CLINICAL TRIAL

Pharmacological Study for Refractory Neuroblastoma

1 Prior Treatment
Metastatic
Recurrent
Refractory
Waitlist Available · < 65 · All Sexes · Cincinnati, OH

This study is evaluating whether a combination of nivolumab and ipilimumab is safe and effective in treating younger patients with solid tumors or sarcomas that have come back (recurrent) or do not respond to treatment (refractory).

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About the trial for Refractory Neuroblastoma

Eligible Conditions
Refractory Neuroblastoma · Refractory Osteosarcoma · Refractory Hodgkin Lymphoma · Hodgkin Disease · Neuroectodermal Tumors · Stage IIIA Cutaneous Melanoma AJCC v7 · Neuroectodermal Tumors, Primitive, Peripheral · Recurrent Hodgkin Lymphoma · Recurrent Malignant Solid Neoplasm · Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor · Sarcoma, Ewing · Stage III Cutaneous Melanoma AJCC v7 · Neoplasms · Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor · Stage IIIC Cutaneous Melanoma AJCC v7 · Stage IV Cutaneous Melanoma AJCC v6 and v7 · Refractory Non-Hodgkin Lymphoma · Skin Neoplasms · Recurrent Melanoma · Unresectable Melanoma · Refractory Malignant Solid Neoplasm · Sarcoma · Neuroblastoma · Stage IIIB Cutaneous Melanoma AJCC v7 · Osteosarcoma · Neuroectodermal Tumors, Primitive · Lymphoma, Non-Hodgkin · Refractory Rhabdomyosarcoma · Recurrent Non-Hodgkin Lymphoma · Lymphoma · Rhabdomyosarcoma · Metastatic Melanoma · Recurrent Neuroblastoma · Recurrent Osteosarcoma · Refractory Melanoma · Melanoma · Recurrent Rhabdomyosarcoma

Treatment Groups

This trial involves 2 different treatments. Pharmacological Study is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Ipilimumab
BIOLOGICAL
Laboratory Biomarker Analysis
OTHER
Nivolumab
BIOLOGICAL
Pharmacological Study
OTHER
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ipilimumab
FDA approved
Nivolumab
FDA approved

Eligibility

This trial is for patients born any sex aged 65 and younger. You must have received 1 prior treatment for Refractory Neuroblastoma or one of the other 36 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Patients must have had histologic verification of malignancy at original diagnosis or relapse
Parts A & C: patients must be >= 12 months and < 18 years of age at the time of study enrollment
Parts B1-B6, B8, D1-D6, E3, E4: patients must be >= 12 months and =< 30 years of age at the time of study enrollment
Part B7: patients must be >= 12 months and < 18 years of age at the time of study enrollment
Parts A & C: patients with recurrent or refractory solid tumors, without central nervous system (CNS) tumors or known CNS metastases, are eligible; note: CNS imaging for patients without a known history of CNS disease is only required if clinically indicated
Part B1: patients with relapsed or refractory neuroblastoma
Part B2: patients with relapsed or refractory osteosarcoma
Part B3: patients with relapsed or refractory rhabdomyosarcoma
Part B4: patients with relapsed or refractory Ewing sarcoma or peripheral primitive neuroectodermal tumor (PNET)
Part B5: patients with relapsed or refractory Hodgkin lymphoma
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Up to 5 years
Screening: ~3 weeks
Treatment: Varies
Reporting: Up to 5 years
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Up to 5 years.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Pharmacological Study will improve 2 primary outcomes and 8 secondary outcomes in patients with Refractory Neuroblastoma. Measurement will happen over the course of Up to 28 days.

Pharmacodynamics of nivolumab as a single agent or in combination with ipilimumab
UP TO 28 DAYS
Median (min,max) concentration by protein, study part, and dose level.
UP TO 28 DAYS
Frequency of dose limiting toxicities of nivolumab as a single agent or in combination with ipilimumab
28 DAYS
The frequency (%) of patients experiencing a dose limiting toxicity at least possibly attributable to nivolumab as a single agent or in combination with ipilimumab by study part and dose level.
28 DAYS
Maximum serum concentration of nivolumab as a single agent or in combination with ipilimumab
UP TO 56 DAYS
Median (min,max) of the maximum serum concentration of nivolumab as a single agent or in combination with ipilimumab by study part and dose level.
UP TO 56 DAYS
Area under the drug concentration curve of nivolumab as a single agent or in combination with ipilimumab
UP TO 56 DAYS
The median (min,max) of the area under the drug concentration curve for nivolumab as a single agent or in combination with ipilimumab by study part and dose level.
UP TO 56 DAYS
Clearance of nivolumab as a single agent or in combination with ipilimumab
UP TO 56 DAYS
Median (min,max) of the clearance of nivolumab as a single agent or in combination with ipilimumab by study part and dose level.
UP TO 56 DAYS
Half-life of nivolumab as a single agent or in combination with ipilimumab
UP TO 56 DAYS
The median (min,max) of the half-life of nivolumab as a single agent or in combination with ipilimumab by study part and dose level.
UP TO 56 DAYS
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Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the signs of neuroectodermal tumors?

Many neuroectodermal tumors are asymptomatic but may present as a painless, well-circumscribed, slow-growing mass on a clinical examination. It can be difficult to obtain a tissue or imaging-based diagnosis. The clinical setting has significantly improved the detection of these tumors. Tumor markers are very specific but limited in their ability to capture the vast majority of tumor types. Imaging via magnetic resonance imaging or computed tomography can be very helpful in the differential diagnosis of neuroectodermal tumors.

Anonymous Patient Answer

What is neuroectodermal tumors?

Neuroectodermal tumors are a group of tumors that form from the cells that form the embryonic ectoderm during prenatal development. Most neuroectodermal tumors result from spontaneous mutations of cells in neural crest as these cells are highly mobile during embryonic development and move to other areas to make other organs. Most neuroectodermal tumors occur in children who have immature immunity, and thus may be more susceptible to other infections and malignancies that are not contagious.

Anonymous Patient Answer

Can neuroectodermal tumors be cured?

Many neuroectodermal tumors can be cured, but surgery remains the treatment of choice in most cases and long-term survival is uncommon. The best prognosis can be achieved with tumors that express AFP but most patients with these tumors will have persistent disease.

Anonymous Patient Answer

What causes neuroectodermal tumors?

These tumors derive from multiple germ layer tissues (ectoderm, mesoendoderm, and endoderm), most of which are composed primarily of neuroblasts. Their growth is highly dependent on cellular amplification in the microenvironment and, as with other low-grade tumors, the accumulation of mutated cells and the formation of pseudostratified epithelium may play a dominant role. Neuroblasts can persist in the adult and eventually assume more undifferentiated, infiltrative growth patterns, and, often, become metastatic. Target therapy, radiotherapy, and surgery have become essential strategies for their management.

Anonymous Patient Answer

What are common treatments for neuroectodermal tumors?

Treatment should be based on the tumor type and site of the tumor. Treatment is almost always neoadjuvant prior to surgery. Treatment for neuroectodermal tumors includes surgery, chemotherapy, radiotherapy, or a combination of these. Patients with metastatic neuroectodermal tumors have a poor prognosis. For patients with non-metastatic neuroectodermal tumors, surgery is often curative with long-term survival. Patients with neuroectodermal tumors are more likely to respond to radiation therapy. Chemotherapy often fails to control the tumor. Postoperative radiation therapy has been proven to control local tumor progression and improve long-term survival.

Anonymous Patient Answer

How many people get neuroectodermal tumors a year in the United States?

Approximately 575,900 patients are diagnosed with a neuroectodermal tumor annually, making up 2.5 % of all visits to hospitals in the United States. This is greater than the incidence in the world (2.1/100,000), suggesting regional variations in the incidence of these tumors.

Anonymous Patient Answer

What is the latest research for neuroectodermal tumors?

We summarize the most recent literature for primary and secondary tumors of the brain, spinal cord, and spine, including neurofibromas, rhabdomyosarcomas, and embryonal carcinomas. We also summarize recent publications for metastatic neuroectodermal tumors. More studies are needed to establish the effectiveness of the treatments and the best ways to administer them.

Anonymous Patient Answer

What is the average age someone gets neuroectodermal tumors?

Given the rarity of neuroectodermal tumors, this dataset is a unique and important resource for researchers. In the future, this dataset could be used to find people at risk of neuroectodermal tumors and to understand the natural history of these neoplasms to help them develop a better understanding and treatment of neuroectodermal tumors in the future.

Anonymous Patient Answer

How serious can neuroectodermal tumors be?

[Neuroectodermal tumors are serious and warrant optimal care and attention] when considering treatment options. [Neuroectodermal tumors are the third most common cancer in adults, (about 20% of all cancers in adults), following lung and breast cancers, and a high percentage of tumors is diagnosed before age 40 years and in the fourth decade of life.] Clinicians should not underestimate their seriousness and the potential need for treatment and follow up.

Anonymous Patient Answer

Is pharmacological study typically used in combination with any other treatments?

There is little support for the use of pharmacological studies as stand-alone treatments, although most are prescribed off-label. Randomised control trials represent one form of such pharmacological study, but there is no consensus about the optimal dose-schedule, dose, route of administration, or frequency of repeated administrations. These factors are generally set by individual specialists, and the results from pharmacological studies are often applied to the management of patients according to their specific treatment plan.

Anonymous Patient Answer

Is pharmacological study safe for people?

Even if not treated, people who received a diagnosis of a neuroectodermal tumor are exposed to all potential risks of cancer treatment and to the potential risks of cancer induction. Although it is widely adopted to apply the principle of 'harm first' and focus only on the side-effects of the treatment after taking into account the benefits, this approach is not valid for the treatment of people with cancer. Hence, we should take into account the possible induction of a malignant neoplasm in the treated individuals with a lower likelihood of a posttherapy remission compared to the expected relapse.

Anonymous Patient Answer

Does neuroectodermal tumors run in families?

The findings presented in this review can lead to improved family-based screening for neuroectodermal tumors. Furthermore, family-based linkage analyses may complement studies done using population-based and clinic-based approaches in searching for hereditary neuroectodermal tumors.

Anonymous Patient Answer
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