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New treatment option for Ganglioneuroblastoma, Nodular
Official title Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
ClinicalTrials.gov ID: NCT06172296
What this study is testing
What is Carboplatin?
Carboplatin is an investigational medicine, given as an once-daily infusion into a vein, being studied as a potential treatment for ganglioneuroblastoma, nodular.
Also referred to as Blastocarb, Carboplat.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This phase III trial tests how well the addition of dinutuximab to Induction chemotherapy along with standard of care surgical resection of the primary tumor, radiation, stem cell transplantation, and immunotherapy works for treating children with newly diagnosed high-risk neuroblastoma. Dinutuximab is a monoclonal antibody that binds to a molecule called GD2, which is found on the surface of neuroblastoma cells, but is not present on many healthy or normal cells in the body.
- Phase 3: a large, late-stage study
- Time commitment: about 10 years
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 30
You may be able to join if
- Patients must be enrolled on APEC14B1 and have consented to testing through the Molecular Characterization Initiative (MCI), prior to enrollment on...
- ≤ 30 years at the time of initial diagnosis with high-risk disease
- \ Must have a diagnosis of neuroblastoma (NBL) or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of...
- Newly diagnosed, high risk neuroblastoma (HRNBL) defined as one of the following:
- Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M and MYCN amplification
You likely can't join if
- Patients who are 365-546 days of age with INRG Stage M and MYCN non-amplified NBL, irrespective of additional biologic features
- Patients ≥ 547 days of age with INRG Stage L2, MYCN non-amplified NBL, regardless of additional biologic features
- Patients with known bone marrow failure syndromes
- Patients on chronic immunosuppressive medications (e.g., tacrolimus, cyclosporine, corticosteroids) for reasons other than prevention/treatment of...
- Patients with a primary immunodeficiency syndrome who require ongoing immune globulin replacement therapy
- Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is...
See the full eligibility criteria
Who can join
- Patients must be enrolled on APEC14B1 and have consented to testing through the Molecular Characterization Initiative (MCI), prior to enrollment on ANBL2131
- ≤ 30 years at the time of initial diagnosis with high-risk disease
- \ Must have a diagnosis of neuroblastoma (NBL) or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamines
- Newly diagnosed, high risk neuroblastoma (HRNBL) defined as one of the following:
- Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M and MYCN amplification
- Age ≥ 547 days and INRG stage M regardless of biologic features (clinical MYCN testing not required prior to enrollment)
- Any age initially diagnosed with INRG Stage L1 MYCN amplified NBL who have progressed to stage M without systemic chemotherapy
- Age ≥ 547 days of age initially diagnosed with INRG Stage L1, L2, or MS who have progressed to stage M without systemic chemotherapy (clinical MYCN testing not required prior to enrollment)
- Patients must have a body surface area (BSA) ≥ 0.25 m\^2
- No prior anti-cancer therapy except as outlined below:
- Patients initially recognized to have high-risk disease treated with topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing, and with consent
- Patients observed or treated with a single cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (e.g., as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high-risk disease...
- Patients who received localized emergency radiation to sites of life threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis
- Human immunodeficiency virus (HIV) -infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
- A serum creatinine based on age/sex as follows:
- 1 month to \< 6 months: Male 0.4 mg/dL and female 0.4mg/dL
- 6 months to \< 1 year: Male 0.5 mg/dL and female 0.5 mg/dL
- 1 to \< 2 years: Male 0.6 mg/dL and female 0.6 mg/dL
- 2 to \< 6 years: Male 0.8 mg/dL and female 0.8 mg/dL
- 6 to \< 10 years: Male 1 mg/dL and female 1 mg/dL
- 10 to \< 13 years: Male 1.2 mg/dL and female 1.2 mg/dL
- 13 to \< 16 years: Male 1.5 mg/dL and female 1.4 mg/dL
- ≥ 16 years: Male 1.7 mg/dL and female 1.4 mg/dL
- The threshold creatinine values were derived from the Schwartz formula for estimating glomerular filtration rate (GFR) utilizing child length and stature data published by the Centers for Disease Control (CDC)
- or a 24-hour urine creatinine clearance ≥ 70 mL/min/1.73 m\^2 or
- or a GFR ≥ 70 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method or direct small molecule clearance method (iothalamate or other molecule per institutional standard)
- Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility
- Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age
- Serum glutamic pyruvic transaminase (SGPT) (Alanine aminotransferase [ALT]) ≤ 10 x ULN\
- Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L
- \ Shortening fraction of ≥ 27% by echocardiogram, or
- Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram
- Ability to tolerate Peripheral Blood Stem Cell (PBSC) collection: No known contraindication to PBSC collection. Examples of contraindications might be a weight or size less than the collecting institution finds...
What rules you out
- Patients who are 365-546 days of age with INRG Stage M and MYCN non-amplified NBL, irrespective of additional biologic features
- Patients ≥ 547 days of age with INRG Stage L2, MYCN non-amplified NBL, regardless of additional biologic features
- Patients with known bone marrow failure syndromes
- Patients on chronic immunosuppressive medications (e.g., tacrolimus, cyclosporine, corticosteroids) for reasons other than prevention/treatment of allergic reactions and adrenal replacement therapy are not eligible...
- Patients with a primary immunodeficiency syndrome who require ongoing immune globulin replacement therapy
- Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required prior to enrollment for female patients of childbearing...
- Lactating females who plan to breastfeed their infants
- Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
- All patients and/or their parents or legal guardians must sign a written informed consent
- All institutional, food and drug administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Mobile, Alabama, United States
- Mesa, Arizona, United States
- Phoenix, Arizona, United States
- Tucson, Arizona, United States
- Little Rock, Arkansas, United States
- Downey, California, United States
- Duarte, California, United States
- Loma Linda, California, United States
- Long Beach, California, United States
- Los Angeles, California, United States
- Madera, California, United States
- Oakland, California, United States
- Orange, California, United States
- Palo Alto, California, United States
- Sacramento, California, United States
- San Diego, California, United States
- San Francisco, California, United States
- Aurora, Colorado, United States
- Hartford, Connecticut, United States
+ 130 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 10 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, up to 30 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Mobile, Alabama, United States; Mesa, Arizona, United States; Phoenix, Arizona, United States; Tucson, Arizona, United States; Little Rock, Arkansas, United States and 144 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.