A Phase 2 Study Evaluating JCPyV-specific T Cell Therapy for PML (ASCEND-JC)
Primary Purpose
Progressive Multifocal Leukoencephalopathy
Status
Not yet recruiting
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
CE-VST01-JC
Sponsored by
About this trial
This is an interventional treatment trial for Progressive Multifocal Leukoencephalopathy focused on measuring VST, Virus specific T-Cell, cell therapy, PML, JC virus
Eligibility 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).
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Placebo Comparator
Experimental
Arm Label
Placebo
CE-VST01-JC
Arm Description
Outcomes
Primary Outcome Measures
To evaluate the effect of CE-VST01-JC on time to disease progression, as measured by mRS (modified Rankin Score)
Secondary Outcome Measures
Full Information
NCT ID
NCT05541549
First Posted
September 13, 2022
Last Updated
September 14, 2022
Sponsor
Cellevolve Bio Inc
1. Study Identification
Unique Protocol Identification Number
NCT05541549
Brief Title
A Phase 2 Study Evaluating JCPyV-specific T Cell Therapy for PML
Acronym
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
Study Type
Interventional
2. Study Status
Record Verification Date
September 2022
Overall Recruitment Status
Not yet recruiting
Study Start Date
February 2023 (Anticipated)
Primary Completion Date
March 2024 (Anticipated)
Study Completion Date
April 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cellevolve Bio Inc
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Progressive Multifocal Leukoencephalopathy
Keywords
VST, Virus specific T-Cell, cell therapy, PML, JC virus
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
randomized, double- blinded, Phase 2 trial in patients with PML due to JCPyV.
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
60 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Title
CE-VST01-JC
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
CE-VST01-JC
Intervention Description
CE-VST01-JC at a dose of 1 × 10^8 cells administered as an intravenous (IV) infusion every 28 days for 4 total infusions
Primary Outcome Measure Information:
Title
To evaluate the effect of CE-VST01-JC on time to disease progression, as measured by mRS (modified Rankin Score)
Time Frame
1 year
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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).
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Phase 2 Study Evaluating JCPyV-specific T Cell Therapy for PML
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