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Alkylating agents
Risk-Directed Therapy for Medulloblastoma
Tampa, FL
Phase 2
Recruiting
Led By Aditi Bagchi, MD, PhD
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.
See full description
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.See study design
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 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline through 6 months after enrollment
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline through 6 months after enrollment
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 moreSecondary 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 moreSide effects data
From 2023 Phase 3 trial • 457 Patients • NCT0400571685%
Anaemia
79%
Alopecia
66%
Neutropenia
56%
White blood cell count decreased
48%
Thrombocytopenia
44%
Nausea
39%
Decreased appetite
37%
Constipation
33%
Leukopenia
30%
Alanine aminotransferase increased
25%
Vomiting
24%
Hypoalbuminaemia
24%
Hyponatraemia
22%
Aspartate aminotransferase increased
22%
Neutrophil count decreased
17%
Hypokalaemia
17%
Insomnia
16%
Diarrhoea
16%
Cough
15%
Arthralgia
15%
Rash
14%
Platelet count decreased
14%
Hyperuricaemia
13%
Malaise
13%
Pyrexia
12%
Fatigue
12%
Hypothyroidism
12%
Gamma-glutamyltransferase increased
12%
Hyperglycaemia
11%
Blood creatine phosphokinase increased
11%
Lymphocyte count decreased
11%
Weight decreased
11%
Pain in extremity
11%
Dizziness
11%
Dyspnoea
10%
Hypertriglyceridaemia
10%
Headache
10%
Haemoptysis
10%
Productive cough
9%
Hypochloraemia
9%
Hypertension
8%
Non-cardiac chest pain
8%
Blood creatinine increased
8%
Weight increased
8%
Hypocalcaemia
7%
Upper respiratory tract infection
7%
Hyperthyroidism
7%
Abdominal pain upper
7%
Asthenia
7%
Blood bilirubin increased
7%
Back pain
7%
Pruritus
6%
Abdominal distension
6%
Chest discomfort
6%
Blood alkaline phosphatase increased
6%
Blood lactate dehydrogenase increased
5%
Oedema peripheral
5%
Toothache
5%
Blood creatine phosphokinase MB increased
5%
Hypercholesterolaemia
5%
Hypomagnesaemia
5%
Hiccups
4%
Sinus tachycardia
4%
Gingival pain
4%
Face oedema
4%
Hypophosphataemia
4%
Infusion related reaction
4%
Cancer pain
4%
Hypoaesthesia
4%
Tumour pain
3%
Mouth ulceration
3%
Abdominal pain
3%
Bilirubin conjugated increased
3%
Pneumonitis
2%
Dysphagia
2%
Pneumonia
2%
Fibrin D dimer increased
1%
Febrile neutropenia
1%
Immune-mediated myocarditis
1%
Ileus
1%
Intestinal obstruction
1%
Death
1%
Immune-mediated hepatitis
1%
COVID-19 pneumonia
1%
Diabetes mellitus
1%
Type 1 diabetes mellitus
1%
Depressed level of consciousness
1%
Neuropathy peripheral
1%
Oral pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arm A: Tislelizumab + Chemotherapy
Arm B: Placebo + Chemotherapy
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
~2970
Carboplatin
2014
Completed Phase 3
~6450
Pegfilgrastim
2013
Completed Phase 3
~4440
Filgrastim
2000
Completed Phase 3
~3990
Topotecan
2017
Completed Phase 3
~2880
Surgical resection
2021
Completed Phase 2
~1740
Methotrexate
2019
Completed Phase 4
~4400
Cisplatin
2013
Completed Phase 3
~3040
Vincristine
2003
Completed Phase 4
~2970
Cyclophosphamide
2010
Completed Phase 4
~2280
Irradiation
2015
N/A
~490
Find a Location
Closest Location:C.S. MOTT Children's Hospital, University of Michigan· Ann Arbor, MI· 166 miles
Who is running the clinical trial?
St. Jude Children's Research HospitalLead Sponsor
450 Previous Clinical Trials
5,326,341 Total Patients Enrolled
13 Trials studying Medulloblastoma
2,635 Patients Enrolled for Medulloblastoma
Aditi Bagchi, MD, PhDPrincipal InvestigatorSt. Jude Children's Research Hospital
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
Media Library
Eligibility Criteria:
This trial includes the following eligibility 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.My organs are functioning well enough for me to join the study.I am under 5 years old.I am less than 3 years old.I have received treatment for medulloblastoma before.I can start treatment within 36 days after my surgery.My medulloblastoma's type has been confirmed by a central review.My parent or guardian can and is willing to sign the consent form for me.I am newly diagnosed with medulloblastoma.I have not had any treatment for my brain tumor except for steroids and surgery.I can do some activities but need help.I am under 3 years old.My cancer is a CNS embryonal tumor, not medulloblastoma.My tumor sample is available for detailed lab analysis.
Research Study Groups:
This trial has the following groups:- Group 1: Stratum S-2
- Group 2: Stratum S-1
- Group 3: Stratum N
- Group 4: Cognitive Study Group I (educational video and games)
- Group 5: Cognitive Study Group II (standard-of-care control)
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.