A Phase I/II Study of Eque-cel in Subjects Withimmune-mediated Necrotizing Myopathy

ConditionRefractory Immune-mediated Necrotizing Myopathy

Trial statusNot yet recruiting

Trial phasePhase 1, Phase 2

Trial typeInterventional

Biological sexAll

Age18+

SponsorNanjing IASO Biotechnology Co., Ltd.

About this trial

This is an open-label, single-arm Phase I/II clinical study to evaluate the efficacy and safety of Eque-cel Injection in patients with refractory immune-mediated necrotizing myopathy.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

1. At the time of signing the informed consent form, the age is ≥ 18 years old.

2.Based on the 2016 ENMC classification criteria, the clinical diagnosis is IMNM.

3.At the time of screening, the anti-SRP antibody or anti-HMGCR antibody is positive.

4. Refractory IMNM that failed or relapsed after standard treatment, and the disease is still active at the time of screening.

Disqualifiers

1. Having end-stage myositis with involvement of terminal organs may pose additional risks or interfere with the study assessment.

2.Presence of irreversible muscle involvement and/or severe atrophy may bring additional risks or interfere with the study assessment.

3.Uncontrolled interstitial lung disease or any other uncontrolled manifestations of IIM, as judged by the investigator, may require the use of prohibited drugs during the study period.

4. Diagnosed with other inflammatory or non-inflammatory myopathies.

Trial design

Design model

Single group

Treatments tested in this trial

Equecabtagene Autoleucel Injection (Eque-cel)
Drug
Eque-cel is a personalized, BCMA-targeted gene-modified autologous T-cell immunotherapy product capable of recognizing and eliminating both malignant and normal cells expressing BCMA. The CAR specifically identifies BCMA via a fully human single-chain variable fragment (scFv) with low immunogenicity, activates, proliferates, secretes cytokines, and kills target cells through the CD3ζ domain, while enhancing CAR-T expansion and persistence via 4-1BB costimulation signaling. Eque-cel demonstrates high affinity for the BCMA antigen both in vitro and in vivo, as well as efficient killing of antigen-loaded cells.

Treatment groups

27 Participants

are divided into 1 treatment group

Group A: Equecabtagene Autoleucel Injection（Eque-cel）Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

Phase I: Safety endpoint - Adverse Events (AEs)

Incidence and severity of adverse events as assessed by NCI-CTCAE v5.0 (except CRS and ICANS assessed according to the criteria of 2019 ASTCT criteria).

Time frame

up to 2 years from Eque-cel Injection infusion

Phase I: Safety endpoint-incidence of Dose-limiting toxicity (DLT)

Percentage of participants who experienced DLT within 28 days after Eque-cel administration.

Time frame

Time Frame: up to 28 days from Eque-cel Injection infusion

Phase II: Efficacy endpoint- TIS response

Proportion of participants achieving TIS response

Time frame

up to the 2 years after Eque-cel Injection infusion

Secondary outcomes

Phase I: Efficacy endpoint -TIS response

Proportion of participants achieving TIS response

Time frame

up to the 2 years after Eque-cel Injection Infusion

Phase II:Efficacy endpoint -Proportion of participants achieving minimal improvement response (TIS ≥ 20 points)

Defined as achieving The Total Improvement Score (TIS)≥ 20 points (minimum improvement)

Time frame

From baseline to 12 months after Eque-cel Injection Infusion

Phase I/II:Efficacy endpoint -Change in mean Total Improvement Score (TIS) value

Defined as achieving the Total Improvement Score (TIS)

Time frame

up to 2 years from Eque-cel Injection infusion

Phase I/II:Efficacy endpoint -The changes in PtGA scores

The PhGA scale will be completed by patients at each study visit.

Time frame

up to 2 years from Eque-cel Injection infusion

Other outcomes

Phase I/II- Immunogenicity

Prevalence and titer of confirmed human anti-CAR antibodies in peripheral blood.

Time frame

up to 2 years from Eque-cel Injection infusion

Phase I/II Replication competent lentivirus (RCL)

Prevalence of replication-competent lentivirus (RCL) in peripheral blood.

Time frame

up to 2 years from Eque-cel Injection infusion

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Nanjing IASO Biotechnology Co., Ltd.

Lead sponsor

Source ClinicalTrials.gov

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