CLINICAL TRIAL

Tipifarnib for Spongioblastoma

1 Prior Treatment
Locally Advanced
Metastatic
Refractory
Relapsed
Recruiting · < 65 · All Sexes · Cleveland, OH

This study is evaluating whether a drug called tipifarnib may help treat children with cancer.

See full description

About the trial for Spongioblastoma

Eligible Conditions
Recurrent Hepatoblastoma · Recurrent Neuroblastoma · Recurrent Adrenal Gland Pheochromocytoma · Ependymoma, Recurrent · Recurrent Ectomesenchymoma · Recurrent Osteosarcoma · recurrent Ewing's Sarcoma · Recurrent Peripheral Primitive Neuroectodermal Tumor · Refractory WHO Grade 2 Glioma · Recurrent Medulloblastoma · Recurrent Melanoma · Histiocytosis, Langerhans-Cell · Thyroid Neoplasms · Kidney Neoplasms · Recurrent Non-Hodgkin Lymphoma · Thyroid Diseases · Malignant Solid Neoplasms · Recurrent Kidney Wilms Tumor · Recurrent Langerhans Cell Histiocytosis · Recurrent Malignant Gliomas · Recurrent Malignant Germ Cell Tumor · Recurrent Rhabdoid Tumor · Recurrent WHO Grade 2 Glioma · Refractory Melanoma · Histiocytosis · Melanoma · Sarcoma · Lymphoma, Non-Hodgkin · Glioma · Neoplasms · Ependymoma · Sarcoma, Ewing · Medulloblastoma · Neuroectodermal Tumors · Neuroectodermal Tumors, Primitive · Wilms Tumor · Pheochromocytoma · Neuroectodermal Tumors, Primitive, Peripheral · Rhabdoid Tumor · Hepatoblastoma · Recurrent Rhabdoid Tumor of the Kidney · Recurrent Rhabdomyosarcoma · Recurrent Soft Tissue Sarcoma · Recurrent Thyroid Gland Carcinoma · Recurrent WHO Grade II Glioma · Refractory Adrenal Gland Pheochromocytoma · Refractory Ependymoma · Refractory Ewing Sarcoma · Refractory Hepatoblastoma · Refractory Langerhans Cell Histiocytosis · Refractory Malignant Germ Cell Tumor · Refractory Malignant Glioma · Refractory Medulloblastoma · Refractory Neuroblastoma · Refractory Non-Hodgkin Lymphoma · Refractory Osteosarcoma · Refractory Peripheral Primitive Neuroectodermal Tumor · Refractory Rhabdoid Tumor · Refractory Rhabdoid Tumor of the Kidney · Refractory Rhabdomyosarcoma · Refractory Soft Tissue Sarcomas · Refractory Thyroid Gland Carcinoma · Refractory WHO Grade II Glioma · Lymphoma · Carcinoma · Neuroblastoma · Neoplasms, Germ Cell and Embryonal · Osteosarcoma · Rhabdomyosarcoma

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.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Tipifarnib
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tipifarnib
Not yet FDA approved

Side Effect Profile for Arm B (Clinical Observation)

Arm B (Clinical Observation)
Show all side effects
19%
Anemia
17%
White blood cell decreased
16%
Platelet count decreased
1%
Fatigue
0%
GGT increased
0%
Acute coronary syndrome
0%
Nausea
0%
Febrile neutropenia
0%
Infections and infestations - Other, specify
0%
Lung infection
0%
Aspartate aminotransferase increased
0%
Peripheral nerve infection
0%
Sinusitis
0%
Alanine aminotransferase increased
0%
Hypokalemia
0%
Confusion
0%
Agitation
0%
Peripheral sensory neuropathy
0%
Psychosis
0%
Treatment related secondary malignancy
0%
Eye disorders - Other, specify
0%
Neutrophil count decreased
0%
Rash maculo-papular
0%
Diarrhea
0%
Catheter related infection
0%
Back pain
0%
Myalgia
Anemia
19%
White blood cell decreased
17%
Platelet count decreased
16%
Fatigue
1%
GGT increased
0%
Acute coronary syndrome
0%
Nausea
0%
Febrile neutropenia
0%
Infections and infestations - Other, specify
0%
Lung infection
0%
Aspartate aminotransferase increased
0%
Peripheral nerve infection
0%
Sinusitis
0%
Alanine aminotransferase increased
0%
Hypokalemia
0%
Confusion
0%
Agitation
0%
Peripheral sensory neuropathy
0%
Psychosis
0%
Treatment related secondary malignancy
0%
Eye disorders - Other, specify
0%
Neutrophil count decreased
0%
Rash maculo-papular
0%
Diarrhea
0%
Catheter related infection
0%
Back pain
0%
Myalgia
0%
This histogram enumerates side effects from a completed undefined Phase 3 trial (NCT00093470) in the Arm B (Clinical Observation) ARM group. Side effects include: Anemia with 19%, White blood cell decreased with 17%, Platelet count decreased with 16%, Fatigue with 1%, GGT increased with 0%.

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.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Patient must have enrolled onto APEC1621SC and must have been given a treatment assignment to MATCH to APEC1621M based on the presence of an actionable mutation as defined in APEC1621SC
You must have a body surface area > 0.29 m^2 at enrollment. show original
Patients must have radiographically measurable disease at the time of study enrollment. Patients with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine (MIBG) positive (+) evaluable disease are eligible. Measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on standard magnetic resonance imaging (MRI) or computed tomography (CT)
Bone marrow infiltration except that detected by MIBG scan for neuroblastoma. show original
The tumor markers in the plasma or CSF are elevated. show original
You have previously had radiation therapy for cancer. show original
A leptomeningeal lesion that does not meet the measurement requirements for RECIST 1. show original
Karnofsky >= 50 for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age. Note: Neurologic deficits in patients with CNS tumors must have been relatively stable for at least 7 days prior to study enrollment. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
You have malignant fluid collections (e.g., ascites, pleural effusions) that are associated with cancer. show original
The diagnosis must be based on clinical findings, and imaging studies are not required. show original
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial

Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Up to 7 years
Screening: ~3 weeks
Treatment: Varies
Reporting: Up to 7 years
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Up to 7 years.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

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.

Percentage of patients experiencing grade 3 or higher adverse events
FROM ENROLLMENT TO THE END OF TREATMENT, UP TO 2 YEARS
Percentage of patients experiencing grade 3 or higher adverse events will be evaluated according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. All patients who receive at least one dose of protocol therapy will be considered in the evaluation of toxicity.
FROM ENROLLMENT TO THE END OF TREATMENT, UP TO 2 YEARS
Objective response rate (complete response + partial response) in pediatric patients treated with tipifarnib
FROM ENROLLMENT TO THE END OF TREATMENT, UP TO 2 YEARS
A responder is defined as a patient who achieves a best response of partial response or complete response on the study. Response rates will be calculated as the percent of evaluable patients who are responders, and confidence intervals will be constructed using the Wilson score interval method.
FROM ENROLLMENT TO THE END OF TREATMENT, UP TO 2 YEARS
Progression free survival (PFS)
FROM THE INITIATION OF SUBPROTOCOL TREATMENT, UNTIL DISEASE PROGRESSION OR DISEASE RECURRENCE OR DEATH FROM ANY CAUSE, ASSESSED UP TO 7 YEARS
PFS along with the confidence intervals will be estimated using the Kaplan-Meier method.
FROM THE INITIATION OF SUBPROTOCOL TREATMENT, UNTIL DISEASE PROGRESSION OR DISEASE RECURRENCE OR DEATH FROM ANY CAUSE, ASSESSED UP TO 7 YEARS
Profiling changes in tumor genomics
BASELINE, ASSESSED UP TO 7 YEARS
Will explore approaches to the profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid.
BASELINE, ASSESSED UP TO 7 YEARS
Biomarker analysis
UP TO 7 YEARS
Will evaluate other biomarkers as predictors of response to tipifarnib and whether tumors that harbor different missense mutations or fusions will demonstrate differential response to treatment.
UP TO 7 YEARS

Patient Q & A Section

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

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer
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