Clinical Trial: Study of NXC-201 CAR-T in Patients With Light Chain (AL) Amyloidosis

Study Status: NOT_YET_RECRUITING
Recruit Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL

Official Title: Phase 1b Dose Expansion Study of NXC-201 for the Treatment of Patients With Relapsed or Refractory AL Amyloidosis

Brief Summary: 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).

Detailed Summary:

Building on the prior NXC-201 results in AL amyloidosis published by Kfir-Erenfeld et.
al (2022) and Asherie et.
al. (2023), this study will enroll additional patients with relapsed or refractory AL amyloidosis and assess the safety and efficacy of NXC-201.

Subjects with relapsed/refractory AL amyloidosis will undergo leukapheresis at least one month prior to lymphodepletion, to provide starting material for NXC-201 CART manufacture.
Subjects will be treated according to the following process for lymphodepletion: Days -5, -4 and -3 Cyclophosphamide 250mg/m2, IV infusion over 30 mins, followed immediately by Fludarabine 25 mg/m2 IV infusion over 30 minutes.

NXC-201 CART is administered on Day 0, after lymphodepletion.

Enrolled subjects will receive a dose of NXC-201 CAR-positive (CAR+) T cells as follows:

Dose Escalation phase: Cohort 1 450 � 106 CAR+ T cells with 30-45 days of safety follow-up per patient (3 patients) Cohort 2 800 � 106 CAR+ T cells with 30-45 days of safety follow-up per patient (3 patients) The dose expansion phase (Phase 1b expansion) will enroll 3 or more additional patients at the same 800 � 106 dose of CAR+ T cells if approved by DSMB.

Sponsor: Nexcella Inc.

Current Primary Outcome: 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.

Original Primary Outcome: 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.

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Nexcella Inc.

Dates:
Date Received: October 14, 2023
Date Started: January 01, 2024
Date Completion: January 01, 2024
Last Updated: October 19, 2023
Last Verified: October 01, 2023