NeuroblastomaPhase 2NCT04909515
Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GMCSF) and Isotretinoin for Consolidation of Patients With High-Risk Neuroblastoma in First Remission.
A Phase 2 study testing Naxitamab, GM-CSF and Isotretinoin in people with Neuroblastoma, run by Y-mAbs Therapeutics at 9 sites in 4 countries.
Trial summary
This is a single-arm, uncontrolled, international, multi-center, clinical,phase 2 trial, in patients ≥ 12 months of age with high-risk neuroblastoma in first remission. 120 patients will be enrolled to receive naxitamab + GM-CSF in combination with isotretinoin. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Naxitamab, GM-CSF and Isotretinoin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 9 sitesin 4 countries
- Study dates
- 2 Dec 2021to Apr 2027
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Documentet neuroblastoma at time of diagnosis defined as
- Histopathology of solid tumor biopsy, or
- Bone marrow aspirate or biopsy indicative of neuroblastoma plus high blood or urine catecholamine metabolite levels
- Documented high-risk disease at time of initial diagnosis defined as
- MYNC-amplified at stage L2, M or MS (according to International Neuroblastoma Risk Group (INRG)) of any age or
- MYCN-nonamplified with stage M (according to INRG) and diagnosed at ≥ 18 months of age or
Show all 10Show fewer
- Patient must have completed frontline therapy, and achieved one of the following:
- verified complete response according to INRC (bone marrow positive minimal residual disease is allowed as assessed by RTqPCR at site) after completion of induction and consolidation with or without autologous stem cell transplantation
- Verified partial response according to INRC for primary site and soft tissue, bone, and bone marrow metastases at pre-autologous stem cell transplantation evaluation (i.e., myeloablative chemotherapy + autologous stem cell transplantation required as part of frontline regimen). Furthermore, bone marrow response must be with ≤ 5% tumor (of total nucleated cellular content) seen on any specimen from a bilateral bone marrow aspirate/biopsy and bone response must be with ≤ 3 areas of abnormal uptake on 123I-MIBG scintigraphy
- Age ≥ 12 months at trial enrollment
Who cannot join
- Verified progressive disease during induction or consolidation therapy
- Any systemic anti-cancer therapy, including chemotherapy, within 3 weeks prior to enrollment
- Autologous stem cell transplantation within 6 weeks prior to enrollment or ongoing toxicity caused by the autologous stem cell transplantation at the discretion of the Investigator
- Therapeutic 131I-MIBG within 6 weeks prior to enrollment
- Prior anti-GD2 therapy
- Performance status of < 50% as per the Lansky scale (patients less than 16 years of age) or Karnofsky scale (patients aged 16 years or older)
Trial locations and contacts
9 sites in 4 countries.
9 sites in 4 countries
Russia3 sites
National Medical Research Center Pediatric Hematology, Oncology and Immunology n.a Dmitry Rogachev
Moscow
Research Institute of Pediatric Oncology ad Hematology of N.N. Blokhin National Medical Research Center of Oncology
Moscow
Raisa Gorbacheva Memorial Institute of Children Hematology and Transplantation Bone marrow Transplant Clinic
Saint Petersburg
South Korea3 sites
Asan Medical Center Childrens Hospital
Seoul
Samsung Medical Center
Seoul
Seoul National University Hospital
Soeul
Singapore2 sites
ICON Cancer Centre Novena
Singapore
KK Women's and Children's Hospital
Singapore
About this study
Official title: Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GMCSF) and Isotretinoin for Consolidation of Patients With High-Risk Neuroblastoma in First Remission. An International, Open-Label,Uncontrolled, Single-Arm, Multicenter, Phase 2 Trial
This is a single-arm, uncontrolled, international, multi-center, clinical,phase 2 trial, in patients ≥ 12 months of age with high-risk neuroblastoma in first remission. 120 patients will be enrolled to receive naxitamab + GM-CSF in combination with isotretinoin.
- Conditions
- Neuroblastoma
- Being tested
- Naxitamab, GM-CSF, Isotretinoin
- Keywords
- antibody, neuroblastoma, pediatric, naxitamab, first remission
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NaxitamabDrug
3.0 mg/kg/day = 9.0 mg/kg/cycle
GM-CSFDrug
250 - 500 microgram/m2/day
IsotretinoinDrug
160 mg/m2/day
| Study group | Role | What they receive |
|---|---|---|
| naxitamab + GM-CSF + isotretinoin | Experimental | 8 cycles. Cycles 1+2 naxitamab + GM-CSF, cycles 3-5 naxitamab + GM-CSF + isotretinoin, cycles 6-8 isotretinoin Receives: Drug: Naxitamab; Drug: GM-CSF; Drug: Isotretinoin |
What is being measured
The primary outcome is the main question the study is designed to answer.
2-year progression free survival (PFS)
2-year PFS defined as the proportion of patients alive and without progressive disease (PD) or relapse 2 years after enrollment
Measured: 2 years
Secondary outcomes (6)
Progression free survival
Progression free survival (defined as time from enrollment until progressive disease/relapse or death, whichever comes first)
Measured: 128 weeks
1-year progression free survival
1-year progression free survival defined as the proportion of patients alive and without progressive disease or relapse 1 year after enrollment
Measured: 1 year
1-year and 2-year overall survival
1-year and 2-year overall survival defined as the proportion of patients alive 1 year and 2 years after enrollment, respectively
Measured: 2 years
2-year event-free survival
2-year event-free survival, defined as the proportion of patients alive and without progressive disease, relapse, secondary malignancy or until last contact if no event occurred, 2 years after enrollment
Measured: 2 years
Proportion of positive to negative minimal residual disease (MRD) after 2 cycles
Proportion of patients changing from minimal residual disease positive in bone marrow at enrollment (defined as patients assessed as minimal residual disease positive by RTqPCR of bone marrow at enrollment) to minimal residual disease negative at completion of Cycle 2
Measured: 6 weeks
Proportion of patients with MD positive convert to MRD negative
Proportion of patients with disease in bone marrow at enrollment (i.e., minimal disease according to INRC) and convert to minimal residual disease negative (assessed by RTqPCR) at completion of Cycle 2
Measured: 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2021
- Study start2 Dec 2021
- Primary completionJun 2026 (estimated)
- Study completionApr 2027 (estimated)
Why it stopped: Study terminated due to business priorities
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Y-mAbs Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04909515
- Sponsor's protocol ID
- 202
- Status last verified
- Aug 2022
- First submitted
- 27 May 2021
- Last updated
- 1 Sep 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GMCSF) and Isotretinoin for Consolidation of Patients With High-Risk Neuroblastoma in First Remission. An International, Open-Label,Uncontrolled, Single-Arm, Multicenter, Phase 2 Trial
- ClinicalTrials.gov ID
- NCT04909515
- Acronym / sponsor ID
- 202
- Conditions
- Neuroblastoma
- Keywords
- antibody, neuroblastoma, pediatric, naxitamab, first remission
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study terminated due to business priorities
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Y-mAbs TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 2 Dec 2021 (actual)
- Primary completion
- Jun 2026 (estimated)
- Study completion
- Apr 2027 (estimated)
- First posted
- 1 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single-arm, uncontrolled, international, multi-center, clinical,phase 2 trial, in patients ≥ 12 months of age with high-risk neuroblastoma in first remission. 120 patients will be enrolled to receive naxitamab + GM-CSF in combination with isotretinoin.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| naxitamab + GM-CSF + isotretinoin | Experimental | Drug: Naxitamab, Drug: GM-CSF, Drug: Isotretinoin8 cycles. Cycles 1+2 naxitamab + GM-CSF, cycles 3-5 naxitamab + GM-CSF + isotretinoin, cycles 6-8 isotretinoin |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Documentet neuroblastoma at time of diagnosis defined as
- Documented high-risk disease at time of initial diagnosis defined as
- Patient must have completed frontline therapy, and achieved one of the following:
- Age ≥ 12 months at trial enrollment
Key exclusion criteria (6 listed)
- Verified progressive disease during induction or consolidation therapy
- Any systemic anti-cancer therapy, including chemotherapy, within 3 weeks prior to enrollment
- Autologous stem cell transplantation within 6 weeks prior to enrollment or ongoing toxicity caused by the autologous stem cell transplantation at the discretion of the Investigator
- Therapeutic 131I-MIBG within 6 weeks prior to enrollment
- Prior anti-GD2 therapy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Naxitamab3.0 mg/kg/day = 9.0 mg/kg/cycle | Drug | naxitamab + GM-CSF + isotretinoin |
| GM-CSF250 - 500 microgram/m2/day | Drug | naxitamab + GM-CSF + isotretinoin |
| Isotretinoin160 mg/m2/day | Drug | naxitamab + GM-CSF + isotretinoin |
Primary outcome
2-year progression free survival (PFS)
Time frame: 2 years
2-year PFS defined as the proportion of patients alive and without progressive disease (PD) or relapse 2 years after enrollment
6 secondary outcomes
Progression free survival
Time frame: 128 weeks
1-year progression free survival
Time frame: 1 year
1-year and 2-year overall survival
Time frame: 2 years
2-year event-free survival
Time frame: 2 years
Proportion of positive to negative minimal residual disease (MRD) after 2 cycles
Time frame: 6 weeks
Proportion of patients with MD positive convert to MRD negative
Time frame: 6 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2022.
Related trials for Neuroblastoma
RecruitingPhase 1/2
Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse
Recurrent Ganglioneuroblastoma, Recurrent Neuroblastoma
Active, not recruitingObservational study
Genomic Structural Variation in Cancer Susceptibility
Breast Cancer, Colon Cancer
RecruitingPhase 2
Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors
Metastatic Chondrosarcoma, Locally Advanced Chondrosarcoma
RecruitingObservational study
Prospective Comprehensive Molecular Analysis of Endocrine Neoplasms
Endocrine Tumors, Thyroid Neoplasms
RecruitingPhase 1
PEACH TRIAL- Precision Medicine and Adoptive Cellular Therapy
Neuroblastoma, Diffuse Intrinsic Pontine Glioma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
9 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Naxitamab, GM-CSF and Isotretinoin in people with Neuroblastoma, run by Y-mAbs Therapeutics at 9 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 1 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Russia, South Korea, Singapore, Hong Kong. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
2-year progression free survival (PFS) (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Sep 2022.