A Study of Humanized BCMA-targeted CAR-T Cells Therapy for Refractory/Relapsed Multiple Myeloma

Clinical Trial for the Safety and Efficacy of Humanized BCMA-targeted CAR-T Cells Therapy for Refractory/Relapsed Multiple Myeloma

Clinical Trial for the safety and efficacy of humanized BCMA-targeted CAR-T cells therapy for refractory/relapsed multiple myeloma

Study Overview

• Status: Not yet recruiting
• Conditions: Refractory Multiple Myeloma; Relapse Multiple Myeloma
• Intervention / Treatment: Drug: BCMA Targeted CAR T-cells

Detailed Description

This is a single arm, open-label, single-center study. This study is indicated for relapsed BCMA+ multiple myeloma, the selections of dose levels and the number of subjects are based on clinical trials of similar foreign products. 50 patients will be enrolled for this trial. Primary objective is to explore the safety, main consideration is dose-related safety.

• Study Type: Interventional
• Enrollment (Anticipated): 50
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• China
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310003
      • The First Affiliated Hospital, Medical College, Zhejiang University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 26 years to 71 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Histologically confirmed diagnosis of multiple myeloma (MM):

   1. Patients with BCMA positive relapsed/refractory MM;
   2. Relapsed after hematopoietic stem cell transplantation;
   3. Cases with recurrent positive minimal residual disease;
   4. Extramedullary leision which is hard to be eradicated by chemotherapy or radiotherapy.
2. Anticipated survival time more than 12 weeks;
3. Male or female aged 30-75 years;
4. Those who voluntarily participated in this trial and provided informed consent.

Exclusion Criteria:

Subjects with any of the following exclusion criteria were not eligible for this trial:

1. History of craniocerebral trauma, conscious disturbance, epilepsy, cerebrovascular ischemia, and cerebrovascular hemorrhagic diseases;
2. Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythmia in the past;
3. Pregnant (or lactating) women;
4. Patients with severe active infections (excluding simple urinary tract infection and bacterial pharyngitis);
5. Active infection of hepatitis B virus or hepatitis C virus;
6. Concurrent therapy with systemic steroids within 2 weeks prior to screening, except for the patients recently or currently receiving in haled steroids;
7. Previously treated with any CAR-T cell product or other genetically-modified T cell therapies;
8. Creatinine>2.5mg/dl, or ALT / AST > 3 times of normal amounts, or bilirubin>2.0 mg/dl;
9. Other uncontrolled diseases that were not suitable for this trial;
10. Patients with HIV infection;
11. Any situations that the investigator believes may increase the risk of patients or interfere with the results of study.

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: Administration of BCMA Targeted CAR T-cells; Dose escalation follows the standard 3+3 dose escalation design. A total of 3 dose levels are set for subjects.	Drug: BCMA Targeted CAR T-cells; Each subject receive BCMA Targeted CAR T-cells by intravenous infusion; Other Names: BCMA Targeted CAR T-cells injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Dose-limiting toxicity (DLT)	Adverse events assessed according to NCI-CTCAE v5.0 criteria	Baseline up to 28 days after BCMA targeted CAR T-cells infusion
Incidence of treatment-emergent adverse events (TEAEs)	Incidence of treatment-emergent adverse events [Safety and Tolerability]	Up to 2 years after BCMA targeted CAR T-cells infusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Activities of Daily Living (ADL) score	Assessment using Activities of Daily Living (ADL) scale (Barthel Index) [max score: 100, min score: 0, higher scores mean a better outcome] at Baseline, Month 1, 3, 6, 9 and 12	At Baseline, Month 1, 3, 6, 9 and 12
Instrumental Activities of Daily Living (IADL) score	Assessment of Instrumental Activities of Daily Living (IADL) scale [max score: 56, min score: 14, higher scores mean a worse outcome] at Baseline, Month 1, 3, 6, 9 and 12	At Baseline, Month 1, 3, 6, 9 and 12
Overall response rate (ORR)	Assessment of ORR at Day 28	At Day 28
Overall survival (OS)	Assessment of OS at Month 6, 12, 24	At Month 6, 12, 24
Quality of life(EORTC QLQ-C30) Core 30 (EORTC QLQ-C30)	Assessment using European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) scale [For item1-28: max score: 112, min score: 28, higher scores mean a better outcome; for item 28-29: max score: 14, min score: 2, higher scores mean a worse outcome] to measure Quality of life at Baseline, Month 1, 3, 6, 9 and 12	At Baseline, Month 1, 3, 6, 9 and 12
Hospital Anxiety and Depression Scale (HADS) score	Assessment of Hospital Anxiety and Depression Scale (HADS) score at Baseline, Month 1, 3, 6, 9 and 12	At Baseline, Month 1, 3, 6, 9 and 12

Collaborators and Investigators

• Sponsor: Zhejiang University
• Collaborators: Yake Biotechnology Ltd.

Study record dates

Study Major Dates

• Study Start (Anticipated): January 1, 2021
• Primary Completion (Anticipated): January 1, 2024
• Study Completion (Anticipated): January 1, 2027

Study Registration Dates

• First Submitted: December 9, 2020
• First Submitted That Met QC Criteria: December 9, 2020
• First Posted (Actual): December 17, 2020

Study Record Updates

• Last Update Posted (Actual): December 17, 2020
• Last Update Submitted That Met QC Criteria: December 9, 2020
• Last Verified: December 1, 2020

More Information

Terms related to this study

• Keywords: CAR T-cell therapy; multiple myeloma; BCMA
• 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: hBCMA-ZhejiangU

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