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

NCT04690595 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: PMB-101
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia

Keywords

B cell; ALL; relapsed or refractory

Outcome Measures

Primary Outcomes (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.

  Up to 1 year post treatment

Secondary Outcomes (6)

• Disease response

  Up to 1 year post treatment

• Minimal residual disease (MRD)

  Up to 1 year post treatment

• B cell frequency

  Up to 1 year post treatment

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

  Up to 1 year post treatment.

• Progression-free survival (PFS)

  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.

Study Arms (1)

BAFFR-CAR T cells

EXPERIMENTAL

B-cell activating factor receptor-Chimeric antigen receptor T cells

Biological: BAFFR-CAR T cells

Interventions

BAFFR-CAR T cells BIOLOGICAL

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

BAFFR-CAR T cells

Eligibility Criteria

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

You may qualify if:

• 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
• 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

You may not qualify if:

• Autologous/allogeneic stem cell transplant within 100 days at the time of enrollment.
• Immunosuppressant medications within 1 months prior to protocol enrollment.
• 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.
• 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.
• a. 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.
• iii. 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.

Sponsors & Collaborators

• PeproMene Bio, Inc.: lead
• City of Hope Medical Center: collaborator

Study Sites (1)

City of Hope Medical Center

Duarte, California, 91010, United States

Location

MeSH Terms

Conditions

Recurrence; Burkitt Lymphoma

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Epstein-Barr Virus Infections; Herpesviridae Infections; DNA Virus Infections; Virus Diseases; Infections; Tumor Virus Infections; Lymphoma, B-Cell; Lymphoma, Non-Hodgkin; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Study Officials

• Ibrahim Aldoss, MD

  City of Hope Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 24, 2020
• First Posted: December 30, 2020
• Study Start: May 18, 2021
• Primary Completion (Estimated): November 18, 2026
• Study Completion (Estimated): November 18, 2027
• Last Updated: February 11, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)