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Alkylating agents

Risk-Directed Therapy for Medulloblastoma

Phase 2
Recruiting
Led By Giles W. Robinson, 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
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline and 6 months after enrollment
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new way to treat children with medulloblastoma that is based on the individual child's risk factors.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
Possible side effects include lowered blood cell counts leading to infection risk, nausea, vomiting, hair loss from chemotherapy; surgical risks like bleeding or infection; irritation from radiation therapy; fatigue and allergic reactions.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline through 6 months after enrollment
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline through 6 months after enrollment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Executive function among infants and young children treated with systemic chemotherapy only compared to patients treated with systemic chemotherapy and intra-ventricular chemotherapy or delayed risk-adapted craniospinal irradiation
Pharmacotherapy
Pharmacotherapy
+3 more
Secondary study objectives
Association of familial factors and environmental factors with cognitive late effects.
Association of familial factors and environmental factors with socioeconomic status
Medulloblastoma
+20 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
9%
Blood sodium decreased
8%
Hypokalaemia
7%
Insomnia
7%
Pyrexia
6%
Hyponatraemia
6%
Blood creatinine increased
6%
Lymphopenia
6%
Diarrhoea
6%
Dyspepsia
6%
Red blood cell count decreased
6%
Cough
4%
Dizziness
2%
Bone marrow failure
1%
Ischaemic stroke
1%
Dyspnoea
1%
Cerebral infarction
1%
Pulmonary embolism
1%
Embolism venous
1%
Superior vena cava syndrome
100%
80%
60%
40%
20%
0%
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

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
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Etoposide
2010
Completed Phase 3
~2960
Carboplatin
2014
Completed Phase 3
~6120
Pegfilgrastim
2013
Completed Phase 3
~4440
Filgrastim
2000
Completed Phase 3
~3690
Topotecan
2017
Completed Phase 3
~2890
Surgical resection
2021
Completed Phase 2
~1740
Methotrexate
2019
Completed Phase 4
~4400
Cisplatin
2013
Completed Phase 3
~3120
Vincristine
2003
Completed Phase 4
~2970
Cyclophosphamide
2010
Completed Phase 4
~2310
Irradiation
2015
N/A
~490

Find a Location

Who is running the clinical trial?

St. Jude Children's Research HospitalLead Sponsor
443 Previous Clinical Trials
5,323,331 Total Patients Enrolled
13 Trials studying Medulloblastoma
2,776 Patients Enrolled for Medulloblastoma
Giles W. Robinson, MDPrincipal InvestigatorSt. Jude Children's Research Hospital
3 Previous Clinical Trials
221 Total Patients Enrolled
2 Trials studying Medulloblastoma
89 Patients Enrolled for Medulloblastoma
Aditi Bagchi, MD, PhDPrincipal InvestigatorSt. Jude Children's Research Hospital

Media Library

Carboplatin (Alkylating agents) Clinical Trial Eligibility Overview. Trial Name: NCT05535166 — Phase 2
Medulloblastoma Research Study Groups: Stratum S-2, Stratum S-1, Stratum N, Cognitive Study Group I (educational video and games), Cognitive Study Group II (standard-of-care control)
Medulloblastoma Clinical Trial 2023: Carboplatin Highlights & Side Effects. Trial Name: NCT05535166 — Phase 2
Carboplatin (Alkylating agents) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05535166 — Phase 2
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