DLT evaluation of the monotherapy cohort for GAIA-102 in the clinical trial for pediatric patients with relapsed/refractory solid malignancies has been completed

Fukuoka, JAPAN – The registration (12 cases) and DLT (Dose Limiting Toxicity) evaluation of the monotherapy cohort (Cohort A, a basket trial for multiple tumor types) in the clinical trial for pediatric cancer patients with recurrent and refractory neuroblastoma or solid pediatric malignancies with pulmonary metastases, which started in November 2022, have been completed. The Independent Data Monitoring Committee (IDMC) confirmed that no serious adverse events leading to trial discontinuation were detected in Cohort A. The most frequently observed adverse event was “fever”, each graded as 1-2. Treatment-emergent laboratory abnormalities observed in a dose-dependent manner were transient increases in eosinophils. Treatment for the final patient is still ongoing.

The breakdown of the 12 cases registered in Cohort A is as follows:

• • • Neuroblastoma: 6 cases
    • Hepatoblastoma and Osteosarcoma: 2 cases each
    • Rhabdomyosarcoma and Ewing sarcoma: 1 case each

*All are pediatric solid tumors resistant to standard treatment or with no-option recurrences, having experienced 2-8 chemotherapy regimens, respectively.

IDMC also convened on February 18, 2024, agreed the recommended dose per administration of GAIA-102 in children as 5×10⁶ [cells/kg] (up to 2×10⁸ [cells/person]) and the recommended administration frequency as three times a week (no limit on the number of administrations). Safety and efficacy data for the 12 cases in Cohort A will be disclosed at domestic and international conferences and in an academic journal in near future.

As for Cohort B (combination cohort with dinutuximab), four cases have been registered so far, with a total of 12 cases planned for registration.

Additionally, a trial amendment application to add Cohort C (combination cohort with nivolumab, a basket trial for multiple solid tumor types) has been accepted by PMDA and approved by the IRB at Kyushu University Hospital. Cohort C will start in June, with a total of 20 cases planned for registration.

https://clinicaltrials.gov/study/NCT05608148

https://jrct.niph.go.jp/latest-detail/jRCT2073220054

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