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

NCT04085159 | Unknown Phase 1

• 1 other identifier: GIMI-IRB-19006
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Cancer

Keywords

immunotherapy T cell cancer gene therapy

Outcome Measures

Primary Outcomes (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.

  up to one month

Secondary Outcomes (3)

• Rate of successful CART/CTL/DCvac cell production

  up to one month

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

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

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

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

Study Arms (1)

CART/CTL/DCvac cells to treat cancer

EXPERIMENTAL

Biological: Antigen-specific T cells CART/CTL and DCvac

Interventions

Antigen-specific T cells CART/CTL and DCvac BIOLOGICAL

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

CART/CTL/DCvac cells to treat cancer

Eligibility Criteria

• Age: 1 Year - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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:
• 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.

Sponsors & Collaborators

• Shenzhen Geno-Immune Medical Institute: lead
• Shenzhen Hospital of Southern Medical University: collaborator
• Shenzhen Children's Hospital: collaborator

Study Sites (3)

Shenzhen Geno-immune Medical Institute

Shenzhen, Guangdong, 518000, China

RECRUITING

Shenzhen Children's Hospital

Shenzhen, Guangdong, 518038, China

RECRUITING

Department of Neurosurgery, Shenzhen Hospital, Southern Medical University

Shenzhen, Guangdong, 518100, China

RECRUITING

MeSH Terms

Conditions

Neoplasms

Interventions

S-1,2-dichlorovinyl-N-acetylcysteine

Central Study Contacts

Lung-Ji Chang, PhD

CONTACT

86-075586725195; c@szgimi.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 9, 2019
• First Posted: September 11, 2019
• Study Start: September 1, 2019
• Primary Completion: January 31, 2021
• Study Completion: December 31, 2022
• Last Updated: June 12, 2020

Record last verified: 2020-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (3)