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Relapsed or Refractory B-cell Acute Lymphoblastic LeukemiaPhase 1NCT04690595

BAFFR Targeting CAR-T Cells for the Treatment of Relapsed or Refractory B-cell ALL

A Phase 1 study testing BAFFR-CAR T cells in people with Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia, run by PeproMene Bio, Inc. at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

A Phase 1 Study Evaluating BAFFR-targeting CAR T Cells for Patients with Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
BAFFR-CAR T cells
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
1 siteUnited States
Study dates
18 May 2021to 18 Nov 2027
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Documented informed consent of the participant and/or legally authorized representative.
  • Agreement to allow the use of archival tissue from diagnostic tumor biopsies. If unavailable, exceptions may be granted with study PI approval.
  • Age ≥ 18 years.
  • ECOG ≤ 2.
  • Life expectancy ≥ 16 weeks.
  • Histologically confirmed B-ALL or B-cell lymphoblastic lymphoma
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  • Relapsed/refractory disease after failure of ≥ 2 prior lines of therapy.
  • Evidence of active BAFF-R expression at the time of enrollment.
  • Recovered to ≤ Grade 1 from the acute toxic effects (except alopecia) of prior anti-cancer therapy.
  • No known contraindications to leukapheresis, steroids or tocilizumab.
  • Ineligible for or failed prior CD19-targeted immunotherapy (e.g., blinatumomab or CD19-CAR T cells).
  • For participants who had prior CD19-CAR T cell therapy:
  • At least 90-days has elapsed since participant received last CD19-CAR T cell therapy.
  • AND
  • Persistence of prior CD19-CAR T cells must be evaluated and found to be <5% prior to leukapheresis procedure
  • Participants with CNS involvement by leukemia (CNS2 and asymptomatic CNS3) may be considered eligible after discussions with the study team.
  • Total serum bilirubin ≤ULN (unless has Gilbert's disease, then ≤3.0)
  • AST ≤ ULN
  • ALT ≤ ULN
  • Creatinine clearance of ≥ 40 mL/min per 24-hour urine test or the Cockcroft-Gault formula
  • Left ventricular ejection fraction (LVEF) ≥ 50%
  • O2 saturation ≥ 92% on room air.
  • Seronegative for HIV Ag/Ab combo, HCV*, and active HBV (Surface Antigen Negative)
  • If positive, Hepatitis C RNA quantitation must be performed and must be undetectable.
  • Women of childbearing potential (WOCBP): negative urine or serum pregnancy test If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required.
  • QuantiFERON-TB Gold or equivalent*
  • Results do not impact patient eligibility, however the test must be initiated prior to enrollment
  • Agreement by females and males of childbearing potential^ to use an effective method of birth control or abstain from heterosexual activity for the course of the study through at least 3 months after the last dose of protocol therapy.
  • ^Childbearing potential defined as not being surgically sterilized (men and women) or have not been free from menses for > 1 year (women only).
  • A complete liver evaluation (which includes ultrasound elastography, MRI of the liver and a hepatology consult) may be done if needed based on PI's recommendation.
  • Evaluation of acquired hemochromatosis (indicated through MRI of the liver and elevated levels of Ferritin) is recommended for participants who have undergone multiple transfusions and prior alloHCT.

Who cannot join

  • Autologous/allogeneic stem cell transplant within 100 days at the time of enrollment.
  • Immunosuppressant medications within 1 months prior to protocol enrollment.
  • Concurrent use of systemic steroids or chronic use of immunosuppressant medications. Recent or current use of inhaled steroids is not exclusionary. Physiologic replacement of steroids (prednisone ≤ 7.5 mg /day or equivalent) is allowed
  • Auto-immune disease or active GVHD within 4 months prior to protocol enrollment requiring systemic immunosuppressant therapy.
  • Class III/IV cardiovascular disability according to the New York Heart Association (NYHA) Classification.
  • Subjects with clinically significant arrhythmia or arrhythmias not stable on medical management within 2 weeks of enrollment.
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  • Any abnormal liver enzyme levels (as defined ≥ULN in ALT, AST, Bilirubin and Alkaline Phosphatase levels) at time of enrollment
  • . Subjects with a known history or prior diagnosis of optic neuritis or other immunologic or inflammatory disease affecting the central nervous system, including seizure disorder.
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to study agent.
  • Known bleeding disorders (e.g., von Willebrand's disease) or hemophilia.
  • History of venous occlusive disease (VOD), or GvHD.
  • Subjects with a history of the following GvHD may still be included in the study: i. Resolved Grade 2 or less steroid-sensitive acute skin GvHD ii. Grade 1 gastro-intestinal (GI)-GvHD developed within 100 days post prior alloHCT.
  • Limited chronic GvHD
  • History of stroke or intracranial hemorrhage within 6 months of enrollment.
  • History of other malignancies, except for malignancy surgically resected (or treated with other modalities) with curative intent, basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin; non-muscle invasive bladder cancer; malignancy treated with curative intent with no known active disease present for ≥ 3 years.
  • Clinically significant uncontrolled illness.
  • Active systemic uncontrolled infection requiring antibiotics.
  • Known history of immunodeficiency virus (HIV) or hepatitis B or hepatitis C infection.
  • Females only: Pregnant or breastfeeding.
  • Any other condition that would, in the investigator's judgment, contraindicate the subject's participation in the clinical study due to safety concerns with clinical study procedures.
  • Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics).

Trial locations and contacts

1 site in 1 country.

United States1 site

  • City of Hope Medical Center

    Duarte, California, 91010

Other trials in United States

About this study

Official title: A Phase 1 Study Evaluating BAFFR-targeting CAR T Cells for Patients With Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia

A Phase 1 Study Evaluating BAFFR-targeting CAR T Cells for Patients with Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia

Read the detailed description

This phase I trial evaluates the side effects and best dose of BAFFR-CAR T cells in treating patients with B-cell Acute Lymphoblastic Leukemia that has come back (recurrent) or does not respond to treatment (refractory). T cells are infection fighting blood cells that can kill cancer cells. The T cells given in this study will come from the patient and will have a new gene put in them that makes them able to recognize BAFFR, a protein on the surface of cancer cells. These BAFFR-specific T cells may help the body's immune system identify and kill BAFFR+ cancer cells.

Being tested
BAFFR-CAR T cells
Keywords
B cell, ALL, relapsed or refractory
Medical subject headings
Recurrence, Burkitt Lymphoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BAFFR-CAR T cellsBiological

    First-in-human trial examining the safety and preliminary efficacy of BAFFR-CAR T cells in participants with r/r B-ALL

Study groups in NCT04690595
Study groupRoleWhat they receive
BAFFR-CAR T cellsExperimentalB-cell activating factor receptor-Chimeric antigen receptor T cells
Receives: Biological: BAFFR-CAR T cells

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of adverse events

    Toxicity will be graded per Common Terminology Criteria for Adverse Events version 5.0, Cytokine Release Syndrome (CRS) and neurotoxicity which use the American Society for Transplantation and Cellular Therapy Consensus Criteria (ASTCT) and Graft versus Host Disease (GVHD) criteria. Toxicities will be followed from the start of lymphodepletion until the end of the study.

    Measured: Up to 1 year post treatment

Secondary outcomes (6)

  1. Disease response

    Defined as complete response [CR], or complete response with incomplete blood count recovery [CRi], or complete response with partial hematological recovery [CRh]. Response will be evaluated using European Leukemia Net (ELN) criteria. Rates and associated 95% binomial exact confidence limits will be estimated (CR/CRi/CRh) rate.

    Measured: Up to 1 year post treatment

  2. Minimal residual disease (MRD)

    Negative MRD is defined by malignant cells < 0.01% by flow cytometry or clonoSEQ.

    Measured: Up to 1 year post treatment

  3. B cell frequency

    Measured by serum IgG level

    Measured: Up to 1 year post treatment

  4. Severity of graft-versus-host disease (GVHD) in recipients of prior allogeneic hematopoietic stem cell transplantation

    Defined per Keystone criteria for acute GVHD and revised National Institute of Health (NIH) consensus on grading of chronic GVHD.

    Measured: Up to 1 year post treatment.

  5. Progression-free survival (PFS)

    Kaplan-Meier product limit method with log-log transformation for the confidence interval will be used to estimate PFS.

    Measured: From T cell infusion to the first observation of disease relapse/progression or death from any cause, whichever occurs first, assessed up to 15 years.

  6. Overall survival (OS)

    Kaplan-Meier product limit method with log-log transformation for the confidence interval will be used to estimate OS.

    Measured: From the day of BAFFR-CAR T cell infusion to death from any cause assessed, up to 15 years.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Dec 2020
  2. Study start18 May 2021
  3. Primary completion18 Nov 2026 (estimated)
  4. Study completion18 Nov 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
24 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04690595
Sponsor's protocol ID
PMB-101
Status last verified
Feb 2026
First submitted
24 Dec 2020
Last updated
11 Feb 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing BAFFR-CAR T cells in people with Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia, run by PeproMene Bio, Inc. at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 31 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 11 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of adverse events (time frame: Up to 1 year post treatment). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04690595.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Feb 2026.

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