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Platinum-based Chemotherapy

Risk-Directed Therapy for Medulloblastoma

Phase 2

Waitlist Available

Led By Amar Gajjar, MD

Research Sponsored by St. Jude Children's Research Hospital

Eligibility Criteria Checklist

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

Must have

No previous radiotherapy, chemotherapy, or other brain tumor directed therapy other than corticosteroid therapy and surgery

Medulloblastoma or medulloblastoma variants including posterior fossa PNET as documented by an institutional pathologist

Must not have

CNS embryonal tumor other than medulloblastoma or PNET in the posterior fossa

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline and at 3 months after baseline

Awards & highlights

No Placebo-Only Group

Summary

This trial will use a new way to treat medulloblastoma, which is a cancer of the brain. The treatment will be based on the amount of leftover disease present after surgery, as well as the type of cancer cells. This will help to determine the best way to treat the cancer and improve the outcome for patients.

Who is the study for?

This trial is for individuals aged 3-22 with newly diagnosed medulloblastoma, or those up to age 40 with SHH subtype. They must have had surgery within the last 36 days and not received prior brain tumor treatments. Good performance status is required, and women of childbearing potential must not be pregnant.

What is being tested?

The study tests a tailored treatment approach based on both clinical risk (low to high) and molecular subtype (WNT, SHH, Non-WNT/Non-SHH) of medulloblastoma. It involves varying doses of radiation and chemotherapy including cyclophosphamide, cisplatin, vincristine; targeted drugs like vismodegib; new agents pemetrexed, gemcitabine; plus supportive therapies such as aerobic training and neurocognitive remediation.

What are the potential side effects?

Possible side effects include nausea from chemotherapy, hair loss due to radiation therapy, fatigue from treatment regimens, muscle weakness or numbness from vincristine. Newer drugs may cause unknown reactions while supportive therapies are generally well-tolerated.

Eligibility Criteria

Inclusion Criteria

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

Select...

I haven't had any treatment for my brain tumor except for steroids and surgery.

Select...

My cancer is a type of brain tumor known as medulloblastoma.

Exclusion Criteria

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

Select...

My brain tumor is not a medulloblastoma or PNET located in the back of my brain.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline and at 3 months after baseline

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline and at 3 months after baseline

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline and at 3 months after baseline for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Change in Spatial Span Backward Standard Score

Change in VO2 Peak Value

Progression-free Survival in Stratum N1

+2 more

Secondary study objectives

Association between baseline cognitive performance and fatigue

Association between baseline cognitive performance and sleep quality

Association between baseline cognitive performance and sleep quantity

+49 more

Side effects data

From 2012 Phase 3 trial • 256 Patients • NCT01005680

51%

Neutropenia

47%

Leukopenia

46%

Nausea

43%

Vomiting

35%

Anaemia

31%

Decreased appetite

26%

Haemoglobin decreased

26%

Fatigue

25%

Constipation

25%

White blood cell count decreased

24%

Neutrophil count decreased

19%

Alanine aminotransferase increased

13%

Platelet count decreased

12%

Rash

10%

Aspartate aminotransferase increased

10%

Thrombocytopenia

Blood sodium decreased

Hypokalaemia

Insomnia

Pyrexia

Hyponatraemia

Blood creatinine increased

Lymphopenia

Diarrhoea

Dyspepsia

Red blood cell count decreased

Cough

Dizziness

Bone marrow failure

Cerebral infarction

Dyspnoea

Ischaemic stroke

Pulmonary embolism

Embolism venous

Superior vena cava syndrome

100%

80%

60%

40%

20%

Study treatment Arm

Gemcitabine Plus Cisplatin (GC)

Pemetrexed Plus Cisplatin (PC)

Awards & Highlights

No Placebo-Only Group

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

Trial Design

8Treatment groups

Experimental Treatment

Group I: Stratum W3: High RiskExperimental Treatment6 Interventions

Participants in stratum W3 will undergo high dose craniospinal radiation with boost to the primary tumor site once daily 5 days a week for 6 weeks. Six weeks after completion of radiotherapy, patients receive one cycle of chemotherapy (cisplatin, vincristine, cyclophosphamide) once every 4 weeks for 4 cycles in absence of unacceptable toxicity. Some participants will complete aerobic training and/or neurocognitive remediation.

Group II: Stratum W2: AtypicalExperimental Treatment6 Interventions

Participants in stratum W2 will undergo standard dose craniospinal radiation with boost to the primary tumor site once daily 5 days a week for 6 weeks. Six weeks after completion of radiotherapy, patients receive one cycle of chemotherapy (cisplatin, vincristine, cyclophosphamide) once every 4 weeks for 4 cycles in absence of unacceptable toxicity. Some participants will complete aerobic training and/or neurocognitive remediation.

Group III: Stratum W1: Low RiskExperimental Treatment6 Interventions

Participants in stratum W1 will undergo reduced dose Craniospinal Irradiation with boost to the primary tumor site once daily 5 days a week for 6 weeks. Six weeks after completion of radiotherapy, patients receive one cycle of chemotherapy (cisplatin, vincristine, cyclophosphamide) once every 4 weeks for 4 cycles in absence of unacceptable toxicity. Some participants will complete aerobic training and/or neurocognitive remediation.

Group IV: Stratum S2: High RiskExperimental Treatment7 Interventions

Participants in stratum S2 will undergo high dose craniospinal radiation with boost to the primary tumor site once daily 5 days a week for 6 weeks. Six weeks after completion of radiotherapy, patients receive one cycle of chemotherapy (cisplatin, vincristine, cyclophosphamide) once every 4 weeks for 4 cycles in absence of unacceptable toxicity. After completion of 4 cycles of chemotherapy, participants who are skeletally mature will receive maintenance chemotherapy with vismodegib. Some participants will complete aerobic training and/or neurocognitive remediation.

Group V: Stratum S1: Standard RiskExperimental Treatment7 Interventions

Participants in stratum S1 will undergo standard dose craniospinal radiation with boost to the primary tumor site once daily 5 days a week for 6 weeks. Six weeks after completion of radiotherapy, patients receive one cycle of chemotherapy (cisplatin, vincristine, cyclophosphamide) once every 4 weeks for 4 cycles in absence of unacceptable toxicity. After completion of 4 cycles of chemotherapy, participants who are skeletally mature will receive maintenance chemotherapy with vismodegib. Some participants will complete aerobic training and/or neurocognitive remediation.

Group VI: Stratum N3: High RiskExperimental Treatment8 Interventions

Participants in stratum N3 will undergo high dose craniospinal radiation with boost to the primary tumor site once daily 5 days a week for 6 weeks. Six weeks after completion of radiotherapy, patients receive standard chemotherapy (cisplatin, vincristine, cyclophosphamide) for 4 cycles intermixed with an additional 3 cycles of chemotherapy with pemetrexed and gemcitabine in absence of unacceptable toxicity. Some participants will complete aerobic training and/or neurocognitive remediation.

Group VII: Stratum N2: Intermediate RiskExperimental Treatment8 Interventions

Participants in stratum N2 will undergo standard dose craniospinal radiation with boost to the primary tumor site once daily 5 days a week for 6 weeks. Six weeks after completion of radiotherapy, patients receive standard chemotherapy (cisplatin, vincristine, cyclophosphamide) for 4 cycles intermixed with an additional 3 cycles of chemotherapy with pemetrexed and gemcitabine in absence of unacceptable toxicity. Some participants will complete aerobic training and/or neurocognitive remediation.

Group VIII: Stratum N1: Standard RiskExperimental Treatment6 Interventions

Participants in stratum N1 will undergo standard dose craniospinal radiation with boost to the primary tumor site once daily 5 days a week for 6 weeks. Six weeks after completion of radiotherapy, patients receive one cycle of chemotherapy (cisplatin, vincristine, cyclophosphamide) once every 4 weeks for 4 cycles in absence of unacceptable toxicity. Some participants will complete aerobic training and/or neurocognitive remediation.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cyclophosphamide

2010

Completed Phase 4

~2310

Cisplatin

2013

Completed Phase 3

~3120