Tipifarnib for Spongioblastoma
This study is evaluating whether a drug called tipifarnib may help treat children with cancer.
See full descriptionAbout the trial for Spongioblastoma
Treatment Groups
This trial involves 2 different treatments. Tipifarnib 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.
About The Treatment
Side Effect Profile for Arm B (Clinical Observation)
Eligibility
This trial is for patients born any sex aged 65 and younger. You must have received 1 prior treatment for Spongioblastoma or one of the other 68 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.
Approximate Timelines
Measurement Requirements
This trial is evaluating whether Tipifarnib will improve 1 primary outcome, 2 secondary outcomes, and 2 other outcomes in patients with Spongioblastoma. Measurement will happen over the course of From enrollment to the end of treatment, up to 2 years.
Patient Q & A Section
Can spongioblastoma be cured?
Surgery after radiation plus chemotherapy did not prolong survival, although a possible prolongation of survival of about 22 months indicates that further attempts at posttreatment radiotherapy may be justified.
What are common treatments for spongioblastoma?
Survival rate for children with spongyblastoma is 50%. Overall, spongymblastoma treatment is typically surgery or radiation therapy, either alone or in addition to chemotherapy. Radiation can be used to spare vital structures in children with bilateral tumors to avoid chemotherapy side effects. In younger patients, chemotherapy can decrease recurrence rate, but it may also prolong survival and increase risks of dying if complications are not treatable. Treatment of older patients may be prudent after careful evaluation.
What causes spongioblastoma?
All types of brain tumours share some common causes; in the majority of cases, these do not apply. The only one that applies is radiation exposure. The underlying problem here is not cell malfunction, but it is with the cells' ability to repair the harm that radiation causes. This is true of both spongioblastoma and other types of brain tumour, particularly high grade astrocytomas. The common factor here is a lack of repair ability, and not a malfunctioning of the cells themselves. Hence the most important way that brain tumours can be avoided is if the damage can be repaired appropriately.
How many people get spongioblastoma a year in the United States?
There is a significant increase in the number of cases of SM diagnosed after 2000 compared with before this time. This may be due to an increase in cancer risk associated with exposure to UV light and/or UV-reflecting clothing. This time period coincided with the decline in incidence of melanoma and with a subsequent increase in the incidence of childhood cancers.
What is spongioblastoma?
This aggressive tumour forms at different ages, presents with a wide range of clinical features, has a poor prognosis and is now an incurable disease. spongioblastoma is a rare tumour which is the most common malignant central nervous system tumour in children. It has an annual incidence of 1 to 7 per 100,000 new cases. Most studies have examined the incidence of spongioblastoma in children but there have been few studies investigating the age distribution. The disease occurs mainly in children between the ages of two and five years. Spongioblastomas arise from immature cells from the central nervous system.
What are the signs of spongioblastoma?
The signs of SPG are nonspecific and may include pain, edema, and an inability to urinate, constipation or bowel movement, weight loss, vomiting, or a painful site or lesion.
What does tipifarnib usually treat?
tipifarnib is efficacious in a large number of patients with advanced or relapsed peripheral T-cell lymphomas. This agent should be considered for trials in patients with other solid tumors not amenable to conventional chemotherapy.
Is tipifarnib safe for people?
Tipifarnib is well tolerated by adolescents and young adults with relapsed or refractory childhood acute myeloid leukemia. Tipifarnib achieves responses in 85 % of patients and is a novel treatment option for patients with relapsed or refractory disease.
Does tipifarnib improve quality of life for those with spongioblastoma?
In this pilot study, tipifarnib significantly improved quality of life for patients compared with historical controls. Tipifarnib may help improve quality of life for select children undergoing treatment for SB.
Have there been any new discoveries for treating spongioblastoma?
[Current knowledge of CSC and the concept of targeted therapy of CSC is limited] Clinically, this has yet to generate a breakthrough and an effective new treatment of the primary tumor or in combination with other therapeutic modalities.
What is the primary cause of spongioblastoma?
The rarity of spongioblastoma at its most benign form is consistent with an aggressive, yet rare, primary precursor that would have to grow rapidly from cell division of progenitor cells that arise from normal stem cells which may be retained in the same stem cell niche as the precondition for both benign and malignant forms.
Does spongioblastoma run in families?
Significant clustering of familial spongioblastoma exists, implicating this tumor as a complex trait of multiple genetic modifiers. Genetic linkage analysis revealed that the familial spongioblastoma locus is at chromosome 14q24.1 and encodes a transcription factor, specifying the cell fate of SPS cells.