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AzTrials

CancerPhase 1/2NCT04085159

Immunotherapy Based on Antigen-specific Immune Effector Cells Targeting Neurofibromatosis or Schwannomatosis

A Phase 1/2 study testing Antigen-specific T cells CART/CTL and DCvac in people with Cancer, run by Shenzhen Geno-Immune Medical Institute at 1 site in China.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 1 year to 80 years
Conditions the study is for
Cancer
What is being tested?
Antigen-specific T cells CART/CTL and DCvac
Could I receive a placebo?
No, everyone receives an active treatment
How many people
100 participants
Where
1 siteChina
Study dates
1 Jun 2026to 31 Jul 2026
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 80 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.
  • 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.
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  • 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.
  • Adequate bone marrow reserve with
  • absolute neutrophil count (ANC) ≥ 1000/mm3.
  • Platelets ≥100,000/mm3.
  • Adequate renal and hepatic function with
  • Serum creatinine ≤ 2 x upper limit of normal (ULN).
  • Serum bilirubin ≤ 2 x ULN.
  • aspartate aminotransferase (AST)/ALT ≤ 2 x ULN.
  • Alkaline phosphatase ≤ 5 x ULN.
  • Serum bilirubin 2.0 is acceptable in the setting of known Gilbert's syndrome.

Who cannot join

  • 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.
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  • Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as:
  • Symptomatic congestive heart failure of New York heart Association Class III or IV
  • unstable angina pectoris, symptomatic congestive heart failure, myocardial infarction within 6 months of start of study drug, serious uncontrolled cardiac arrhythmia or any other clinically significant cardiac disease
  • severely impaired lung function as defined as spirometry and DLCO that is 50% of the normal predicted value and/or 02 saturation that is 88% or less at rest on room air
  • uncontrolled diabetes as defined by fasting serum glucose >1.5 x ULN (Note: Optimal glycemic control should be achieved before starting trial therapy.)
  • active (acute or chronic) or uncontrolled severe infections
  • liver disease such as cirrhosis or severe hepatic impairment (Child-Pugh class C).
  • Inadequate bone marrow function:
  • Absolute neutrophil count < 1.0 x 10e9/L.• Platelet count < 100 x 10e9/L.
  • Hb < 9 g/dL.
  • Inadequate liver and renal function:
  • Serum (total) bilirubin > 1.5 x ULN.
  • AST \& ALT > 2.5 x ULN (> 5 x ULN in patients with liver metastases).
  • Alkaline phosphatase > 2.5 x ULN (or > 5 x ULN in case of liver metastases or > 10 x ULN in case of bone metastases).
  • Serum creatinine >2.0 mg/dl (> 177 μmol/L).
  • Urine dipstick for protein uria should be < 2+. Patients with ≥ 2+ proteinuria on dipstick urinalysis at baseline should undergo 24 hour urine collection and must demonstrate < 1 g of protein/24 hr.
  • Subject infected with HIV (HIV antibody positive), Treponema pallidum antibody positive or TB culture positive.

Trial locations and contacts

1 site in 1 country.

China1 site

  • Shenzhen Geno-immune Medical Institute

    Shenzhen, Guangdong, 518000

Other trials in China

About this study

Official title: Immunotherapy Targeting Neurofibromatosis or Schwannomatosis

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.

Read the detailed description

Neurofibromatosis (NF) is caused by a genetic change that tends to develop benign tumors around nerves. NF is a lifelong condition that affects all populations equally, regardless of gender or ethnicity. Neurofibromatosis has been classified into three distinct types: NF1, NF2, and schwannomatosis. The hallmark tumors seen in NF2 are vestibular schwannomas, formerly known as acoustic neuromas. Vestibular schwannomas are benign tumors made up of abnormal Schwann cells, which are the cells that give the nerves the lining and insulation needed to conduct information. Vestibular schwannomas can cause hearing loss in one or both ears, depending on whether the tumors are unilateral or bilateral.

Schwannomatosis is the same type of tumor as that of NF2 patients. Tumors are all related to Schwann cells. There is no cure for NF or schwannomatosis. Surgery is the only clinical method at present, and no drugs have been proved to be effective in the treatment of these tumors.

Adoptive immunotherapy based on cytotoxic T lymphocytes reactive with specific antigens has proven to be effective. In vitro induction of tumor antigen-specific immune cells and engineering of target specific immune cells have great potential for cancer eradication. If CAR-T/CTL immunotherapy is effective, it is expected that Neurofibromatosis or Schwannomatosis tumors should shrink or disappear completely. However, the minimal residual tumor cells or cancer stem cells may exist and cause relapse to other tissues and organs. Follow-up immunotherapy must focus on enhancing the anti-tumor immunity. Therefore, this protocol includes follow-up application of DCvac to prevent recurrence. This study proposes a novel protocol of immunotherapy to evaluate the safety and effectiveness of targeting tumor antigens in patients.

Conditions
Cancer
Being tested
Antigen-specific T cells CART/CTL and DCvac
Keywords
immunotherapy T cell cancer gene therapy
Medical subject headings
Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Antigen-specific T cells CART/CTL and DCvacBiological

    Antigen-specific T cells CART/CTL and DCvac cells to treat cancer

Study groups in NCT04085159
Study groupRoleWhat they receive
CART/CTL/DCvac cells to treat cancerExperimentalNot described
Receives: Biological: Antigen-specific T cells CART/CTL and DCvac

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of adverse effects after CART/CTL/DCvac cells injection

    To assess the safety of autologous CART/CTL/DCvac cells in patients. The percentage of patients who have adverse effects will be evaluated by using the NCI CTCAE V4.0 criteria.

    Measured: up to one month

Secondary outcomes (3)

  1. Rate of successful CART/CTL/DCvac cell production

    The percentage of successful CART/CTL/DCvac cell generation, which is based on products which pass the safety test after standard culture procedures, viable for at least one preparation, will be evaluated.

    Measured: up to one month

  2. Ability of CART/CTL/DCvac cells to induce anti-cancer reaction

    measurement of concentration of tumor associated markers

    Measured: after 1 month from CART/CTL/DCvac cells infusion until 12 months after infusion

  3. Ability of CART/CTL/DCvac cells for anti-tumor reaction

    Objective response (complete response (CR) + partial response (PR)) was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria. CR is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. Partial response is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.

    Measured: after 1 month from CART/CTL/DCvac cells infusion until 24 months after infusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Sep 2019
  2. Study start1 Jun 2026
  3. Primary completion1 Jul 2026
  4. Study completion31 Jul 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
100 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04085159
Sponsor's protocol ID
GIMI-IRB-19006
Status last verified
Aug 2026
First submitted
9 Sep 2019
Last updated
24 Aug 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Antigen-specific T cells CART/CTL and DCvac in people with Cancer, run by Shenzhen Geno-Immune Medical Institute at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 17 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 24 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early. Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of adverse effects after CART/CTL/DCvac cells injection (time frame: up to one month). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04085159.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2026.

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