Vincristine Sulfate for Medulloblastoma

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Children's Hospital Medical Center of Akron, Akron, OH
Medulloblastoma+6 More
Vincristine Sulfate - Drug
Eligibility
< 18
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a combination of chemotherapy drugs is effective in treating younger patients with newly diagnosed, non-metastatic desmoplastic medulloblastoma.

See full description

Eligible Conditions

  • Medulloblastoma
  • Medulloblastoma With Extensive Nodularity
  • Desmoplastic/Nodular Medulloblastoma
  • Untreated Childhood Medulloblastoma
  • Nevoid Basal Cell Carcinoma Syndrome

Treatment Effectiveness

Study Objectives

This trial is evaluating whether Vincristine Sulfate will improve 5 primary outcomes and 3 other outcomes in patients with Medulloblastoma. Measurement will happen over the course of Up to 10 days.

Day 189
Percentage of Patients With Responses at 189 Days
2 years from diagnosis
Event-free Survival (EFS)
Progression-free Survival (PFS)
Day 273
Percentage of Patients With Responses at 273 Days
Year 2
Overall Survival (OS)
Month 60
Change in Neurocognitive and Adaptive Functioning Assessed Using Full Scale IQ Score (FSIQ) and General Adaptive Composite Score (GAC)
Up to 10 days
Feasibility of Rapid Central Pathology Screening Review Defined as Success Rate of Timely Central Pathology Review Based on the Expectation That at Least 95% of the Cases Will be Reviewed Within 10 Days
Up to 2 years
Molecular Profile

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Trial Design

1 Treatment Group

Treatment (combination chemotherapy)
1 of 1
Experimental Treatment

This trial requires 26 total participants across 1 different treatment group

This trial involves a single treatment. Vincristine Sulfate 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 2 and have already been tested with other people.

Treatment (combination chemotherapy)INDUCTION THERAPY: Patients receive vincristine sulfate IV over 1 minute or infused via minibag on days 1, 15, and 29; cyclophosphamide IV over 1 hour on days 1-3; methotrexate IV over 24 hours on days 15 and 29; etoposide IV over 60-120 minutes on days 43-45; and carboplatin IV over 1 hour on days 43-45. Treatment repeats every 63 days for 3 courses in the absence of disease progression or unacceptable toxicity. CONTINUATION THERAPY: Patients receive vincristine sulfate IV over 1 minute or infused via minibag on day 1, cyclophosphamide IV over 1 hour on days 1-3, etoposide IV over 60-120 minutes on days 21-23, and carboplatin IV over 1 hour on days 21-23. Treatment repeats every 42 days for 2 courses in the absence of disease progression or unacceptable toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Carboplatin
FDA approved
Cyclophosphamide
FDA approved
Methotrexate
FDA approved
Vincristine
FDA approved
Beta-D-Glucose
Not yet FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 2 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 2 years for reporting.

Closest Location

Children's Hospital Medical Center of Akron - Akron, OH

Eligibility Criteria

This trial is for patients born any sex aged 18 and younger. You must have received newly diagnosed for Medulloblastoma or one of the other 6 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Patient must have negative lumbar cerebrospinal fluid (CSF) cytology (lumbar CSF must be obtained unless medically contraindicated); CSF cytology for staging should be performed preferably no sooner than 14 days post operatively to avoid false positive CSF, ideally, CSF should be obtained between day 14 and day 21 to allow for final staging status before enrollment onto the study; Note: patients with positive CSF cytology obtained prior to 14 days after surgery may have cytology repeated to determine eligibility and final CSF status
Post-operative cranial MRI with and without gadolinium within 72 hours of surgery
Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2; use Lansky for patients =< 16 years of age
Patients must have a life expectancy of >= 8 weeks
Pre-operative cranial magnetic resonance imaging (MRI) (recommended with gadolinium) or pre-operative computed tomography (CT) (recommended with contrast)
Patients must be newly diagnosed and have a confirmed histologic diagnosis of nodular desmoplastic (ND) medulloblastoma or medulloblastoma with extensive nodularity (MBEN) from rapid central pathology screening review; please note: patients with Gorlin syndrome are eligible
Spinal MRI pre-op with and without gadolinium or post-op with and without gadolinium preferably within 72 hours of surgery
Patients must be enrolled on study within 31 days of definitive surgical resection at which time tissue is acquired to determine a diagnosis; patients must be enrolled before treatment begins; the date protocol therapy is projected to start must be no later than five (5) calendar days after the date of study enrollment; patients who are started on protocol therapy on a Phase II study prior to study enrollment will be considered ineligible
Patients who are receiving dexamethasone must be on a stable dose for at least 1 week prior to study entry
Peripheral absolute neutrophil count (ANC) >= 1000/uL

Patient Q&A Section

What are the signs of medulloblastoma?

"Results from a recent paper provides a useful tool to parents and pediatricians working with children with brain tumors to assess the health status and possible manifestations of these cancers." - Anonymous Online Contributor

Unverified Answer

What are common treatments for medulloblastoma?

"Children's Hospitals' experience with treatment plans must be individualized on the basis of the children's age, tumor location and treatment history and their quality of life and physical demands." - Anonymous Online Contributor

Unverified Answer

What causes medulloblastoma?

"Medulloblastoma is extremely rare, but the number is being rapidly increasing in Western countries. There are few known environmental risk factors and the tumour-causing genetic alterations arise spontaneously in children. This cancer has two major clinical and biological subgroups that can have very different courses and results from therapy." - Anonymous Online Contributor

Unverified Answer

What is medulloblastoma?

"Medulloblastoma is a brain tumor that typically forms in the cerebellum. It generally appears in children between the ages 2 and 5, and can be fatal. Medulloblastoma is a spectrum of neuronal tissue tumors that are most frequently malignant but can also be benign. This cancer is often treated with cranial irradiation and chemotherapy.\n" - Anonymous Online Contributor

Unverified Answer

Can medulloblastoma be cured?

"We conclude that current chemotherapy protocols in pediatric oncology are highly effective for treatment, yet not curative, irrespective of the stage of disease. In this patient population chemotherapy alone can be curative for a subset of patients with localized disease." - Anonymous Online Contributor

Unverified Answer

How many people get medulloblastoma a year in the United States?

"The number of cases of medulloblastoma reported in the USA has increased in recent years. Although the reason for this remains unclear, there is no evidence of an increase in tumour age. Possible causes for the increase must be considered, including a greater sensitivity of detection by diagnostic investigations, earlier diagnosis, and early removal by surgery. In addition, the current OS was similar to those reported in other studies, while the incidence was almost double that of the UK, in contrast to a significantly higher rate of other childhood cancers." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in vincristine sulfate for therapeutic use?

"The newest drugs in vincristine are used to treat patients in a variety of malignancies including medulloblastoma. Vincristine is a chemotherapy drug that damages cells in the cell division cycle by blocking the ability of cells to produce DNA. The most commonly used dosage of vincristine sulfate is two to three milligrams per kilogram of body weight. Vincristine sulfate is sometimes given by intravenous infusion, but some newer forms of vincristine use a nasal spray to deliver the drug to the blood." - Anonymous Online Contributor

Unverified Answer

Does vincristine sulfate improve quality of life for those with medulloblastoma?

"Vincristine sulfate improves both QOL and PFS for children with newly diagnosed and refractory/recurrent medulloblastomas. Vincristine sulfate should be considered to improve the survival of these children." - Anonymous Online Contributor

Unverified Answer

Is vincristine sulfate typically used in combination with any other treatments?

"As the most common combination of vincristine sulfate is always infused concurrently with other drugs in pediatric patients, it is unlikely that our study design would adequately address the question of whether or not the additional treatment agents affect the toxicity and toxicity-related treatment-related adverse events of vincristine. However, the conclusions are of limited utility as vincristine sulfate may be used in isolation to treat children with medulloblastoma as part of a high-risk therapy regimen despite the fact that this does not result in any greater toxicity or risk of toxicity to other agents." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving vincristine sulfate?

"In this population-based study we found only one other vincristine sulfate clinical trial that was not a phase III study and did not report any of the above favorable outcomes. Other clinical trialists interested in conducting a phase III randomized trial of the effects of vincristine sulfate may need to consider obtaining more information about the positive findings of the only other existing clinical trial." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating medulloblastoma?

"Current modalities are adequate and have enabled many patients with medulloblastoma to survive 2 or more years after the initial resection of the tumor. [Advances in surgery and radiotherapy are improving survival for this disease (medulloblastoma). In recent years, treatment using high-dose chemotherapy before surgery has increased the success of treatment and increased patients' survival compared with the older treatment approach (neoadjuvant chemotherapy followed by surgery). More recently, the use of postoperative radiotherapy has been demonstrated to provide additional benefit in reducing the risk of recurrence and prolonging progression-free survival [medulloblastoma.org/medulloblastoma]." - Anonymous Online Contributor

Unverified Answer

How quickly does medulloblastoma spread?

"The histological and molecular biology characteristics of medulloblastoma suggest rapid spread of the tumor to distant sites. In addition, we found no evidence of a tumor-specific survival disparity between children and adults." - Anonymous Online Contributor

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