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New treatment option for Medulloblastoma
Official title GPC2-CAR T Cell Therapy for Relapsed or Refractory Medulloblastoma in Children and Young Adults
ClinicalTrials.gov ID: NCT07087002
What this study is testing
What is GPC2-CAR T cells?
GPC2-CAR T cells is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for medulloblastoma.
Also referred to as GPC2-directed CAR T cells.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a single-site, open-label Phase 1 clinical trial evaluating the feasibility, safety, and preliminary activity of autologous GPC2-targeted chimeric antigen receptor (CAR) T cells administered via intracerebroventricular (ICV) infusion in children and young adults with relapsed or refractory medulloblastoma or other eligible Central Nervous System (CNS) embryonal tumors.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 30
You may be able to join if
- Diagnosis: Histologically confirmed diagnosis of medulloblastoma or other primary CNS embryonal tumor according to 2021 CNS WHO Classification (5th...
- Other acceptable CNS embryonal tumors include:
- Embryonal Tumor with Multilayered Rosettes (ETMR)
- Pineoblastoma
- Atypical Teratoid/Rhabdoid Tumor (ATRT) of the CNS
You likely can't join if
- Any patient with metastatic disease OUTSIDE the CNS.
- Unwilling or unable, in the investigator's judgement, to have a CSF reservoir (Ommaya or Rickham) placed. Does not apply to people who have a...
- Clinical evidence of active/on-going significant increased intracranial pressure (i.e., impending herniation) or uncontrolled seizures.
- Prior receipt of a chimeric antigen receptor (CAR)-based therapy.
- Currently receiving anticoagulation therapy.
- Known history of infection with HIV or hepatitis B (HBsAg positive) or hepatitis C virus (anti-HCV positive). EXCEPTION: A history of hepatitis B or...
See the full eligibility criteria
Who can join
- Diagnosis: Histologically confirmed diagnosis of medulloblastoma or other primary CNS embryonal tumor according to 2021 CNS WHO Classification (5th edition)
- Other acceptable CNS embryonal tumors include:
- Embryonal Tumor with Multilayered Rosettes (ETMR)
- Pineoblastoma
- Atypical Teratoid/Rhabdoid Tumor (ATRT) of the CNS
- CNS neuroblastoma, FOXR2-activated
- CNS Embryonal Tumor NOS
- Recurrent/Refractory Disease: History of relapsed and/or recurrent disease defined as tumor progression or recurrence following initial diagnosis and upfront treatment with curative intent, or failure to achieve disease...
- GPC2 Positive: H-score ≥ 100 by IHC staining performed on the (Prescreening Protocol IRB-78780, PI: Katherine Ryan, DO) at Stanford Clinical Anatomic Pathology Lab for GPC2 from a tumor sample any time since initial...
- Evaluable Disease: Evaluable disease as per radiographic findings and/or positive cerebrospinal fluid cytology within 28 days of enrollment.
- Patients with VP shunts: Patients with pre-existing ventriculo-peritoneal (VP) shunt devices must have a programmable shunt device to enroll on this study. A VP shunt is not a requirement for this study.
- Prior therapy: No limit to the number of prior treatment regimens. Toxicities due to prior therapy must be stable or recovered to ≤ Grade 1 (except for clinically non-significant toxicities such as alopecia, nutritional...
- Age: ≥ 12 months to ≤ 30 years of age at time of enrollment The first 3 people treated with GPC2-CAR T cells must be ≥ 3 years old at time of infusion
- Performance Status: people ≥ 16 years of age must have Karnofsky ≥ 60%. people \< 16 years of age must have Lansky scale 60%; or ECOG performance status ≤ 2 (see Section 11.3).
- Normal Organ and Marrow Function [supportive care is allowed per institutional standards, i.e., filgrastim, transfusion]
- Hemoglobin ≥ 8 g/dL
- Absolute Neutrophil Count (ANC) ≥ 1,000/μL
- Platelet count ≥ 75,000/μL, with no platelet transfusion within 96 hours prior to enrollment
- Absolute lymphocyte count (ALC) ≥ 150/μL
- PT/INR, PTT ≤ 1.5 x ULN for age Adequate renal, hepatic, cardiac, and pulmonary function defined as:
- Serum creatinine \< 1.5 x ULN for age and gender, OR creatinine clearance or GFR (radioisotope or iothalamate) ≥ 70 mL/min/1.73 m2
- Serum ALT or AST ≤ 3x ULN
- Total bilirubin ≤ 1.5 mg/dL, unless subject has Gilbert's Syndrome
- Cardiac ejection fraction ≥ 45%
- No evidence of physiologically significant pericardial effusion as determined by an ECHO
- No clinically significant ECG findings
- No clinically significant pleural effusion
- Pulse oximetry ≥ 92% on room air, OR forced vital capacity ≥ 50% of predicted value
- Not Pregnant: Females of childbearing potential must have a negative pregnancy test.
- Contraception: people 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 regimen or...
- Must provide informed consent. All people ≥ 18 years of age must be able to give informed consent. For people \ 7 years of age, when appropriate. If a minor becomes of age during participation of this study, he/she will...
What rules you out
- Any patient with metastatic disease OUTSIDE the CNS.
- Unwilling or unable, in the investigator's judgement, to have a CSF reservoir (Ommaya or Rickham) placed. Does not apply to people who have a pre-existing device suitable for ICV delivery of CAR T cells and ICP...
- Clinical evidence of active/on-going significant increased intracranial pressure (i.e., impending herniation) or uncontrolled seizures.
- Prior receipt of a chimeric antigen receptor (CAR)-based therapy.
- Currently receiving anticoagulation therapy.
- Known history of infection with HIV or hepatitis B (HBsAg positive) or hepatitis C virus (anti-HCV positive). EXCEPTION: A history of hepatitis B or hepatitis C is permitted if the viral load is undetectable per...
- Pregnancy or breastfeeding in a postpartum female.
- Known sensitivity or allergy to any agents/reagents used in this study.
- History of prior other malignancy. EXCEPTION: Previously diagnosed and definitively treated more than 5 years prior to enrollment or whose prognosis is deemed good enough to not warrant surveillance.
- Primary immunodeficiency or history of autoimmune disease (e.g., Crohn's, rheumatoid arthritis, systemic lupus) resulting in end organ injury or requiring systemic immunosuppression/systemic disease modifying agents...
- History of myocardial infarction, cardiac angioplasty or stenting, unstable angina, or other clinically significant cardiac disease within 12 months of enrollment.
- Significant medical diseases or poorly controlled conditions that, in the judgement of the investigator, put the subject at an unacceptable risk of complications, including but not limited to: uncontrolled diabetes...
- In the Investigator's judgment, the subject or parents/caregivers (as required) will not be able to comply with the study procedures outlined in the study protocol including follow-up visits.
The study team makes the final eligibility decision.
Where it's taking place
- Palo Alto, California, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 30 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Palo Alto, California, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.