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NeuroblastomaPhase 4NCT06047535

Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) Combined With Isotretinoin for Maintenance Treatment of Patients With High-Risk Neuroblastoma in First Complete Response.

A Phase 4 study testing Naxitamab in people with Neuroblastoma, run by SciClone Pharmaceuticals.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Official record
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Trial summary

This is a single-arm, multicenter clinical trial conducted in patients ≥ 12 months of age with high-risk neuroblastoma in first complete response. 62 patients will be enrolled to receive naxitamab + GM-CSF in combination with isotretinoin. Read more about this study

Who is it for?
Ages 12 months and older
Conditions the study is for
Neuroblastoma
What is being tested?
Naxitamab
Type of treatment
Injection or infusion
How many people
62 participants
Where
No sites listed yet
Study dates
31 Oct 2023to 15 Aug 2027
Phase
Phase 4
Are there results?
Not yet: the study is under way

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 12 months and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Documented NB at time of diagnosis defined as26:
  • histopathology of solid tumor biopsy, or
  • BM aspirate or biopsy indicative of NB plus high blood or urine catecholamine metabolite levels
  • Documented high-risk disease at time of initial diagnosis defined as24, 26:
  • MYCN-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
  • Subjects must have completed frontline therapy described in 6.3.2 and have verified complete response according to INRC25 (BM MRD is allowed as assessed by RTqPCR at site28) after completion of induction and consolidation with or without ASCT
  • Age ≥ 12 months at trial enrollment
  • Life expectancy of greater than 6 months, as judged by the Investigator
  • Written informed consent from legal guardian(s) and/or patient in accordance with local regulations. Children must provide assent as required by local regulations

Who cannot join

  • Verified PD during induction or consolidation therapy
  • Any systemic anti-cancer therapy, including chemotherapy, within 3 weeks prior to enrollment
  • ASCT within 6 weeks prior to enrollment or ongoing toxicity caused by the ASCT 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)
Show all 27Show fewer
  • Left ventricular ejection fraction < 50% by echocardiography
  • Inadequate pulmonary function defined as evidence of dyspnea at rest, exercise intolerance, and/or chronic oxygen requirement. In addition, room air pulse oximetry < 94% and/or abnormal pulmonary function tests if these assessments are necessary at the discretion of the Investigator
  • Life threatening infection(s)
  • Treatment with long-acting myeloid growth factor within 14 days or short-acting myeloid growth factor within 7 days prior to first dose of GM-CSF
  • Treatment with immunosuppressive agents (local steroids excluded) within 4 weeks prior to enrollment
  • History of allergy or known hypersensitivity to GM-CSF, E. coli-derived products, or any component of GM-CSF, naxitamab, isotretinoin or vitamin A.
  • NB in the Central Nervous System (CNS) or leptomeningeal disease within 6 months prior to enrollment
  • Patients with uncontrolled seizure disorders despite anticonvulsant therapy (defined as a seizure event within 3 months prior to enrollment)
  • Unacceptable hematological status prior to first dosing, defined as one of the following:
  • Hemoglobin <5.0 mmol/L (<8 g/dL)
  • White blood cell (WBC) count <1000/µL
  • Absolute neutrophil count (ANC)<750/µL
  • Platelet count <75,000/µL
  • Unacceptable liver function prior to first dosing, defined as one of the following:
  • Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >5 times upper normal limit (UNL)
  • Bilirubin >1.5 x UNL
  • Unacceptable kidney function prior to first dosing, defined as:
  • Estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2 calculated by the 2009 revised Bedside Schwartz Equation27 (Please refer to Appendix 1)
  • Inability to comply with protocol, as judged by the Investigator
  • Patients with a significant intercurrent illness (any ongoing serious medical problem unrelated to cancer or its treatment) that is not covered by the detailed exclusion criteria and that is expected to interfere with the action of trial agents or to significantly increase the severity of the toxicities experienced from trial treatment
  • Females of childbearing potential who are pregnant, breast feeding, intend to become pregnant, or are not using adequate contraceptive methods or males who are not using adequate contraceptive methods. Contraception must be used for 1 month after last isotretinoin treatment and 42 days after last naxitamab treatment whichever comes last for both genders

About this study

Official title: Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) Combined With Isotretinoin for Maintenance Treatment of Patients With High-Risk Neuroblastoma in First Complete Response. A Single-Arm, Multicenter Clinical Trial

This is a single-arm, multicenter clinical trial conducted in patients ≥ 12 months of age with high-risk neuroblastoma in first complete response. 62 patients will be enrolled to receive naxitamab + GM-CSF in combination with isotretinoin.

In line with post-consolidation maintenance treatment of high-risk neuroblastoma, this trial will include patients with high-risk neuroblastoma in first complete response. Patients must have completed a multimodal frontline regimen (induction and consolidation) and have achieved complete response (positive bone marrow minimal residual disease as assessed by RTqPCR is allowed) following the multi agent induction and consolidation therapy.

Conditions
Neuroblastoma
Being tested
Naxitamab
Keywords
high-risk neuroblastoma with first complete response
Medical subject headings
Neuroblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NaxitamabDrug

    Also called GM-CSF, isotretinoin

    Naxitamab + GM-CSF:

    Cycles 1-5: Patients will receive naxitamab IV at 3 mg/kg/infusion on Day 1, 3 and 5 and GM-CSF sc at 5 µg/kg/day on Days -4 to 0 and at 10 µg/kg/day on Days 1-5 for 5 cycles.

    Isotretinoin:

    Cycles 3-8: Patients will receive isotretinoin PO at 160 mg/m2/day divided into two daily doses on Days 15-28 for 6 cycles.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 2-year progression free survival

    2-year progression free survival defined as the proportion of patients alive and without progressive disease or relapse 2 years after enrollment

    Measured: 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Sep 2023
  2. Study start31 Oct 2023 (estimated)
  3. Primary completion15 Jun 2027 (estimated)
  4. Study completion15 Aug 2027 (estimated)

Resources

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 4
Primary purpose
Prevention
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
62 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06047535
Sponsor's protocol ID
ONN203
Status last verified
Sep 2023
First submitted
12 Sep 2023
Last updated
21 Sep 2023
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Naxitamab in people with Neuroblastoma, run by SciClone Pharmaceuticals.

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 27 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: opening soon, as of the sponsor's last update on 21 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.

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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

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 (time frame: 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06047535.

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 21 Sep 2023.

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