Refractory/Relapse NeuroblastomaPhase 1NCT05608148
Clinical Trial of GAIA-102 for Refractory/Relapse Neuroblastomas and Other Malignant Pediatric Solid Tumors
A Phase 1 study testing Biological in people with Refractory/Relapse Neuroblastoma and Pediatric Solid Tumors, run by Kyushu University at 1 site in Japan.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 1 year to 24 years
- Conditions the study is for
- Refractory/Relapse Neuroblastoma, Pediatric Solid Tumors
- What is being tested?
- Biological
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 61 participants
- Where
- 1 siteJapan
- Study dates
- 26 Oct 2022to 25 Aug 2027
- 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 24 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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 \& D : neuroblastoma and other malignant solid tumors, rhabdomyosarcoma, Ewing's sarcoma family, hepatoblastoma.
- 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.
Show all 10Show fewer
- cohort C \& D : 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.
- Patients who have medical history for serious side effect , allergy reaction with regards to concomitant drugs.
- Patients aged from 1years to 24 years at the time of obtaining consent.
- 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.
Who cannot join
- Patients with brain metastases.
- Patients diagnosed with cancerous meningitis
- Patients who received allogeneic hematopoietic stem cell transplant.
- Patients with active autoimmune disease.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Japan1 site, 1 recruiting
- Recruiting
Kyushu University Hospital
Fukuoka, Fukuoka, 812-8582
Naonori Kawakubo, +81-92-642-5573, kawakubo.naonori.061@m.kyushu-u.ac.jp
About this study
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 D(GAIA-102 with Nivolumab, Teceleukin):
Confirm the safety of GAIA-102(Follow the recommended doses in Cohort A) with Nivolumab, Teceleukin.
- Being tested
- Biological
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BiologicalBiological
Intravenous injection of GAIA-102 alone
BiologicalBiological
Intravenous injection of GAIA-102 with dinutuximab, filgrastim, teceleukin combination
BiologicalBiological
Intravenous injection of GAIA-102 with nivolumab combination
BiologicalBiological
Intravenous injection of GAIA-102 with nivolumab, teceleukin combination
| Study group | Role | What they receive |
|---|---|---|
| GAIA-102 alone | Experimental | GAIA-102: 5 x 10^6 cells /㎏/ dose at a fixed dose, 1 to 3 doses / week for 3 consecutive weeks Receives: Biological: Biological |
| GAIA-102 with Dinutuximab, Filgrastim, Teceleukin combination | Experimental | 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 Receives: Biological: Biological |
| GAIA-102 with Nivolumab combination | Experimental | 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 Day1,15 Receives: Biological: Biological |
| GAIA-102 with Nivolumab, Teceleukin combination | Experimental | 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 Day8,22 Teceleukin: 750,000 units/m2/day on Day1-4 and 1,000,000 units/m2/day on Day 15-18 Receives: Biological: Biological |
What is being measured
The primary outcome is the main question the study is designed to answer.
Presence or absence of Dose Limiting Toxicity(DLT) expression
Measured: At the end of Cycle1 (Cohort A & C & D: Cycle period is 28 days, Cohort B: Cycle period is 56 days)
Frequency and severerity of adverse events(Cohort C＆D)
Measured: 2 year
Secondary outcomes (5)
Objective reponse rate and presence or absence of new lesions
Measured: 2 year
Overall survival rate and progression free survival rate
Measured: 2 year
Best overall response and lesion control rate
Measured: 2 year
Frequency and severity of adverse events
Measured: 2 year
Frequency and severerity of immune-related adverse events
Measured: 2 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2022
- Study start26 Oct 2022
- Primary completion25 Aug 2027 (estimated)
- Study completion25 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory/Relapse NeuroblastomaStudies 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 61 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kyushu University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05608148
- Sponsor's protocol ID
- GAIA-102-PT
- Status last verified
- Aug 2026
- First submitted
- 24 Oct 2022
- Last updated
- 7 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No