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

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

Status

UNKNOWN

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05541549

Acronym

ASCEND-JC

Enrollment

Registered

2022-09-15

Start date

2023-02-28

Completion date

2025-04-30

Last updated

2022-09-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Progressive Multifocal Leukoencephalopathy

Keywords

VST, Virus specific T-Cell, cell therapy, PML, JC virus

Brief summary

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

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).

Interventions

BIOLOGICALCE-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

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

TRIPLE (Subject, Caregiver, Investigator)

Intervention model description

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

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* 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).

Design outcomes

Primary

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

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026