Study of NXC-201 CAR-T in Patients With Light Chain (AL) Amyloidosis
ConditionLight Chain (AL) Amyloidosis
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-120
SponsorNexcella Inc.
About 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).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
≥18 years of age.
Voluntarily signed informed consent form (ICF).
Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
Histologically proven systemic AL amyloidosis confirmed by positive Congo red staining with green birefringence on polarized light microscopy in an organ outside the bone marrow and evidence of a measurable clonal plasma cell disease that requires active treatment.
Disqualifiers
Prior treatment with CAR T therapy directed at any target.
Any therapy that is targeted to BCMA.
Stroke or seizure within 6 months of signing ICF.
Bone marrow plasma cells >30% and clinically symptomatic multiple myeloma with end organ damage (i.e. lytic bone lesions).
Trial design
Design model
Sequential
Treatments tested in this trial
NXC-201 CAR-T
Biological/VaccineNXC-201 (formerly HBI0101) CAR-T is defined as autologous T cells transduced ex-vivo with anti-BCMA CAR retroviral vector encoding the chimeric antigen receptor (CAR) targeted to human BCMA. The NXC-201 CAR-T is provided fresh without cryopreservation.
Treatment groups
40 Participants
are divided into 1 treatment groupGroup A: NXC-201 CAR-TExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Number of Participants With Treatment-Related Adverse Events
An adverse event (AE) can be any unfavorable and unintended sign (including an abnormal. laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not considered related to the medicinal (investigational) product.
Time frame
24 months2
Number of Participants with Adverse Events by Severity as Assessed by CTCAE v5.0
An assessment of severity grade will be made according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0, with the exception of cytokine release syndrome (CRS), and immune effector cell-associated neurotoxicity syndrome (ICANS). CRS and ICANS should be evaluated according to the American Society for Transplantation and Cellular Therapy (ASTCT) consensus grading.
Time frame
24 months3
To confirm the maximum tolerated dose (MTD)
According to Common Terminology Criteria for Adverse Events (CTCAE) criteria, version 5.0, and Cytokine release syndrome (CRS) and Immune effector cell-associated neurotoxicity syndrome (ICANS) per American Society for Transplantation and Cellular Therapy (ASTCT) Consensus Grading
Time frame
24 months4
To confirm the recommended phase 2 dose (RP2D)
According to Common Terminology Criteria for Adverse Events (CTCAE) criteria, version 5.0, and Cytokine release syndrome (CRS) and Immune effector cell-associated neurotoxicity syndrome (ICANS) per American Society for Transplantation and Cellular Therapy (ASTCT) Consensus Grading
Time frame
24 monthsSecondary outcomes
1
Percentage of participants with hematologic and organ response
According to consensus recommendations for AL amyloidosis treatment response criteria in AL (Palladini, 2012)
Time frame
24 monthsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Nexcella Inc.
Lead sponsor
Immix Biopharma, Inc.
Collaborator
Source ClinicalTrials.gov