ASCEND-JC: A Phase 2 Study Evaluating JCPyV-specific T Cell Therapy for PML

Sponsor

Cellevolve Bio Inc (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05541549

Collaborator

(none)

Enrollment

Arms

Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

Condition or Disease	Intervention/Treatment	Phase
Progressive Multifocal Leukoencephalopathy	Biological: CE-VST01-JC	Phase 2

Detailed Description

Progressive multifocal leukoencephalopathy (PML) is a demyelinating disease of the central nervous system caused by JC polyomavirus (JCPyV) occurring in immunocompromised patients who face a disease that is usually progressive and often fatal (Padgett 1971; Tan 2010). Annually, it is estimated that 4,000 people develop PML in the United States and Europe combined (NORD 2015).

This is a randomized, double- blinded, Phase 2 trial in patients with PML due to JCPyV. Patients will receive treatment with a matched virus-specific T-cell product (CE VST01-JC) or placebo, and will then be monitored for response to therapy. The study is designed to evaluate whether CE-VST01-JC infusions will slow and ultimately halt neurological progression in patients with PML compared with placebo as evaluated by modified Rankin Score (mRS).

Study Design

Study Type:

Interventional

Anticipated Enrollment :

60 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

randomized, double- blinded, Phase 2 trial in patients with PML due to JCPyV.randomized, double- blinded, Phase 2 trial in patients with PML due to JCPyV.

Masking:

Triple (Participant, Care Provider, Investigator)

Primary Purpose:

Treatment

Official Title:

ASCEND-JC: A Multi-center, Randomized, Double-blind, Phase 2 Study, Evaluating JCPyV-specific T Cell Therapy for the Treatment of PML

Anticipated Study Start Date :

Feb 1, 2023

Anticipated Primary Completion Date :

Mar 1, 2024

Anticipated Study Completion Date :

Apr 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
Placebo Comparator: Placebo	Biological: CE-VST01-JC CE-VST01-JC at a dose of 1 × 10^8 cells administered as an intravenous (IV) infusion every 28 days for 4 total infusions
Experimental: CE-VST01-JC	Biological: CE-VST01-JC CE-VST01-JC at a dose of 1 × 10^8 cells administered as an intravenous (IV) infusion every 28 days for 4 total infusions

Arm

Intervention/Treatment

Placebo Comparator: Placebo

Biological: CE-VST01-JC

CE-VST01-JC at a dose of 1 × 10^8 cells administered as an intravenous (IV) infusion every 28 days for 4 total infusions

Experimental: CE-VST01-JC

Biological: CE-VST01-JC

CE-VST01-JC at a dose of 1 × 10^8 cells administered as an intravenous (IV) infusion every 28 days for 4 total infusions

Outcome Measures

Primary Outcome Measures

To evaluate the effect of CE-VST01-JC on time to disease progression, as measured by mRS (modified Rankin Score) [1 year]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Male or female subjects aged 18 years or older, with actively progressing PML confirmed by MRI and JCPyV present in CSF or in brain biopsy tissue (diagnosis confirmed according to algorithm diagnostic criteria for PML developed by the American Academy of Neurology).