40 Participants Needed

NXC-201 CAR-T for Amyloidosis

(NEXICART-2 Trial)

Recruiting at 3 trial locations
MA
N
Overseen ByNexcella
Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: Nexcella Inc.
Must be taking: CD38 monoclonal antibody
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 2 JurisdictionsThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial requires a washout period of at least 4 weeks from any previous investigational treatment and no systemic therapy for AL amyloidosis within 14 days prior to leukapheresis. Additionally, therapeutic doses of steroids are not allowed within 2 weeks prior to leukapheresis.

What data supports the effectiveness of the treatment NXC-201 CAR-T for Amyloidosis?

Research shows that CAR T cell therapy, which is a type of treatment that uses modified immune cells to target cancer cells, has been effective in treating conditions like multiple myeloma and amyloidosis. In some cases, patients with amyloidosis who received CAR T therapy targeting specific proteins showed good responses and manageable side effects.12345

Is NXC-201 CAR-T therapy safe for humans?

The safety of CAR-T therapy, similar to NXC-201, has been studied in patients with AL amyloidosis and multiple myeloma, showing that it can be tolerated with manageable side effects when patients are carefully selected and their heart and kidney functions are optimized before treatment.16789

What makes the NXC-201 CAR-T treatment unique for amyloidosis?

NXC-201 CAR-T treatment is unique for amyloidosis because it uses a novel approach of targeting specific proteins on the surface of B cells, which are involved in the disease process, potentially offering a new treatment option for patients with limited existing therapies.1371011

What is the purpose of this trial?

Open-label Phase 1b Dose Escalation/Dose Expansion study exploring the safety and efficacy of NXC-201 in patients with relapsed or refractory light chain amyloidosis (AL).

Research Team

Mehrdad Abedi, M.D. for UC Davis Health

Mehrdad Abedi, MD

Principal Investigator

University of California, Davis

Eligibility Criteria

This trial is for adults with relapsed or refractory light chain amyloidosis who have had previous treatments. They must not be pregnant, agree to birth control, and have measurable disease. People with inadequate organ function, recent other therapies, certain blood disorders, active infections or second malignancies are excluded.

Inclusion Criteria

I have recovered from previous treatment side effects, except for hair loss and severe nerve pain.
Ability and willingness to adhere to the study visit schedule and all protocol requirements
My cancer can be measured by blood tests.
See 6 more

Exclusion Criteria

Significant co-morbid condition or disease which in the judgment of the Investigator would place the subject at undue risk or interfere with the study
I have had treatment for AL amyloidosis before certain medical procedures.
My blood clotting tests are within safe limits, or I'm on stable blood thinners.
See 18 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Leukapheresis

Subjects undergo leukapheresis to provide starting material for NXC-201 CART manufacture

1 day
1 visit (in-person)

Lymphodepletion

Subjects receive Cyclophosphamide and Fludarabine infusions for lymphodepletion

3 days
3 visits (in-person)

Treatment

NXC-201 CART is administered to subjects after lymphodepletion

1 day
1 visit (in-person)

Dose Escalation and Expansion

Subjects receive escalating doses of NXC-201 CAR-positive T cells, guided by safety review committee

24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 months

Treatment Details

Interventions

  • NXC-201
Trial Overview The study tests NXC-201 CAR-T cells' safety and effectiveness in treating AL amyloidosis that has returned or resisted treatment. It's an early-phase trial where the dose of NXC-201 will be increased gradually to find the right balance between efficacy and safety.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: NXC-201 CAR-TExperimental Treatment1 Intervention
The dose escalation phase will include the following doses: Cohort 1 - 150×10\^6 CAR-positive (CAR+) T cells (3 patients) Cohort 2 - 450×10\^6 CAR-positive (CAR+) T cells (3 patients) The dose expansion phase will then proceed.

NXC-201 is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as NXC-201 for:
  • None approved yet; under investigation for AL Amyloidosis and Multiple Myeloma
🇪🇺
Approved in European Union as NXC-201 for:
  • None approved yet; under investigation for AL Amyloidosis and Multiple Myeloma

Find a Clinic Near You

Who Is Running the Clinical Trial?

Nexcella Inc.

Lead Sponsor

Trials
2
Recruited
200+

Immix Biopharma, Inc.

Industry Sponsor

Trials
2
Recruited
100+

Findings from Research

Two patients with AL amyloidosis and concurrent multiple myeloma successfully underwent anti-BCMA CAR T cell therapy, demonstrating that careful patient selection and optimization of cardiac and renal function can lead to manageable toxicities.
Both patients achieved a minimal residual disease (MRD)-negative state, suggesting that CAR T therapy can be effective even in those with underlying organ dysfunction, which was previously a concern in this population.
Anti-B Cell Maturation Antigen Chimeric Antigen Receptor T Cell Therapy for the Treatment of AL Amyloidosis and Concurrent Relapsed/Refractory Multiple Myeloma: Preliminary Efficacy and Safety.Das, S., Ailawadhi, S., Sher, T., et al.[2023]
In a study of 345 patients with light chain amyloidosis (AL) who achieved normal free light chain ratios after treatment, those with elevated involved free light chains (hiFLC) had significantly worse outcomes, including lower progression-free survival (PFS) and overall survival (OS).
Patients with hiFLC after initial therapy showed higher rates of multi-organ involvement and advanced disease stage, indicating that persistent elevation of involved free light chains is a poor prognostic factor, even in patients who initially respond to treatment.
Impact of involved free light chain (FLC) levels in patients achieving normal FLC ratio after initial therapy in light chain amyloidosis (AL).Tandon, N., Sidana, S., Dispenzieri, A., et al.[2018]
A 71-year-old male with systemic AL kappa amyloidosis and marginal zone lymphoma successfully received third-generation CAR T cell therapy targeting CD19, demonstrating that this approach can be effective for treating AL amyloidosis associated with B-cell malignancies.
The treatment was well tolerated, with only mild early toxicities, and resulted in a significant reduction in IgM and kappa light chains, indicating a deep hematological response six months post-treatment.
First third-generation CAR T cell application targeting CD19 for the treatment of systemic IgM AL amyloidosis with underlying marginal zone lymphoma.Korell, F., Schönland, S., Schmitt, A., et al.[2023]

References

Anti-B Cell Maturation Antigen Chimeric Antigen Receptor T Cell Therapy for the Treatment of AL Amyloidosis and Concurrent Relapsed/Refractory Multiple Myeloma: Preliminary Efficacy and Safety. [2023]
Impact of involved free light chain (FLC) levels in patients achieving normal FLC ratio after initial therapy in light chain amyloidosis (AL). [2018]
First third-generation CAR T cell application targeting CD19 for the treatment of systemic IgM AL amyloidosis with underlying marginal zone lymphoma. [2023]
Bortezomib-based induction for transplant ineligible AL amyloidosis and feasibility of later transplantation. [2018]
Delineation of the timing of second-line therapy post-autologous stem cell transplant in patients with AL amyloidosis. [2021]
High-dose melphalan and autologous peripheral blood stem cell transplantation in patients with AL amyloidosis and cardiac defibrillators. [2020]
The Cardiac Amyloidosis Registry Study (CARS): Rationale, Design and Methodology. [2023]
Incidence and risk factors for pacemaker implantation in light-chain and transthyretin cardiac amyloidosis. [2022]
Hematopoietic cell transplantation for primary systemic amyloidosis: what have we learned. [2019]
10.United Statespubmed.ncbi.nlm.nih.gov
Target Mediated Drug Disposition Model of CPHPC in Patients with Systemic Amyloidosis. [2018]
Beyond Andromeda: Improving Therapy for Light Chain Amyloidosis. [2023]
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