ARI0008 for Diffuse intrinsic pontine glioma (DIPG)

Inclusion criteria

• {"criterion_text":"- Written informed consent signed by the patient or legal representative and, if applicable, informed assent (for minors aged ≥12 years)."}
• {"criterion_text":"- Candidates for placement of an Ommaya reservoir with intraventricular catheter."}
• {"criterion_text":"- Fertile males and females must use a highly effective contraceptive method."}
• {"criterion_text":"- Newly diagnosed DIPG based on clinical-radiological criteria and/or histological and molecular confirmation. Biopsied patients must show K27M mutation. Clinical and radiological criteria will be assessed by the PI with support from a neuroradiologist at Hospital Sant Joan de Déu."}
• {"criterion_text":"- Prior neoadjuvant radiotherapy (standard of care for DIPG) before receiving the trial treatment. Radiotherapy defined as 54–60 Gy in 1.8–2.2 Gy/fraction."}
• {"criterion_text":"- Age between 3 and 24 years inclusive."}
• {"criterion_text":"- Lansky performance score ≥50%. For patients ≥16 years, Karnofsky score ≥50%."}
• {"criterion_text":"- Life expectancy greater than 12 weeks."}
• {"criterion_text":"- Preserved bone marrow function: absolute neutrophil count >1,000/mcL, platelets >100,000/mcL (independent of transfusions), haemoglobin >8 g/dL (may be transfusion-dependent)."}
• {"criterion_text":"- Normal liver and kidney function."}
• {"criterion_text":"- Adequate venous access for leukapheresis."}

Exclusion criteria

• {"criterion_text":"- Patients with disease progression or disseminated disease on MRI at diagnosis."}
• {"criterion_text":"- HIV+, HCV+, HBV+ patients or those not meeting the serological screening criteria described in Annex 5."}
• {"criterion_text":"- Pregnancy."}
• {"criterion_text":"- Breastfeeding."}
• {"criterion_text":"- Participation in another experimental study within the last 3 months."}
• {"criterion_text":"- Patients receiving other antitumor treatments. If previously treated, a washout period of at least 4 weeks is required."}
• {"criterion_text":"- Associated comorbidities that, in the investigator’s clinical judgement, cannot be adequately controlled and may compromise the patient’s ability to tolerate the study treatment."}
• {"criterion_text":"- Patients with neoplasms other than DIPG."}
• {"criterion_text":"- Patients requiring corticosteroid treatment at a daily dose >2 mg/day dexamethasone (or equivalent)."}
• {"criterion_text":"- Patients for whom corticosteroid treatment cannot be suspended during the week prior to leukapheresis."}
• {"criterion_text":"- Patients receiving bevacizumab for pseudoprogression who do not show neurological stability."}
• {"criterion_text":"- Patients with uncontrolled infections."}

Primary endpoints

• {"endpoint_text":"- Number of Grade 3–4 serious adverse events (SAEs) according to Common Toxicity Criteria (CTC) from the start of treatment (first administration of DCs) until the end of the study per patient.","definition_or_measurement_approach":"Number of Grade 3–4 SAEs as classified by Common Toxicity Criteria (CTC) measured from first administration of DCs until end of study for each patient."}

Secondary endpoints

• {"endpoint_text":"- Proportion of patients with adverse events (AEs) and serious adverse events (SAEs), as well as the proportion of patients who discontinue treatment due to AEs.","definition_or_measurement_approach":"Proportion of patients experiencing AEs/SAEs and proportion discontinuing treatment due to AEs as recorded during study follow-up."}
• {"endpoint_text":"- Percentage of patients receiving the full treatment regimen, defined as the complete administration of both DCs and CAR-T cells.","definition_or_measurement_approach":"Percentage of patients who receive the complete administration of both DCs and CAR-T cells per protocol."}
• {"endpoint_text":"- Evaluation of immunological parameters according to the study schedule.","definition_or_measurement_approach":"Assessment of specified immunological parameters at timepoints defined in the study schedule."}
• {"endpoint_text":"- Overall survival (OS) and progression-free survival (PFS) in DIPG patients treated with the proposed regimen. A comparison will be made with historical controls from the institution (HSJD) and other international institutions (COG and SIOP).","definition_or_measurement_approach":"Measurement of OS and PFS for treated patients; comparison with historical control cohorts (HSJD, COG, SIOP)."}
• {"endpoint_text":"- Evaluation of radiological changes following RAPNO criteria (see Annex 3: Criteria for Radiological and Clinical Response Assessment).","definition_or_measurement_approach":"Radiological response assessed per RAPNO criteria as specified in protocol Annex 3."}
• {"endpoint_text":"- Evaluation of the distribution of anti-IL13Ra2 CAR-T cells in CSF, peripheral blood, and, if available, tumour samples, according to the study schedule.","definition_or_measurement_approach":"Detection/quantification of anti-IL13Ra2 CAR-T cells in CSF, peripheral blood, and tumour samples at scheduled timepoints."}
• {"endpoint_text":"- Correlation between histological and molecular characterisation, clinical-radiological response, and cellular response generated in peripheral blood and CSF following treatment.","definition_or_measurement_approach":"Analyses correlating histological/molecular features with clinical-radiological outcomes and cellular immune responses in blood and CSF."}
• {"endpoint_text":"- Correlation between immunological response and clinical progression. Determination of whether cellular response in peripheral blood and CSF correlates with increased overall survival and progression-free survival.","definition_or_measurement_approach":"Correlation analyses between measured cellular immune responses and OS/PFS/clinical progression."}
• {"endpoint_text":"- Evaluation of performance (QoL) as an indicator of quality of life. QoL will be assessed through prospective evaluation of each patient’s functionality using the PedsQL questionnaire (see Annex 2: Neurological and Functional Assessment).","definition_or_measurement_approach":"Quality of life assessed prospectively using the PedsQL questionnaire per protocol (Annex 2)."}

Spain

Earliest CTIS Part Ii Submission Date

11-12-2025

Latest Decision Or Authorization Date

22-12-2025

Processing Time Days

11

Number Of Sites

1

Number Of Participants

15

Primary sponsor

Full Name

Fundacio Sant Joan De Deu

Organisation Type

Patient organisation/association

Country Of Registered Address

Spain