A Study of EXP039 Treatment in Subjects With r/r NHL Subjects

A Phase 1 Study Evaluating Safety and Efficacy of EXP039 Treatment in Subjects With Relapsed and/or Refractory NHL

This is a single-center, non-randomized study to evaluate the safety and efficacy of EXP039 in relapsed and/or refractory NHL patients.

Study Overview

• Status: Unknown
• Conditions: Non-Hodgkin's B-cell Lymphoma
• Intervention / Treatment: Biological: CD19/CD20-directed CAR-T cells

Detailed Description

The study will include the following sequential phases: Screening, Apheresis, Baseline, Pre-Treatment (Cell Product Preparation, Lymphodepleting Chemotherapy), EXP039 infusion and Follow-up Visit.

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Phase 1

Contacts and Locations

Study Locations

• China
  • Beijing/China
    • Beijing, Beijing/China, China, 100000
      • Recruiting
      • Peking Union Medical College Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. The patient volunteered to participate in the study and signed the Informed Consent
2. Age ≥18 years old ≤70 Years old, male or female
3. Expected survival ≥ 12 weeks
4. ECOG score 0-2
5. CD19 or CD20 positive B-NHL confirmed by cytology or histology according to WHO2016 criteria
6. Patients with a clear diagnosis of relapsed and/or refractory B-NHL, including DLBCL, FL and MCL
7. For CD20-positive subjects, they should have received at least one regimen containing anti-CD20-targeted therapy (such as rituximab). If they do not complete the regimen due to intolerance, the cause of intolerance should be recorded
8. No contraindications of apheresis
9. At least one measurable lesion according to Lugano 2014 criteria
10. Adequate organ function and adequate bone marrow reserve

Exclusion Criteria:

1. Malignant tumors other than B-NHL within 5 years prior to screening, except cervical carcinoma in situ, basal cell or squamous cell skin cancer, local prostate cancer after radical surgery, and breast ductal carcinoma in situ after radical surgery
2. Active HIV, HBV, HCV or treponema pallidum infection
3. Any instability of systemic disease, including but not limited to active infection (except local infection), severe cardiac, liver, kidney, or metabolic disease need therapy
4. Female subjects who have been pregnant or breastfeeding, or who plan to conceive during or within 1 year after treatment, or male subjects' partner plans to conceive within 1 year after their cell transfusion
5. Active or uncontrolled infections requiring systemic treatment within 14 days before enrollment
6. Patients who have been previously infected with tuberculosis
7. Administered Corticosteroids and/or other immunosuppressants within 7 days before apheresis. and 5 days before the infusion of EXP039
8. Patients with central nervous system involvement
9. Any systemic antitumor therapy performed within 2 weeks before enrollment
10. Previous use of any CAR T cell product or other genetically modified T cell therapy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Autologous EXP039 administered by intravenous (IV) infusion	Biological: CD19/CD20-directed CAR-T cells; Autologous 2nd generation CD19/CD20-directed CAR-T cells, single infusion intravenously; Other Names: EXP039

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of study related adverse events	Incidence and severity of Treatment emergent adverse events	12 Months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum concentration (Cmax) of EXP039 in the peripheral blood	Detect CAR-T copies number by qPCR	up to 12 months
Time to maximum concentration (Tmax) of EXP039 in the peripheral blood	Detect CAR-T copies number by qPCR	up to 12 months
Tlast of EXP039 in the peripheral blood after infusion	Detect CAR-T copies number by qPCR	up to 12 months
AUC0h-28d of EXP039 in the peripheral blood	Detect CAR-T copies number by qPCR	4 weeks
Objective response rate (ORR)	Complete response (CR) rate plus partial response (PR) rate by Lugano 2014 criteria	4 weeks, 12 weeks, 6 months, 9 months, 12 months
Duration of response (DOR)	The time from the date of first response (PR or better) until the date of disease progression after EXP039 infusion	up to 12 months
Progression-free survival (PFS)	The time from EXP039 infusion to the date of progression as assessed by Lugano 2014 criteria or death	4 weeks, 12 weeks, 6 months, 9 months, 12 months
Overall survival rate (OSR)	The time from EXP039 infusion to the date of death	12 weeks, 6 months, 12 months

Collaborators and Investigators

• Sponsor: Peking Union Medical College Hospital
• Investigators: Principal Investigator: Daobin Zhou, PhD&MD, Peking Union Medical College Hospital

Study record dates

Study Major Dates

• Study Start (ACTUAL): August 25, 2020
• Primary Completion (ANTICIPATED): July 1, 2022
• Study Completion (ANTICIPATED): October 1, 2022

Study Registration Dates

• First Submitted: September 25, 2020
• First Submitted That Met QC Criteria: November 30, 2020
• First Posted (ACTUAL): December 7, 2020

Study Record Updates

• Last Update Posted (ACTUAL): December 7, 2020
• Last Update Submitted That Met QC Criteria: November 30, 2020
• Last Verified: July 1, 2020

More Information

Terms related to this study

• Keywords: CD19/CD20-directed CAR-T cells
• Additional Relevant MeSH Terms: Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Lymphoma, Non-Hodgkin; Lymphoma; Lymphoma, B-Cell
• Other Study ID Numbers: 0702-023

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