Evaluating a new CAR T-cell therapy for children with DIPG
Safety and Efficacy of Intraventricular Infusion of B7H3 With IL-7 Receptor Alpha Signaling Chimeric Antigen Receptor T Cell in Diffuse Intrinsic Pontine Glioma
This study is testing a new CAR T-cell therapy to see if it can help children with DIPG fight their tumors after standard treatments.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 9 (estimated) |
| Ages | 1 Year to 18 Years |
| Sex | All |
| Sponsor | Chulalongkorn University Academic / other |
| Drugs / interventions | CAR-T, chemotherapy, CAR T, immunotherapy, radiation |
| Locations | 1 site (Bangkok, Pathumwan) |
| Trial ID | NCT06221553 on ClinicalTrials.gov |
What this trial studies
This Phase 1 clinical trial aims to assess the safety and early efficacy of a novel CAR T-cell therapy targeting the B7H3 antigen in children diagnosed with diffuse intrinsic pontine glioma (DIPG) after they have completed standard treatments. The study involves genetically modifying the patient's immune cells to enhance their ability to attack the tumor, utilizing a signaling domain from the interleukin-7 receptor alpha. Given the aggressive nature of DIPG and the limited treatment options available, this innovative approach seeks to provide a new avenue for therapy in this challenging pediatric population.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 1-18 years who have been diagnosed with DIPG and have completed standard radiotherapy.
Not a fit: Patients who have not completed standard treatments or have other significant health issues may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could offer a new, effective option for controlling DIPG in children, potentially improving survival rates.
How similar studies have performed: While CAR T-cell therapies have shown promise in other cancers, this specific approach targeting DIPG is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Participants must have diffuse intrinsic pontine glioma at any timepoint following completion of standard radiotherapy 2. Age 1-18 years 3. Sex: Male or female 4. CNS reservoir catheter, such as an Ommaya or Rickham catheter, present in the proper location for CNS-directed therapy 5. Performance status: Lansky or Karnofsky score \>= 60 6. Life expectancy \>= 8 weeks 7. Normal organ function: 7.1 AST (SGOT) \< 5 times the upper limit of normal (ULN) 7.2 ALT (SGPT) \< 5 times the upper limit of normal (ULN) 7.3 Total bilirubin \< 3 times the upper limit of normal (ULN) 7.4 Creatinine \< 5 times the upper limit of normal (ULN) 7.5 SpO2 room air \>=90% 8. Prior therapy wash-out before planned leukapheresis 8.1 \>= 7 days post last chemotherapy/biologic therapy administration 8.2 3 half-lives or 30 days, whichever is shorter after the last dose of antitumor antibody therapy 8.3 At least 30 days from most recent cellular infusion 8.4 All systemically administered corticosteroid treatment therapy must be stable or decreasing within 1 week prior to enrollment with a maximum dexamethasone dose of 2.5 mg/m2/day. Corticosteroid physiologic replacement therapy is allowed 9. Participants and/or legal guardians must have the ability to understand and willingness to sign a written informed consent and/or assent document Exclusion Criteria: 1. Presence of \>= grade 3 cardiac dysfunction or symptomatic arrythmia requiring intervention 2. Presence of primary immunodeficiency or bone marrow failure syndrome 3. Presence of clinical and/or radiographic evidence of impending herniation of CNS 4. Presence of \> Grade 3 dysphagia 5. History of active malignancy other than nonmelanoma skin cancer and carcinoma in situ (e.g., cervix, bladder, breast). 6. Presence of uncontrolled intercurrent illness including but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, pulmonary abnormalities, or psychiatric illness/social situations that would limit compliance with study requirements. 7. Pregnant or breastfeeding women were excluded from this study because CAR-T-cell therapy may be associated with the potential for teratogenic or abortifacient effects. Women of childbearing potential must have a negative serum pregnancy test. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with CAR-T cells, breastfeeding should be discontinued. These potential risks may also apply to other agents used in this study. Participants of childbearing or child-fathering potential must be willing to practice birth control from the time of enrollment in this study and for four months after receiving CAR-T-cell infusion. 8. Serologic status reflecting active HIV, hepatitis B or C infection. Participants who are positive for hepatitis B core antibody, hepatitis B surface antigen or hepatitis C antibody must have negative PCR prior to enrollment.
Where this trial is running
Bangkok, Pathumwan
- King Chulalongkorn Memorial Hospital — Bangkok, Pathumwan, Thailand (Recruiting)
Study contacts
- Principal investigator: Piti Techavichit, MD — Chulalongkorn University
- Study coordinator: Piti Techavichit, MD
- Email: piti.t@chula.ac.th
- Phone: 6622564900
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.