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

Progressive Multifocal Leukoencephalopathy Clinical Trial

— ASCEND-JC  

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05541549
• Other study ID #: 20210001
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: February 2023
• Est. completion date: April 2025

Study information

• Verified date: September 2022
• Source: Cellevolve Bio Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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).

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 60
• Est. completion date: April 2025
• Est. primary completion date: March 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

• 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).

Related Conditions & MeSH terms

• Leukoencephalopathies
• Leukoencephalopathy, Progressive Multifocal
• Progressive Multifocal Leukoencephalopathy

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Cellevolve Bio Inc

Outcome

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