A Clinical Study of CD19/BCMA CAR-T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis

NCT05263817 | Unknown Early Phase 1 DMC

• 1 other identifier: CD19/BCMA-005
• Study Type: interventional
• Target: 75
• Locations: 1 country
• Sites: 1

Brief Summary

A Clinical Study on the Safety and Effectiveness of CD19/BCMA Chimeric Antigen Receptor T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis

Conditions

POEMS Syndrome; Amyloidosis; Autoimmune Hemolytic Anemia; Vasculitis

Keywords

CD19 CAR-T; BCMA CAR-T; POEMS Syndrome; Amyloidosis; Autoimmune Hemolytic Anemia; Vasculitis

Outcome Measures

Primary Outcomes (2)

• Dose-limiting toxicity (DLT)

  Adverse events assessed according to NCI-CTCAE v5.0 criteria

  Baseline up to 28 days after CD19/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 CD19/BCMA targeted CAR T-cells infusion

Secondary Outcomes (5)

• Titer of auto-antibody Titer of auto-antibody titer of auto-antibody

  Up to 2 years after CD19/BCMA targeted CAR T-cells infusion

• Overall response rate (ORR)

  Up to 2 years after CD19/BCMA targeted CAR T-cells infusion

• Best overall response, BOR

  At ≤3 month

• Overall survival (OS)

  From CD19/BCMA CAR-T infusion to death，up to 2 years

• Duration of remission, DOR

  2 years post CD19/BCMA CAR-T cells infusion

Study Arms (4)

POEMS Syndrome

EXPERIMENTAL

Biological: CD19/BCMA CAR T-cells

Amyloidosis

EXPERIMENTAL

Biological: CD19/BCMA CAR T-cells

Autoimmune Hemolytic Anemia

EXPERIMENTAL

Biological: CD19/BCMA CAR T-cells

Vasculitis

EXPERIMENTAL

Biological: CD19/BCMA CAR T-cells

Interventions

CD19/BCMA CAR T-cells BIOLOGICAL

Each subject receive CD19/BCMA CAR T-cells by intravenous infusion

Also known as: CD19/BCMA CAR T-cells injection

Amyloidosis; Autoimmune Hemolytic Anemia; POEMS Syndrome; Vasculitis

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• \. Diagnosed with POEMS syndrome, amyloidosis, autoimmune hemolytic anemia, vasculitis, and the curative effect of conventional hormones, radiotherapy and chemotherapy, protease inhibitors is not good and (or) no effective treatment means.
• \. After glucocorticoids, cyclophosphamide or methotrexate treatments there are still relapsed and refractory diseases, or clearly show intolerance/toxicity to these drugs.
• \. Estimated survival time\> 12 weeks; 4. Patients had a negative urine pregnancy test before the start of administration and agreed to take effective contraceptive measures during the test period until the last follow-up; 5. Patients or their legal guardians volunteer to participate in the study and sign the informed consent.

You may not qualify if:

• History of craniocerebral trauma, conscious disturbance, epilepsy,cerebrovascular ischemia, and cerebrovascular, hemorrhagic diseases;
• Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythm ia in the past;
• Pregnant (or lactating) women;
• Patients with severe active infections (excluding simple urinary tract infection and bacterial pharyngitis);
• Active infection of hepatitis B virus or hepatitis C virus;
• Systemic steroids have used in the 4 weeks before participating in the treatment (except recently or currently using inhaled steroids);
• Those who have used any gene therapy products before.
• The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
• Serum creatinine \> 2.5mg/dl or ALT / AST \> 3 times ULN or bilirubin \> 2.0mg/dl;
• Those who suffer from other uncontrolled diseases are not suitable to join the study;
• HIV infection;
• Any situation that the researchers believe may increase the risk of patients or interfere with the test results.

Sponsors & Collaborators

• Zhejiang University: lead
• Yake Biotechnology Ltd.: collaborator

Study Sites (1)

The first affiliated hospital of medical college of zhejiang university

Hangzhou, Zhejiang, 310003, China

RECRUITING

MeSH Terms

Conditions

POEMS Syndrome; Amyloidosis; Anemia, Hemolytic, Autoimmune; Vasculitis

Condition Hierarchy (Ancestors)

Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunoproliferative Disorders; Immune System Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Anemia, Hemolytic; Anemia; Autoimmune Diseases; Vascular Diseases; Cardiovascular Diseases

Study Officials

• He Huang, PhD

  First Affiliated Hospital of Zhejiang University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

He Huang, PhD

CONTACT

+8613605714822; hehuangyu@126.com

Yongxian Hu, PhD

CONTACT

+8615957162012; huyongxian2000@aliyun.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: The President of The First Affiliated Hospital, College of Medicine, Zhejiang University

Study Record Dates

• First Submitted: December 1, 2021
• First Posted: March 3, 2022
• Study Start: October 8, 2021
• Primary Completion: October 1, 2024
• Study Completion: October 1, 2024
• Last Updated: March 3, 2022

Record last verified: 2022-02

Locations

CN (1)