Trastuzumab + GM-CSF for Ependymoma
Trial Summary
What is the purpose of this trial?
This study plans to detect the presence of trastuzumab by mass spectroscopy in relapsed posterior fossa ependymoma (PFEPN) tumor specimen pre-treated with a single dose of intrathecal (IT) trastuzumab, as well as to evaluate toxicity of intrathecal trastuzumab in combination with subcutaneous (subQ) Granulocyte-macrophage colony-stimulating factor (GM-CSF) in children with relapsed PFEPN
Do I need to stop my current medications to join the trial?
Yes, you will need to stop certain medications. You cannot take systemic corticosteroids, investigational drugs, or other anti-cancer agents while participating in the trial.
Do I need to stop taking my current medications to join the trial?
Yes, you will need to stop taking certain medications. Specifically, you cannot be on systemic corticosteroids, other investigational drugs, or anti-cancer agents to participate in the trial.
What data supports the idea that Trastuzumab + GM-CSF for Ependymoma is an effective treatment?
The available research shows that Trastuzumab, when used for HER2-positive breast cancer with leptomeningeal metastases, has been effective in improving symptoms and slowing disease progression. For example, patients treated with intrathecal Trastuzumab experienced rapid symptom relief and remained stable for several months. Although this data is specific to breast cancer, it suggests that Trastuzumab can be effective in treating conditions involving the central nervous system. However, there is no direct data available for its use in Ependymoma, so its effectiveness for this condition remains uncertain.12345
What data supports the effectiveness of the drug Trastuzumab for treating leptomeningeal metastases in HER2-positive breast cancer?
What safety data exists for Trastuzumab + GM-CSF treatment?
The safety data for GM-CSF (Granulocyte-Macrophage Colony Stimulating Factor) includes potential side effects such as capillary leak syndrome, fever, skin rashes, and flu-like symptoms. In one case, a patient developed capillary leak syndrome during low-dose GM-CSF treatment, which was potentially fatal. GM-CSF has been used to treat chemotherapy-induced leukopenia and neutropenia, showing efficacy in increasing white cell counts but with noted side effects. Long-term safety, particularly regarding the risk of leukemia, has not been fully evaluated. There is no specific safety data for the combination of Trastuzumab and GM-CSF in ependymoma treatment in the provided research.678910
What safety data exists for Trastuzumab and GM-CSF treatments?
GM-CSF (also known as Sargramostim or Leukine) has been used in cancer treatments and can cause side effects like fever, skin rashes, and flu-like symptoms. In rare cases, it may lead to serious conditions like capillary leak syndrome, which involves fluid buildup in the body. Trastuzumab (also known as Herceptin) is generally considered safe but can have side effects like heart problems, so patients are usually monitored closely during treatment.678910
Is the drug GM-CSF, Trastuzumab a promising treatment for ependymoma?
Trastuzumab, when used in certain brain-related conditions like leptomeningeal carcinomatosis in breast cancer, has shown promise. It can be effective and safe, especially when administered directly into the spinal fluid, helping patients improve and manage their symptoms. This suggests that Trastuzumab could be a promising option for treating ependymoma, a type of brain tumor.1251112
How is the drug Trastuzumab + GM-CSF unique for treating ependymoma?
This treatment is unique because it combines trastuzumab, a drug that targets HER2-positive cancer cells, with GM-CSF, which stimulates the immune system, potentially offering a novel approach for ependymoma, a condition with limited standard treatment options. Trastuzumab has been used intrathecally (directly into the spinal fluid) in other cancers to overcome the blood-brain barrier, which may be relevant for treating brain-related conditions like ependymoma.1251112
Research Team
Margaret Macy, MD
Principal Investigator
University of Colorado, Denver
Eligibility Criteria
This trial is for children and young adults aged between 1 and 21 years with a specific type of brain tumor called relapsed posterior fossa ependymoma. They must have good organ function, no widespread cancer in the spine or brain, and be able to undergo surgery if needed. Participants need acceptable performance scores indicating they can perform daily activities.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single dose of intrathecal trastuzumab following a 5-day course of subcutaneous GM-CSF, with surgical resection planned 2-7 days after IT trastuzumab dosage.
Dose Finding
Participants receive IT trastuzumab in combination with GM-CSF to establish a maximum tolerated dosage.
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- GM-CSF
- Trastuzumab
GM-CSF is already approved in United States, European Union, Canada for the following indications:
- Neutropenia
- Bone Marrow Transplantation
- Leukemia
- Lymphoma
- HIV Infection
- Neutropenia
- Bone Marrow Transplantation
- Leukemia
- Lymphoma
- Neutropenia
- Bone Marrow Transplantation
- Leukemia
- Lymphoma
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Colorado, Denver
Lead Sponsor
Children's Hospital Colorado
Collaborator