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Alkylating agents

Risk Factor-Guided Therapy for Neuroblastoma

Phase 3

Waitlist Available

Led By Holly J Meany

Research Sponsored by Children's Oncology Group

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Group A1 patients must be > 6 months and < 12 months of age with an adrenal primary tumor < 5 cm in greatest diameter, or patients less than 6 months of age with an adrenal primary tumor > 3.1 and < 5 cm in greatest diameter, or < 12 months of age with a non-adrenal primary site < 5 cm in greatest diameter

Patients must meet specified criteria for one of the treatment groups based on genomic features

Must not have

Patients with MYCN amplified tumors

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 3 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial looks at a new way to treat neuroblastoma that may be more effective and have fewer side effects.

Who is the study for?

This trial is for young patients with non-high risk neuroblastoma or ganglioneuroblastoma. Eligible participants are under 18 months old, have specific tumor sizes and locations, and must not have received prior cancer treatment except dexamethasone. They should also exhibit certain genomic features without MYCN gene amplification.

What is being tested?

The study tests observation versus intervention based on the progression of tumors in young patients with neuroblastoma. It aims to determine when and what treatment might be necessary by examining biomarkers within tumor cells to guide therapy decisions.

What are the potential side effects?

Potential side effects may include those associated with biopsies, bone marrow procedures, chemotherapy drugs like Carboplatin, Cyclophosphamide, Doxorubicin Hydrochloride, Etoposide (such as nausea, hair loss), and imaging studies' risks.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My child is under 1 year old with a small tumor in the adrenal gland or elsewhere.

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My cancer's genetic features match one of the study's treatment groups.

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My largest tumor is smaller than 5 cm, and I haven't had it removed or biopsied.

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My baby is under 6 months old with a small adrenal tumor.

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I am under 18 months old with a specific type of newly diagnosed neuroblastoma.

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My cancer is a newly diagnosed type of neuroblastoma or ganglioneuroblastoma without MYCN gene amplification.

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My child was diagnosed with neuroblastoma before turning 12 or 18 months, depending on its stage.

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My child is under 18 months old with a new diagnosis of advanced neuroblastoma.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

My tumor has MYCN amplification.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Overall survival (OS) (Strata 1-4)

Other study objectives

Neoplasms

Histology of tumor specimens

Etoposide

+6 more

Side effects data

From 2023 Phase 2 trial • 27 Patients • NCT04002401

88%

Pyrexia

65%

Neutrophil count decreased

62%

Nausea

58%

Hypotension

50%

Anaemia

46%

Headache

38%

Fatigue

38%

Decreased appetite

35%

Confusional state

31%

Tachycardia

31%

Diarrhoea

31%

Hypokalaemia

27%

Constipation

27%

Hypophosphataemia

27%

Back pain

23%

Dizziness

23%

Tremor

23%

B-cell lymphoma

23%

Platelet count decreased

23%

White blood cell count decreased

19%

Cough

19%

Agitation

19%

Hyponatraemia

19%

Neutropenia

19%

Tachypnoea

19%

Hypogammaglobulinaemia

19%

Oedema peripheral

15%

Hypomagnesaemia

15%

Thrombocytopenia

15%

Dysphagia

15%

Alanine aminotransferase increased

15%

Sinus tachycardia

15%

Chills

15%

Dyspnoea

12%

Aspartate aminotransferase increased

12%

Pain

12%

Arthralgia

12%

Myalgia

12%

Hypertension

12%

Abdominal pain

12%

Hyperglycaemia

12%

Hypoxia

12%

Vomiting

12%

Peripheral sensory neuropathy

12%

Covid-19

12%

Malaise

Dysuria

Muscular weakness

Blood creatinine increased

Hyperhidrosis

Insomnia

Acute myeloid leukaemia

Encephalopathy

Sepsis

Pancytopenia

Asthenia

Eye pain

Urinary tract infection

Lymphocyte count decreased

Somnolence

Oral candidiasis

Pneumonia

Gait disturbance

Aphasia

Pleural effusion

Depression

Embolism

Syncope

Respiratory failure

Febrile neutropenia

Covid-19 pneumonia

100%

80%

60%

40%

20%

Study treatment Arm

Axicabtagene Ciloleucel and Rituximab Combination

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups

Experimental Treatment

Group I: Group C (clinical observation, first-line chemotherapy)Experimental Treatment11 Interventions

Patients at high risk for deterioration and a poor outcome immediately receive first-line chemotherapy as in Group B. All other patients undergo clinical observation for 3 years in the absence of disease progression. Upon disease progression, patients receive first-line chemotherapy as in Group B. Once a PR or better is achieved, patients undergo clinical observation for 3 years. Patients also undergo CT, MRI, and/or ultrasound throughout the trial and undergo bone marrow aspiration, bone marrow biopsy, and tumor biopsy at screening and time of progression.

Group II: Group B (clinical observation, first-line chemotherapy)Experimental Treatment11 Interventions

Patients undergo clinical observation for 3 years in the absence of disease progression. Upon disease progression, patients undergo surgery or receive first-line chemotherapy comprising carboplatin IV over 1 hour on day 1 (courses 1, 2, 4, 6, and 7), etoposide IV over 1 hour on days 1-3 (courses 1, 3, 4, 5, and 7), cyclophosphamide IV over 1 hour on day 1 (courses 2, 3, 5, 6, and 8), and doxorubicin hydrochloride IV over 15 minutes on day 1 (courses 2, 4, 6 and 8). Treatment with chemotherapy repeats every 21 days for 2-8 courses in the absence of disease progression or unacceptable toxicity. Once a PR or better is achieved, patients undergo clinical observation for 3 years. Patients also undergo CT, MRI, and/or ultrasound throughout the trial and undergo bone marrow aspiration, bone marrow biopsy, and tumor biopsy at screening and time of progression.

Group III: Group A (clinical observation)Experimental Treatment4 Interventions

Patients undergo clinical observation for 96 weeks in the absence of disease progression. Patients also undergo CT, MRI, and/or ultrasound throughout the trial.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Carboplatin

2014

Completed Phase 3

~6120

Cyclophosphamide

2010

Completed Phase 4

~2310