Clinical Trial of GAIA-102 for Refractory/Relapse Neuroblastomas and Other Malignant Pediatric Solid Tumors

Phase 1

Recruiting

• Conditions: Pediatric Solid Tumors; Refractory/Relapse Neuroblastoma
• Interventions: Biological: Biological

• Registration Number: NCT05608148
• Lead Sponsor: Kyushu University

Brief Summary

Single Cohort A(GAIA-102 alone):

Confirm the safety of GAIA-102 alone for refractory/relapse neuroblastoma or pediatric solid tumors with lung metastases, and decide recommended dose for Phase II.

Cohort B(GAIA-102 with Dinutuximab):

Confirm the safety of GAIA-102 with Dinutuximab, Filgrastim, Teceleukin combination for refractory/relapse neuroblastoma and decide recommended dose for Phase II.

Cohort C(GAIA-102 with Nivolumab):Confirm the safety of GAIA-102(Follow the recommended doses in Cohort A) with Nivolumab.

Cohort C(GAIA-102 with Nivolumab):Confirm the safety of GAIA-102(Follow the recommended doses in Cohort A) with Nivolumab

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-10-24
• Study Start: 2022-10-26
• Study First Submitted QC: 2022-11-07
• Study First Posted: 2022-11-08
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-26
• Last Update Posted: 2024-03-28
• Primary Completion: 2027-08-25
• Study Completion: 2027-08-25

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 56

Inclusion Criteria

1. Patients who have been confirmed to have the following malignant tumor by histological examination

   • single cohort : 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) .
   • combination cohort : neuroblastoma.

2. Patients 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.

Exclusion 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
GAIA-102 alone	Biological	GAIA-102: 5 x 10\^6 cells / dose at a fixed dose, 1 to 3 doses / week for 3 consecutive weeks
GAIA-102 with Nivolumab combination	Biological	GAIA-102: 5 x 10\^6 cells / dose at a fixed dose, 3 doses / week for 3 consecutive weeks Nivolumab: 3mg/kg/day(Children) or 240mg/day(Adults) on Day1
GAIA-102 with Dinutuximab, Filgrastim, Teceleukin combination	Biological	GAIA-102: 5 x 10\^6 cells / dose at a fixed dose, 1 to 3 doses / week for 3 consecutive weeks Filgrastim: 5 µg/kg/day on Day1-14 Teceleukin: 750,000 units/m2/day on Day29-31 and 1,000,000 units/m2/day on Day 36- 39 Dinutuximab: 17.5mg/m2/day on Day4-7 and Day36-39

Primary Outcome Measures

Name	Time	Method
Presence or absence of Dose Limiting Toxicity(DLT) expression	At the end of Cycle1 (Single Cohort: Cycle period is 28 days, Combination Cohort: Cycle period is 56 days)

Secondary Outcome Measures

Name	Time	Method
Objective Response Rate and Disease Control Rate	Cycle 1 (Single Cohort: Cycle period is 28 days, Combination Cohort: Cycle period is 56 days)
Progression free Survival	2 year
Overall Survival	2 year
Frequency and severity of adverse events	2 year

Trial Locations

Locations (1)

Kyushu University Hospital; 🇯🇵 Fukuoka, Japan