Phase I clinical trial of GAIA-102 for refractory/relapse neuroblastomas and other malignant pediatric solid tumors

JRCT ID: jRCT2073220054

Registered date:23/09/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: refractory/relapse neuroblastoma and other pediatric solid tumors
• Date of first enrollment: 02/11/2022
• Target sample size: 56
• Study type: Interventional

Intervention(s)

Cohort A(GAIA-102 alone):dose escalation study(3+3 design)
Cohort B(GAIA-102 with Dinutuximab+Filgrastim+Teceleukin):dose escalation study(3+3 design)
Cohort C(GAIA-102 with Nivolumab):apply mutatis mutandis to 3+3 design

Outcome(s)

Primary Outcome

cohort A, cohort B : Presence or absense of DLT expression
cohort C : Presence or absense of DLT expression, Presence or absense of Adverse event

Secondary Outcome

Objective Reponse Rate and presence or absence of new lesions
Overall Survival rate and progression free survival rate(6, 12, 18, 24M)
Best overall response and lesion control rate(CT/MRI imaging using RECIST ver1.1)
Frequency and severerity of adverse events(using CTCAE ver5.0)
Frequency and severerity of immune-related adverse events

Key inclusion & exclusion criteria

• Age minimum: >= 1age old
• Age maximum: <= 24age old
• Gender: Both

Include criteria

1. Patients who have been confirmed to have the following malignant tumor by histological examination
- cohort A : neuroblastoma or malignant solid tumor with pulmonary metastases, rhabdomyosarcoma, undifferentiated sarcoma, Ewing's sarcoma family, osteosarcoma, other cartilage sarcoma, nephroblastoma, hepatoblastoma, germ cell neoplasma, other rare solid tumor (except brain tumor and brain metastases) .
- cohort B : neuroblastoma.
- cohort C : neuroblastoma and other malignant solid tumors, rhabdomyosarcoma, Ewing's sarcoma family, hepatoblastoma.
2. Undergoing the following treatment.
- cohort A & B : Patients who have the resistance for more than two treatment regimens, and the resistance for all standard regimens based on the guideline.
- cohort C : Patients with neuroblastoma who have completed the dinutuximab regimen and still have residual tumor. Patients with rhabdomyosarcoma, Ewing's sarcoma family, hepatoblastoma who have the resistance for more than two treatment regimens, and the resistance for all standard regimens based on the guideline.
3. Patients who have medical history for serious side effect , allergy reaction with regards to concomitant drugs.
4. Patients aged from 1years to 24 years at the time of obtaining consent.
5. Patients with performance status(PS) over 50 (Lansky Performance Status Score less than 16 years old) or (Karnofsky Performance Status over 16 years old) at the time of obtaining consent.

Exclude criteria

1.Patients with brain metastases.
2.Patients diagnosed with cancerous meningitis
3.Patients who received allogeneic hematopoietic stem cell transplant.
4.Patients with active autoimmune disease.

Related Information

• Primary Sponsor: Kawakubo Naonori
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development