GD2 CAR T Cells in Diffuse Intrinsic Pontine Gliomas(DIPG) & Spinal Diffuse Midline Glioma(DMG)


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Trial ID: NCT04196413


The primary purpose of this study is to test whether GD2-CAR T cells can be successfully made from immune cells collected from children and young adults with H3K27M-mutant diffuse intrinsic pontine glioma (DIPG) or spinal H3K27M-mutant diffuse midline glioma (DMG). H3K27Mmutant testing will occur as part of standard of care prior to enrollment.

Official Title

Phase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor (CAR) T Cells (GD2CART) for Diffuse Intrinsic Pontine Gliomas (DIPG) and Spinal Diffuse Midline Glioma (DMG)

Stanford Investigator(s)

Susan Hiniker
Susan Hiniker

Associate Professor of Radiation Oncology (Radiation Therapy)

Paul Graham Fisher, MD
Paul Graham Fisher, MD

Beirne Family Professor of Pediatric Neuro-Oncology, Professor of Pediatrics and, by courtesy, of Neurosurgery and of Epidemiology and Population Health

Jasia Mahdi

Clinical Instructor, Neurology

Laura Prolo

Assistant Professor of Neurosurgery


International patients are not currently eligible to enroll.

Inclusion Criteria:

   - Currently accepting US patients only

   - Disease Status:

   - Tissue diagnosis of H3K27M-mutated Diffuse Intrinsic Pontine Glioma (DIPG) with
   radiographically evident tumor restricted to the brainstem, OR

   - Tissue diagnosis of H3K27M-mutated Diffuse Midline Glioma (DMG) of the spinal cord

   - Age: Greater than or equal to 2 year of age and less than or equal to 50 years of age

Prior Therapy:

   - At least 6 weeks following completion of front line radiation therapy.

   - At least 3 weeks post chemotherapy or 5 half-lives, whichever is shorter must have
   elapsed since any prior systemic therapy, except for systemic inhibitory/stimulatory
   immune checkpoint therapy, which requires 5 half-lives.

   - Performance Status: Subjects > 16 years of age: Karnofsky ≥ 60% OR Eastern Cooperative
   Oncology Group (ECOG) performance status of 0 or 1; Subjects ≤ 16 years of age: Lansky
   scale ≥ 60%

   - Normal Organ and Marrow Function (supportive care is allowed per institutional
   standards, i.e. filgrastim, transfusion)

      1. Absolute neutrophil count (ANC) ≥ 1,000/uL

      2. Platelet count ≥ 100,000/uL

      3. Absolute lymphocyte count ≥ 150/uL

      4. Hemoglobin ≥ 8 g/dL

      5. Adequate renal, hepatic, pulmonary and cardiac function defined as:

         - Creatinine within institutional norms for age (i.e. ≤ 2 mg/dL in adults or
         according to table below in children <18 years) OR creatinine clearance (as
         estimated by Cockcroft Gault Equation) ≥ 60 mL/min

         - Age (Years) -- Maximum Serum Creatinine (mg/dL)

         - ≤5 Years ---------------- 0.8mg/dL

         - 5 < age ≤ 10 Years ----1.0mg/dL

         - >10-18 Years -----------1.2mg/dL

         - >18 Years -----------2.0mg/dL

         - Serum Alanine aminotransferase( ALT)/Aspartate Aminotransferase (AST) ≤ 3.0
         Upper limit of normal (ULN )(grade 1)

         - Total bilirubin ≤ 1.5 mg/dl, except in subjects with Gilbert's syndrome.

         - Cardiac ejection fraction ≥ 45%, no evidence of physiologically significant
         pericardial effusion as determined by an echocardiogram (ECHO), and no
         clinically significant ECG findings

         - Baseline oxygen saturation > 92% on room air

   - Pregnancy Test Females of childbearing potential must have a negative serum or urine
   pregnancy test (females who have undergone surgical sterilization are not considered
   to be of childbearing potential).

   - Contraception Subjects of child bearing or child fathering potential must be willing
   to practice birth control from the time of enrollment on this study and for four (4)
   months after receiving the preparative lymphodepletion regimen or for as long as
   GD2-CAR T cells are detectable in peripheral blood or cerebrospinal fluid (CSF).

   - Ability to give informed consent. Pediatric subjects will be included in age
   appropriate discussion and verbal assent will be obtained for those > 7 years of age,
   when appropriate.

Exclusion Criteria:

   - Tumor involvement of cerebellar vermis or hemispheres (pontocerebellar peduncle
   involvement is allowed), thalamic lesions, or supratentorial lesions.

   - Clinically significant swallowing dysfunction/dysphagia or prominent medullary
   dysfunction as judged by clinical assessment.

   - Current systemic corticosteroid therapy

   - Prior CAR therapy.

   - Prior GD2-antibody therapy

   - Ongoing use of dietary supplements, alternative therapies or extreme diets or any
   medication not approved by the investigators

   - Uncontrolled fungal, bacterial, viral, or other infection. Previously diagnosed
   infection for which the patient continues to receive antimicrobial therapy is
   permitted if responding to treatment and clinically stable.

   - Ongoing infection with HIV or hepatitis B (HBsAg positive) or hepatitis C virus (anti
   Hepatitis C Virus (HCV) positive). A history of hepatitis B or hepatitis C is
   permitted if the viral load is undetectable per quantitative polymerase chan reaction
   (PCR) and/or nucleic acid testing.

   - Clinically significant systemic illness or medical condition (e.g. significant
   cardiac, pulmonary, hepatic or other organ dysfunction), that in the judgement of the
   principal investigator is likely to interfere with assessment of safety or efficacy of
   the investigational regimen and its requirements.

   - In the investigator's judgment, the subject is unlikely to complete all protocol
   required study visits or procedures, including follow up visits, or comply with the
   study requirements for participation.

   - Known sensitivity or allergy to any agents/reagents used in this study.

   - Primary immunodeficiency or history of autoimmune disease (e.g. Crohns, rheumatoid
   arthritis, systemic lupus) requiring systemic immunosuppression/systemic disease
   modifying agents within the last 2 years


drug: GD2 CAR T cells

drug: Fludarabine

drug: Cyclophosphamide


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Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305

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