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

Study Overview
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Brief Summary
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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.

Detailed Description
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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).

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

Conditions
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Progressive Multifocal Leukoencephalopathy

Intervention / Treatment
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* Biological: CE-VST01-JC


Participation Criteria
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Eligibility Criteria
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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).

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: randomized, double- blinded, Phase 2 trial in patients with PML due to JCPyV.
Masking: Triple


Arms and Interventions

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

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the effect of CE-VST01-JC on time to disease progression, as measured by mRS (modified Rankin Score) |  | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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VST, Virus specific T-Cell, cell therapy, PML, JC virus