Dose-escalation Study of Safety of PBCAR20A in Subjects With r/r NHL or r/r CLL/SLL
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04030195 |
Recruitment Status :
Recruiting
First Posted : July 23, 2019
Last Update Posted : November 2, 2020
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Non-Hodgkin's Lymphoma, Relapsed Chronic Lymphoid Leukemia in Relapse Non-Hodgkin's Lymphoma Refractory Chronic Lymphocytic Leukemia Lymphoma, Non-Hodgkin Leukemia, Lymphocytic, Chronic B-cell Chronic Lymphocytic Leukemia B-cell Non Hodgkin Lymphoma Small Lymphocytic Lymphoma | Genetic: PBCAR20A Drug: Fludarabine Drug: Cyclophosphamide | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 90 participants |
Allocation: | Non-Randomized |
Intervention Model: | Sequential Assignment |
Intervention Model Description: | Part 1: In each cohort, B-cell NHL (Cohort A) or r/r CLL/SLL (Cohort B), 3 escalating dose groups will be enrolled and treated sequentially, with the possibility of a single de-escalation. Within each dose group, at least 3 and at most 6 study participants will be treated with a single dose of PBCAR20A using a standard 3 + 3 design. The starting dose of PBCAR20A will be 3 × 10^5 CAR T cells/kg body weight. Subsequent dose groups will be treated with escalating doses to a maximum dose of 3 × 10^6 CAR T cells/kg. In the absence of DLTs (as described in Section 3.7), the dose will be increased using a fixed-dose scheme. Part 2: Upon the determination of the MTDs for Cohorts A and B, these respective doses will be used in the dose-expansion part, in which additional study participants will be enrolled and treated. Study participants in the expanded cohorts will be treated with a single dose of PBCAR20A. |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 1/2a, Open-label, Dose-escalation, Dose-expansion, Parallel Assignment Study to Evaluate the Safety and Clinical Activity of PBCAR20A in Study Participants With Relapsed/Refractory (r/r) Non-Hodgkin Lymphoma (NHL) or r/r Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL) |
Actual Study Start Date : | March 24, 2020 |
Estimated Primary Completion Date : | March 2022 |
Estimated Study Completion Date : | February 2023 |

Arm | Intervention/treatment |
---|---|
Experimental: Dose Level 1 of PBCAR20A CAR T cells
1 x 10^6 CAR T cells per kg body weight. In this study, PBCAR20A, allogeneic anti-CD20 CAR T Cells, is used to treat patients with relapsed or refractory (r/r) Non-Hodgkin Lymphoma (NHL) or r/r Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL). Route of Administration: Intravenous infusion. Lymphodepletion Conditioning: Lymphodepletion will be conducted several days prior to PBCAR20A infusion. A combination of fludarabine and cyclophosphamide will be used for lymphodepletion. |
Genetic: PBCAR20A
Single dose of Allogeneic Anti-CD20 CAR T cells will be infused, and a classic "3+3" dose escalation will be applied.
Other Name: Allogeneic Anti-CD20 CAR T cells Drug: Fludarabine Fludarabine is used for lymphodepletion. Drug: Cyclophosphamide Cyclophosphamide is used for lymphodepletion. |
Experimental: Dose Level 2 of PBCAR20A CAR T cells
3 x 10^6 CAR T cells per kg body weight.
|
Genetic: PBCAR20A
Single dose of Allogeneic Anti-CD20 CAR T cells will be infused, and a classic "3+3" dose escalation will be applied.
Other Name: Allogeneic Anti-CD20 CAR T cells Drug: Fludarabine Fludarabine is used for lymphodepletion. Drug: Cyclophosphamide Cyclophosphamide is used for lymphodepletion. |
Experimental: Dose Level 3 of PBCAR20A CAR T cells
6 x 10^6 CAR T cells per kg body weight.
|
Genetic: PBCAR20A
Single dose of Allogeneic Anti-CD20 CAR T cells will be infused, and a classic "3+3" dose escalation will be applied.
Other Name: Allogeneic Anti-CD20 CAR T cells Drug: Fludarabine Fludarabine is used for lymphodepletion. Drug: Cyclophosphamide Cyclophosphamide is used for lymphodepletion. |
- Maximum Tolerated Dose (MTD) [ Time Frame: Day 1 - Day 28 ]To determine the maximum tolerated dose (MTD), which is defined as the dose level at which fewer than 33% of patients experience a dose limiting toxicity (DLT) using a 3+3 strategy.
- Number of Participants with Dose Limiting Toxicity(ies) [ Time Frame: 1 year ]To assess adverse events as dose limiting toxicities as defined by the protocol and CTCAE v5.0.
- Objective Response Rate of Patients [ Time Frame: 1 year ]To assess clinical activity as response in B-ALL by the NCCN Guidelines on ALL (NCCN, 2017) and in NHL by the revised Lugano Classification (Cheson et al, 2016), both reported as objective response rate.
- Area Under the Curve [AUC] [ Time Frame: 1 year ]To evaluate Area Under the Curve [AUC] of PBCAR20A in patients tested.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Key Inclusion Criteria*
Criteria for NHL:
- r/r B-cell NHL that is histologically confirmed by archived tumor biopsy tissue from the last relapse and corresponding pathology report.
- Measurable or detectable disease according to the Lugano classification.
- Primary refractory disease or r/r disease after a response to 2 prior regimens.
Criteria for CLL/SLL:
- Diagnosis of CLL with indication for treatment based on the iwCLL guidelines and clinically measurable disease or SLL with measurable disease that is biopsy-proven SLL.
- Previously failed/tolerant to at least 2 prior lines of systemic targeted therapy of known benefit.
Criteria for both NHL and CLL/SLL:
- Study participant has an Eastern Cooperative Oncology Group (ECOG) Performance Status score of 0 or 1.
- Study participant has adequate bone marrow, renal, hepatic, pulmonary, and cardiac function.
Key Exclusion Criteria*:
Criteria for NHL:
- Requirement for urgent therapy due to mass effects such as bowel obstruction, spinal cord, or blood vessel compression.
- Active central nervous system (CNS) disease. A negative computed tomography (CT)/magnetic resonance imaging (MRI) is required at Screening if the study participant has a history of CNS lymphoma.
Criteria for CLL/SLL:
- Active CNS disease. A negative lumbar puncture is required at Screening if the study participant has a history of CNS disease.
Criteria for NHL and CLL/SLL:
- Previous malignancy, besides the malignancies of inclusion (B-cell NHL or CLL/SLL), that in the investigator's opinion, has a high risk of relapse in the next 2 years.
- Active uncontrolled fungal, bacterial, viral, protozoal, or other infection.
- Any form of primary immunodeficiency.
- History of human immunodeficiency virus (HIV) infection.
- Active hepatitis B or C.
- Uncontrolled cardiovascular disease.
- Hypertension crisis or hypertensive encephalopathy within 3 months prior to Screening.
- Presence of a CNS disorder that renders ineligible for treatment.
- History of a genetic syndrome such as Fanconi anemia, Kostmann syndrome, Shwachman Diamond syndrome, or any other known bone marrow failure syndrome.
- Active hemolytic anemia.
- Received ASCT within 45 days of Screening if the study participant has met the rest of the count requirements.
- Must not have received systemic corticosteroid therapy for at least 1 day prior to initiating lymphodepletion chemotherapy.
- Received a systemic biologic agent within 30 days or 5 half-lives.
- Received a live vaccine within 4 weeks before Screening.
- Received standard cytotoxic chemotherapy within 10 days of Screening.
- Radiotherapy within 4 weeks determined on a case-by-case basis.
- Presence of a pleural/peritoneal/pericardial catheter.
-
Current use of any anticoagulant or antiplatelet therapy.
- Additional criteria apply

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04030195
Contact: Clinical Precision BioSciences, Inc. | 919-314-5512 | clinical@precisionbiosciences.com |
United States, California | |
City of Hope | Recruiting |
Duarte, California, United States, 91010 | |
Contact: Geoffrey Shouse, MD 800-826-4673 | |
Principal Investigator: Geoffrey Shouse, MD | |
Stanford University | Recruiting |
Stanford, California, United States, 94305 | |
Contact: David Miklos, MD 650-498-6000 | |
Principal Investigator: David Miklos, MD | |
United States, New York | |
Columbia University | Recruiting |
New York, New York, United States, 10032 | |
Contact: Ran Reshef, MD 212-305-5098 | |
Principal Investigator: Ran Reshef, MD | |
United States, Ohio | |
Cleveland Clinic | Recruiting |
Cleveland, Ohio, United States, 44195 | |
Contact: Brian Hill, MD, PhD 216-445-9451 | |
Principal Investigator: Brian Hill, MD, PhD | |
United States, Texas | |
MD Anderson Cancer Center | Recruiting |
Houston, Texas, United States, 77030 | |
Contact: Sattva Neelapu, MD 713-792-2860 | |
Principal Investigator: Sattva Neelapu, MD |
Study Chair: | Chris Heery, MD | Chief Medical Officer |
Responsible Party: | Precision BioSciences, Inc. |
ClinicalTrials.gov Identifier: | NCT04030195 |
Other Study ID Numbers: |
PBCAR20A-01 |
First Posted: | July 23, 2019 Key Record Dates |
Last Update Posted: | November 2, 2020 |
Last Verified: | October 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Lymphoma Leukemia Lymphoma, Non-Hodgkin Leukemia, Lymphoid Leukemia, Lymphocytic, Chronic, B-Cell Lymphoma, B-Cell Neoplasms by Histologic Type Neoplasms Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases |
Leukemia, B-Cell Cyclophosphamide Fludarabine Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Antirheumatic Agents Antineoplastic Agents, Alkylating Alkylating Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Myeloablative Agonists |