeMethod
Duration of best responseMinimum of 2 years

According to IMWG response criteria at the end of the research.

MRD negative conversion ratio and persistenceMinimum of 2 years

MRD negative by flow cytometry

Proportion of subjects who achieved Complete Response (CR) RateMinimum of 2 years

Percentage of subjects who achieved CR or stringent CR according to IMWG Uniform Response Criteria for Multiple Myeloma

Pharmacokinetics - TmaxApproximately 2.5 years after first CAR infused

Time to peak transgene level

Pharmacokinetics - CmaxApproximately 2.5 years after first CAR infused

Maximum transgene level

Pharmacokinetics - AUCApproximately 2.5 years after first CAR infused

Area under the curve of the transgene level

Duration of persistence of CAR T cells in the bloodApproximately 2.5 years after first CAR infused

Duration of persistence of CAR T cells in the blood

Trial Locations

Locations (1)

First Affiliated Hospital, Soochow University

🇨🇳

Suzhou, Jiangsu, China

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