120 Participants Needed

Risk-Directed Therapy for Medulloblastoma

Recruiting at 9 trial locations
TE
JL
Overseen ByJean Laboe, MSN, RN
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

Do I need to stop my current medications to join the trial?

The trial requires that any chemotherapy, immunotherapy, or targeted agents for non-cancer conditions be stopped at least 14 days before starting the trial treatment. Corticosteroid therapy is allowed.

What data supports the effectiveness of the drugs used in the Risk-Directed Therapy for Medulloblastoma?

The research articles provided focus on managing chemotherapy-induced nausea and vomiting (CINV) in patients receiving treatments like cisplatin and carboplatin, which are part of the medulloblastoma therapy. These studies suggest that antiemetic combinations, such as ondansetron with dexamethasone, are effective in reducing CINV, which can improve the overall treatment experience for patients undergoing similar chemotherapy regimens.12345

Is the treatment for medulloblastoma generally safe in humans?

The treatment for medulloblastoma using carboplatin, etoposide, and other drugs has been studied for safety. Some patients experienced hearing loss and blood-related side effects, but these treatments are generally considered safe with manageable side effects.678910

What makes the Risk-Directed Therapy for Medulloblastoma treatment unique?

This treatment is unique because it combines multiple chemotherapy drugs, radiation, and surgery to target medulloblastoma, a type of brain cancer, based on the patient's specific risk factors. This personalized approach aims to maximize effectiveness while minimizing side effects, unlike standard treatments that may not be tailored to individual risk levels.2451112

What is the purpose of this trial?

This is a multi-center, multinational phase 2 trial that aims to explore the use of molecular and clinical risk-directed therapy in treatment of children 0-4.99 years of age with newly diagnosed medulloblastoma.

Research Team

Dr. Giles W. Robinson, MD | Memphis, TN ...

Giles Robinson, MD

Principal Investigator

St. Jude Children's Research Hospital

AB

Aditi Bagchi

Principal Investigator

St. Jude Children's Research Hospital

Eligibility Criteria

This trial is for children under 5 years with newly diagnosed medulloblastoma. They must have a certain level of organ function, no prior brain tumor treatments except surgery, and a performance score over 30. Parents can consent and participate in educational interventions.

Inclusion Criteria

My disease was confirmed with brain and spine MRI and CSF test.
My medulloblastoma diagnosis has been confirmed by a central review.
My medulloblastoma belongs to a specific molecular subgroup.
See 12 more

Exclusion Criteria

I have received treatment for medulloblastoma before.
Participant who is actively receiving any other investigational agents
Participants with other clinically significant medical disorders that could compromise their ability to tolerate protocol therapy or would interfere with the study procedure
See 1 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Surgery

Surgical resection of the tumor prior to chemotherapy

1 week

Chemotherapy

Participants receive systemic high-dose methotrexate (HD-MTX) and conventional chemotherapy, with variations based on stratum assignment

8-12 months
Courses repeat every 4 weeks

Radiation

Risk-stratified craniospinal irradiation (CSI) for Stratum N participants at 36 months of age

4-6 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

84 months

Treatment Details

Interventions

  • Carboplatin
  • Cisplatin
  • Cyclophosphamide
  • Etoposide
  • Filgrastim
  • Irradiation
  • Methotrexate
  • Ommaya/VPS
  • Pegfilgrastim
  • Surgical resection
  • Topotecan
  • Vincristine
Trial Overview The study tests molecular and clinical risk-directed therapies including chemotherapy drugs like Carboplatin, Topotecan, Etoposide; surgical procedures; Methotrexate; irradiation; and supportive care with Pegfilgrastim or Filgrastim to boost white blood cells.
Participant Groups
5Treatment groups
Experimental Treatment
Active Control
Group I: Stratum S-2Experimental Treatment9 Interventions
Patients with Sonic Hedgehog subgroup 2 (SHH-2), 0-2.99 years, or M0 and 3-4.99 years, will receive systemic high-dose methotrexate (HD-MTX) and conventional chemotherapy. Interventions: Surgery, Methotrexate, Cisplatin, Vincristine, Cyclophosphamide, Carboplatin, Topotecan, Pegfilgrastim, Filgrastim
Group II: Stratum S-1Experimental Treatment10 Interventions
Patients with SHH-1, SHH-3, SHH-4, or SHH-Not otherwise specified (NOS), 0-2.99 years, will receive intraventricular methotrexate (IVT-MTX) in parallel with systemic HD-MTX and conventional chemotherapy. Interventions: Surgery, Methotrexate, Cisplatin, Vincristine, Cyclophosphamide, Carboplatin, Topotecan, Pegfilgrastim, Filgrastim
Group III: Stratum NExperimental Treatment11 Interventions
Patients with Medulloblastoma (MB) group 3 or group 4 (G3/G4) or MB \[including Non-WNT non-SHH medulloblastoma (NWNS) NOS or otherwise indeterminate cases\] (0-2.99 years) will receive systemic HD-MTX and conventional chemotherapy only for radiation delaying purposes. At 3 years of age, these patients will receive risk-stratified craniospinal irradiation (CSI). Interventions: Surgery, Methotrexate, Cisplatin, Vincristine, Cyclophosphamide, Carboplatin, Topotecan, Etoposide, Pegfilgrastim, Filgrastim, Radiation
Group IV: Cognitive Study Group I (educational video and games)Experimental Treatment1 Intervention
Educational video and games
Group V: Cognitive Study Group II (standard-of-care control)Active Control1 Intervention
Standard-of-care (SOC) followed by educational video and games

Carboplatin is already approved in United States, European Union, Canada for the following indications:

๐Ÿ‡บ๐Ÿ‡ธ
Approved in United States as Paraplatin for:
  • Ovarian cancer
  • Testicular cancer
  • Lung cancer
  • Head and neck cancer
  • Brain cancer
๐Ÿ‡ช๐Ÿ‡บ
Approved in European Union as Carboplatin for:
  • Ovarian cancer
  • Small cell lung cancer
๐Ÿ‡จ๐Ÿ‡ฆ
Approved in Canada as Carboplatin for:
  • Ovarian cancer
  • Small cell lung cancer
  • Testicular cancer

Find a Clinic Near You

Who Is Running the Clinical Trial?

St. Jude Children's Research Hospital

Lead Sponsor

Trials
451
Recruited
5,326,000+

National Cancer Institute (NCI)

Collaborator

Trials
14,080
Recruited
41,180,000+

Findings from Research

In a study of 89 ovarian cancer patients receiving paclitaxel and carboplatin, the combination of aprepitant, ramosetron, and dexamethasone effectively prevented chemotherapy-induced nausea and vomiting (CINV), achieving a complete response rate of 98.8% in the acute phase after the first cycle.
The treatment was well-tolerated, with only 38.9% of cycles experiencing adverse events, and none of the patients discontinued due to these events, indicating a favorable safety profile for this antiemetic regimen.
Safety and efficacy of aprepitant, ramosetron, and dexamethasone for chemotherapy-induced nausea and vomiting in patients with ovarian cancer treated with paclitaxel/carboplatin.Choi, CH., Kim, MK., Park, JY., et al.[2021]
In a study of 79 pediatric patients undergoing chemotherapy, the combination of fosaprepitant and ondansetron significantly reduced the frequency and severity of chemotherapy-induced nausea and vomiting (CINV) compared to ondansetron alone.
Fosaprepitant was found to be safe and well-tolerated, with no significant difference in adverse events between the two groups, making it a promising alternative for CINV prophylaxis in pediatric patients who cannot use dexamethasone.
Efficacy, safety and feasibility of fosaprepitant for the prevention of chemotherapy-induced nausea and vomiting in pediatric patients receiving moderately and highly emetogenic chemotherapy - results of a non-interventional observation study.Willier, S., Cabanillas Stanchi, KM., von Have, M., et al.[2020]
In a study of 30 patients with testicular germ cell tumors undergoing 5-day cisplatin chemotherapy, the combination of palonosetron, aprepitant, and dexamethasone achieved a high complete response rate of 90% for preventing vomiting during the first chemotherapy course.
The antiemetic regimen was well-tolerated, with only mild adverse effects reported, such as hiccups and anorexia, indicating a favorable safety profile for this treatment approach.
Antiemetic efficacy and safety of a combination of palonosetron, aprepitant, and dexamethasone in patients with testicular germ cell tumor receiving 5-day cisplatin-based combination chemotherapy.Hamada, S., Hinotsu, S., Kawai, K., et al.[2021]

References

Safety and efficacy of aprepitant, ramosetron, and dexamethasone for chemotherapy-induced nausea and vomiting in patients with ovarian cancer treated with paclitaxel/carboplatin. [2021]
Efficacy, safety and feasibility of fosaprepitant for the prevention of chemotherapy-induced nausea and vomiting in pediatric patients receiving moderately and highly emetogenic chemotherapy - results of a non-interventional observation study. [2020]
Antiemetic efficacy and safety of a combination of palonosetron, aprepitant, and dexamethasone in patients with testicular germ cell tumor receiving 5-day cisplatin-based combination chemotherapy. [2021]
Role of ondansetron plus dexamethasone in fractionated chemotherapy. [2018]
Optimizing emetic control in children receiving antineoplastic therapy: beyond the guidelines. [2021]
Outcome of newly diagnosed high risk medulloblastoma treated with carboplatin, vincristine, cyclophosphamide and etoposide. [2018]
Concomitant weekly vincristine and radiation followed by adjuvant vincristine and carboplatin in the treatment of high risk medulloblastoma: Ain Shams University Hospital and Sohag Cancer Center study. [2020]
Survival of patients with medulloblastoma treated with carboplatin and etoposide before and after radiotherapy. [2018]
Carboplatin and ototoxicity: hearing loss rates among survivors of childhood medulloblastoma. [2021]
10.United Statespubmed.ncbi.nlm.nih.gov
Activity of postoperative carboplatin, etoposide, and high-dose methotrexate in pediatric CNS embryonal tumors: results of a phase II study in newly diagnosed children. [2013]
11.United Statespubmed.ncbi.nlm.nih.gov
A survey of antiemetic use in children with cancer. [2015]
12.United Statespubmed.ncbi.nlm.nih.gov
Randomized, double-blind, placebo-controlled, phase III cross-over study evaluating the oral neurokinin-1 antagonist aprepitant in combination with a 5HT3 receptor antagonist and dexamethasone in patients with germ cell tumors receiving 5-day cisplatin combination chemotherapy regimens: a hoosier oncology group study. [2022]
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