Immunotherapy Based on Antigen-specific Immune Effector Cells Targeting Neurofibromatosis or Schw… (NCT04085159) | Clinical Trial Compass
RecruitingPhase 1/2
Immunotherapy Based on Antigen-specific Immune Effector Cells Targeting Neurofibromatosis or Schwannomatosis
China100 participantsStarted 2026-06-01
Plain-language summary
The primary objective of this study is to verify the safety of antigen-specific T cells (CAR-T) and engineered immune effector cytotoxic T cells (EIE) modified by immunoregulatory genes and immune modified dendritic cell vaccine (DCvac) in the treatment of neurofibromatosis or schwannoma.
Who can participate
Age range
1 Year – 80 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Written, informed consent obtained prior to any study-specific procedures.
. Diagnosis of neurofibromatosis, or schwannomatosis
. The results of immune staining of the patient's cancer specimens positive for any one or more of a list of tumor-associated antigens.
. Age ≥ 1 years
. At least one volumetrically measurable and ≥ 0.5 cc NF-related tumor (schwannoma, ependymoma, meningioma - histological confirmation not required) with radiographic evidence of progression (either as unequivocal progression on conventional MRI, or a \>10% volume increase by 3D volumetrics) over the past ≤12 months, designated as the primary target tumor OR Volumetrically measurable and ≥ 0.5 cc VS with ipsilateral progressive hearing loss over the past ≤12 months, designated as the primary target tumor.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial is testing CART cells, CTL cells, and DCvac cells for neurofibromatosis or schwannomatosis — which of those three immunotherapy approaches would apply to my specific situation, and why?
2Since this is a Phase 1/2 trial and the main thing being measured is the rate of adverse effects after the cell injections, what are the most serious side effects that have been seen with this type of immunotherapy so far, and how would they be managed if they happened to me?
3How does the cell injection process work in practice — where would I need to go, how often, and over what period of time, and is that realistic given my current health and daily life?
4Because the trial is still in an early phase focused on safety rather than proving effectiveness, is there a standard treatment option I should consider first, or is participating in this study a reasonable path at my stage of disease?
5Are there specific features of my tumor — such as its genetic markers or location — that would affect whether I'm a good candidate for this immunotherapy approach, and is that something worth discussing with the research team?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percentage of adverse effects after CART/CTL/DCvac cells injection
. Progressive Hearing Loss Criteria for Enrollment: Audiogram showing drop in pure tone average (PTA) of 10dB HL at ≥ 2 nonconsecutive or consecutive frequencies or drop in speech discrimination score (SDS) below the 95% critical difference threshold, compared to previous audiogram ≤ 1 year prior.
. Karnofsky/Lansky performance status (PS) 50-100%. Note: Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
. Any neurologic deficits must be stable for ≥ 1 week.
Exclusion criteria
. The results of immune staining of the patient's tumor-associated antigens are all negative.
. Participation in any other cell therapy protocols within one year.
. Current or recent treatment (within the 28-day period prior to Day 0) with another investigational drug.
. Patients who have had a major surgery or significant traumatic injury within 4 weeks of start of study.
. Pregnant or lactating females.
. Uncontrolled brain or leptomeningeal metastases, including patients who continue to require glucocorticoids for brain or leptomeningeal metastases.
. Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as: