What side effects are associated with this type of intervention?

Common treatments for medulloblastoma, such as chemotherapy and radiation therapy, often result in side effects including fatigue, nausea, vomiting, hair loss, and increased risk of infections due to bone marrow suppression. Targeted therapies, like CK2 inhibitors such as Silmitasertib Sodium, may also cause gastrointestinal disturbances, liver enzyme abnormalities, and fatigue. These side effects can vary in severity and may require supportive care to manage.

What prior FDA approvals exist?

The FDA has approved several treatments for medulloblastoma, including traditional chemotherapy agents like cisplatin, cyclophosphamide, and vincristine, as well as radiation therapy. Targeted therapies and novel agents are also being explored, but specific FDA approvals for CK2 inhibitors like Silmitasertib Sodium in medulloblastoma are not well-documented. Broadly, the treatment landscape includes a combination of surgery, radiation, and chemotherapy, with ongoing research into targeted molecular therapies.

What other types of research exist?

Promising novel alternatives to Silmitasertib Sodium for Medulloblastoma patients include: 1) Sonic Hedgehog (SHH) pathway inhibitors such as Vismodegib and Sonidegib, which target the SHH signaling pathway involved in some medulloblastomas. 2) Immune checkpoint inhibitors like Pembrolizumab, which have shown potential in treating various cancers, including brain tumors. 3) Targeted therapies like Dabrafenib and Trametinib for patients with BRAF V600E mutations. These alternatives are in various stages of clinical trials and have shown potential efficacy in treating medulloblastoma.

← Back to Search

Protein Kinase Inhibitor

CX-4945 for Medulloblastoma

Phase 1 & 2

Waitlist Available

Led By Ralph Salloum, MD

Research Sponsored by Pediatric Brain Tumor Consortium

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

All Phases: Must have a diagnosis of SHH medulloblastoma that is recurrent or progressive which was confirmed histologically and subgrouping was completed using a CLIA certified methylation based test.

Phase II Skeletally-mature: Patients must be skeletally-mature, defined as females with a bone age ≥14 years and males with a bone age ≥ 16 years OR have a chronological age >18 years. Must have bi-dimensionally measurable disease.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 3 years from enrollment

Awards & highlights

No Placebo-Only Group

Summary

This trial tests an oral drug called CX-4945 for patients with a recurring type of brain tumor. The drug aims to block a protein that helps cancer cells grow. The study will determine the best dose and check for side effects. CX-4945 has shown promising results in other types of cancer and may be developed as a new therapy.

Who is the study for?

This trial is for patients with recurrent SHH medulloblastoma, a type of brain cancer. Participants must be skeletally mature or over 18 years old, have measurable disease, and have undergone prior treatments including radiation. They should not be nursing mothers or have other cancers (unless in remission for 5+ years), significant unrelated illnesses, certain gastrointestinal disorders, or an abnormal heart rhythm (QTc >480ms).

What is being tested?

The trial is testing the safety and effects of CX-4945 (silmitasertib sodium) on patients with recurrent SHH medulloblastoma. It includes both Phase I and II stages as well as a surgical study where some participants may undergo surgery.

What are the potential side effects?

While specific side effects are not listed here, common ones associated with cancer drugs like CX-4945 could include nausea, fatigue, liver issues, changes in blood counts leading to increased infection risk or bleeding problems. Patients will be monitored closely for any adverse reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My medulloblastoma is recurring or worsening and was confirmed with specific tests.

Select...

I am an adult or a teen with mature bones and my disease can be measured in two ways.

Select...

I am under 18, my growth plates are not fully developed, and my body size fits the study requirements.

Select...

I need surgery, am over 3 years old, and can take CX-4945 before my surgery.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 3 years from enrollment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 3 years from enrollment

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 3 years from enrollment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Phase I: Maximum tolerated dose of CX-4945

Phase I: Plasma pharmacokinetics of CX-4945 in skeletally-immature children

Phase II: Sustained objective response rate (PR-CR) rate in the skeletally mature cohort

+1 more

Secondary study objectives

Objective response rate in the skeletally-immature cohort

Plasma pharmacokinetics of CX-4945 in skeletally-mature subjects

Progression free survival

+2 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: SurgicalExperimental Treatment1 Intervention

Subjects who are eligible for the Phase I or Phase II arm of the trial and are candidates for surgery, may be enrolled in the surgical arm prior to initiation of the Phase I or Phase II treatment.

Group II: Phase II - Skeletally-matureExperimental Treatment1 Intervention

Skeletally-mature subjects with refractory or recurrent medulloblastoma of the SHH group

Group III: Phase I - Skeletally-immatureExperimental Treatment1 Intervention

Skeletally-immature children with refractory or recurrent medulloblastoma of the SHH group

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Medulloblastoma treatments often target specific molecular pathways involved in tumor growth and survival. Silmitasertib Sodium, for example, inhibits CK2, a protein kinase that regulates cell proliferation and survival. By inhibiting CK2, Silmitasertib disrupts these processes, potentially reducing tumor growth. This is crucial for Medulloblastoma patients as it offers a targeted approach that may be more effective and have fewer side effects compared to traditional therapies. Other common treatments include chemotherapy, which targets rapidly dividing cells, and radiation therapy, which damages the DNA of cancer cells. Targeted therapies like CK2 inhibitors are particularly promising as they aim to minimize damage to healthy cells while effectively combating the tumor.