DIPG Brain TumorPhase 1CMD03DIPGNCT06221553
Safety and Efficacy of Loco-regional B7H3 IL-7Ra CAR T Cell in DIPG
A Phase 1 study testing B7H3 specific CAR T cell with IL-7Ra signaling domain in people with DIPG Brain Tumor and Diffuse Intrinsic Pontine Glioma, run by Chulalongkorn University at 1 site in Thailand.
Trial summary
A Phase 1 clinical trial to evaluate the safety and early efficacy of CAR T-cell with IL-7Ra signal targeting B7H3 in children with diffuse intrinsic pontine glioma (DIPG) patients after complete standard treatments. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- DIPG Brain Tumor, Diffuse Intrinsic Pontine Glioma
- What is being tested?
- B7H3 specific CAR T cell with IL-7Ra signaling domain
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteThailand
- Study dates
- 1 Mar 2024to 1 Sep 2026
- Phase
- Phase 1
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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participants must have diffuse intrinsic pontine glioma at any timepoint following completion of standard radiotherapy
- Age 1-18 years
- Sex: Male or female
- CNS reservoir catheter, such as an Ommaya or Rickham catheter, present in the proper location for CNS-directed therapy
- Performance status: Lansky or Karnofsky score \>= 60
- Life expectancy \>= 8 weeks
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- 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%
- 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
- Participants and/or legal guardians must have the ability to understand and willingness to sign a written informed consent and/or assent document
Who cannot join
- Presence of \>= grade 3 cardiac dysfunction or symptomatic arrythmia requiring intervention
- Presence of primary immunodeficiency or bone marrow failure syndrome
- Presence of clinical and/or radiographic evidence of impending herniation of CNS
- Presence of \> Grade 3 dysphagia
- History of active malignancy other than nonmelanoma skin cancer and carcinoma in situ (e.g., cervix, bladder, breast).
- 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.
- 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.
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Thailand1 site, 1 recruiting
- Recruiting
King Chulalongkorn Memorial Hospital
Bangkok, Pathumwan, 10330
Piti Techavichit, MD, +66817515363, piti.t@chula.ac.th
Kanhatai Chiengthong, MD, +66814430961, kanhatai.c@chula.ac.th
About this study
Official title: Safety and Efficacy of Intraventricular Infusion of B7H3 With IL-7 Receptor Alpha Signaling Chimeric Antigen Receptor T Cell in Diffuse Intrinsic Pontine Glioma
A Phase 1 clinical trial to evaluate the safety and early efficacy of CAR T-cell with IL-7Ra signal targeting B7H3 in children with diffuse intrinsic pontine glioma (DIPG) patients after complete standard treatments.
Read the detailed description
The brain tumor known as Diffuse Intrinsic Pontine Glioma is commonly found in children aged between 5 to 10 years. This type of tumor is aggressive and infiltrates the structures of the brain stem. Treatment options are limited due to the location of the tumor, making it impossible to surgically remove the mass like other brain cancers. The standard treatment for this type of tumor is radiation therapy, as this type of cancer does not respond to chemotherapy or currently available targeted drugs. However, radiation therapy has been found to be ineffective and does not improve survival rates.
Currently, there is development in cancer treatment using immunotherapy, where the patient's immune cells are genetically modified to target the cancer, also known as CAR T cells, for the treatment of recurrent or refractory cancers in solid tumors and brain cancers. The research project aims to study the efficacy and safety of treating patients with pontine glioma using T cells that are antigen-specific and have signals from the interleukin-7 receptor alpha and are specific to the B7H3 antigen on the tumor surface. This research is the first of its kind in Thai patients. The research project expects that this treatment will be highly safe and effective in controlling diffuse pontine glioma.
- Conditions
- DIPG Brain Tumor, Diffuse Intrinsic Pontine Glioma
- Being tested
- B7H3 specific CAR T cell with IL-7Ra signaling domain
- Keywords
- CAR T cell, DIPG, B7H3, IL-7 receptor alpha, Chimeric antigen receptor T cell, Adoptive cellular therapy
- Medical subject headings
- Diffuse Intrinsic Pontine Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
B7H3 specific CAR T cell with IL-7Ra signaling domainBiological
Autologous T cells lentivirally transduced to express a B7H3 specific chimeric antigen receptor (CAR) with additional of IL-7 receptor alpha signalingdomain given via indwelling central nervous system (CNS) catheter
| Study group | Role | What they receive |
|---|---|---|
| Locoregional delivery of B7H3/IL-7Ra CAR T cell in DIPG | Experimental | Patients with DIPG for whom CAR T cells will be delivered into the ventricular system
Interventions:
Biological: Autologous B7H3-specific chimeric antigen receptor (CAR) T cell with additional of IL-7Ra signaling domain Dose level: 1x10e7 CAR T cell, 3x10e7 CAR T cell, 10x10e7 CAR T cell Receives: Biological: B7H3 specific CAR T cell with IL-7Ra signaling domain |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of B7H3-IL7Ra CAR T cells infusion in diffuse intrinsic pontine glioma (DIPE) patients.
The incidence of adverse events assessed by the National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0
Measured: Up to 28 days after B7H3-IL7Ra CAR-T cell infusion
Secondary outcomes (1)
The overall response rate of diffuse intrinsic pontine glioma (DIPE)
The overall response rate will be assessed using radiologic response criteria that use the standard sum of the two longest 2D perpendicular diameters to distinguish stable disease, progressive disease (>25% increase), partial response (>50% decrease), and complete response (no evaluable or measurable disease).
Measured: 3, 6, and 12 months after B7H3-IL7Ra CAR-T cell infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2024
- Study start1 Mar 2024
- Primary completion1 Mar 2026 (estimated)
- Study completion1 Sep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DIPG Brain TumorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 3+3 dose escalation study
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chulalongkorn University
- Sponsor type
- University, hospital or other
- Collaborators
- King Chulalongkorn Memorial Hospital
- Responsible party
- Sponsor
- Investigators
- Piti Techavichit, MD, Principal investigator, Chulalongkorn University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06221553
- Sponsor's protocol ID
- LV-B7H3IL7-CART-DPG-P1-2023
- Other IDs
- Chulalongkorn University
- Acronym
- CMD03DIPG
- Status last verified
- Mar 2025
- First submitted
- 15 Jan 2024
- Last updated
- 30 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No