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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 3 years
Awards & highlights
Study 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.Check my eligibility
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.See study design
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 belowSelect...
My child is under 1 year old with a small tumor in the adrenal gland or elsewhere.
Select...
My cancer's genetic features match one of the study's treatment groups.
Select...
My largest tumor is smaller than 5 cm, and I haven't had it removed or biopsied.
Select...
My baby is under 6 months old with a small adrenal tumor.
Select...
I am under 18 months old with a specific type of newly diagnosed neuroblastoma.
Select...
My cancer is a newly diagnosed type of neuroblastoma or ganglioneuroblastoma without MYCN gene amplification.
Select...
My child was diagnosed with neuroblastoma before turning 12 or 18 months, depending on its stage.
Select...
My child is under 18 months old with a new diagnosis of advanced neuroblastoma.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 3 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 3 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Overall survival (OS) (Strata 1-4)
Other outcome measures
Neoplasms
Histology of tumor specimens
Etoposide
+6 moreSide effects data
From 2023 Phase 2 trial • 27 Patients • NCT0400240188%
Pyrexia
65%
Neutrophil count decreased
62%
Nausea
58%
Hypotension
50%
Anaemia
46%
Headache
38%
Decreased appetite
38%
Fatigue
35%
Confusional state
31%
Hypokalaemia
31%
Diarrhoea
31%
Tachycardia
27%
Constipation
27%
Back pain
27%
Hypophosphataemia
23%
Dizziness
23%
Platelet count decreased
23%
Tremor
23%
B-cell lymphoma
23%
White blood cell count decreased
19%
Oedema peripheral
19%
Neutropenia
19%
Cough
19%
Hypogammaglobulinaemia
19%
Hyponatraemia
19%
Tachypnoea
19%
Agitation
15%
Alanine aminotransferase increased
15%
Thrombocytopenia
15%
Chills
15%
Dyspnoea
15%
Hypomagnesaemia
15%
Sinus tachycardia
15%
Dysphagia
12%
Hypertension
12%
Vomiting
12%
Abdominal pain
12%
Aspartate aminotransferase increased
12%
Pain
12%
Malaise
12%
Myalgia
12%
Hypoxia
12%
Arthralgia
12%
Hyperglycaemia
12%
Covid-19
12%
Peripheral sensory neuropathy
8%
Hyperhidrosis
8%
Aphasia
8%
Pancytopenia
8%
Muscular weakness
8%
Pneumonia
8%
Encephalopathy
8%
Eye pain
8%
Gait disturbance
8%
Oral candidiasis
8%
Urinary tract infection
8%
Sepsis
8%
Blood creatinine increased
8%
Acute myeloid leukaemia
8%
Insomnia
8%
Somnolence
8%
Dysuria
8%
Asthenia
8%
Lymphocyte count decreased
4%
Pleural effusion
4%
Covid-19 pneumonia
4%
Respiratory failure
4%
Febrile neutropenia
4%
Embolism
4%
Depression
4%
Syncope
100%
80%
60%
40%
20%
0%
Study treatment Arm
Axicabtagene Ciloleucel and Rituximab Combination
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
Cyclophosphamide
1995
Completed Phase 3
~3780
Doxorubicin Hydrochloride
2019
Completed Phase 3
~17850
Carboplatin
2014
Completed Phase 3
~6670
Magnetic Resonance Imaging
2017
Completed Phase 3
~1190
Computed Tomography
2017
Completed Phase 2
~2720
Biopsy
2014
Completed Phase 4
~850
Bone Marrow Aspiration
2011
Completed Phase 2
~1740
Bone Marrow Biopsy
2021
Completed Phase 2
~10
Etoposide
2010
Completed Phase 3
~2440
Ultrasound
2013
Completed Phase 1
~1950
Find a Location
Who is running the clinical trial?
National Cancer Institute (NCI)NIH
13,654 Previous Clinical Trials
40,932,532 Total Patients Enrolled
205 Trials studying Neuroblastoma
53,107 Patients Enrolled for Neuroblastoma
Children's Oncology GroupLead Sponsor
453 Previous Clinical Trials
237,079 Total Patients Enrolled
63 Trials studying Neuroblastoma
31,248 Patients Enrolled for Neuroblastoma
Holly J MeanyPrincipal InvestigatorChildren's Oncology Group
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My cancer's genetic features match one of the study's treatment groups.My largest tumor is smaller than 5 cm, and I haven't had it removed or biopsied.My baby is under 6 months old with a small adrenal tumor.My tumor has MYCN amplification.I am under 18 months old with a specific type of newly diagnosed neuroblastoma.My cancer is a newly diagnosed type of neuroblastoma or ganglioneuroblastoma without MYCN gene amplification.My child was diagnosed with neuroblastoma before turning 12 or 18 months, depending on its stage.My child is under 1 year old with a small tumor in the adrenal gland or elsewhere.My child is under 18 months old with a new diagnosis of advanced neuroblastoma.
Research Study Groups:
This trial has the following groups:- Group 1: Group A (clinical observation)
- Group 2: Group B (clinical observation, first-line chemotherapy)
- Group 3: Group C (clinical observation, first-line chemotherapy)
Awards:
This trial has 2 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is Cyclophosphamide a drug that is commonly studied?
"Cyclophosphamide was first introduced in 1997 at Spectrum Health Hospital - Butterworth Campus. In the two decades since, there have been 2829 completed clinical trials. At the moment, there are 1699 active trials, a majority of which are based out of Park Ridge, Illinois."
Answered by AI
Has Cyclophosphamide been deemed effective and safe by the FDA?
"Cyclophosphamide was given a score of 3 because there is both efficacy data and multiple rounds of safety data from Phase 3 trials."
Answered by AI
Is this study prominent in North America?
"Enrollment locations for this 100-person clinical trial include Advocate Children's Hospital-Park Ridge in Park Ridge, Saint Jude Midwest Affiliate in Peoria, University of Iowa/Holden Comprehensive Cancer Center in Iowa City, and many others."
Answered by AI
Is this research the inaugural project of its kind?
"A global search on clinicaltrials.gov indicates that there are 1699 ongoing trials involving cyclophosphamide in 84 countries and 3514 cities. The first trial for this drug was conducted in 1997 and completed its Phase 3 approval stage in 300 patients. Since then, 2829 studies have been completed."
Answered by AI
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