Selinexor for Pediatric Solid Tumors or Brain Tumors

No longer recruiting at 25 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Children's Oncology Group

Must not be taking: Corticosteroids, Investigational drugs, Anti-cancer agents

Disqualifiers: Pregnancy, Uncontrolled infection, Organ transplant, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: Up to 24 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the chemotherapy drug selinexor to determine the best dose and identify side effects in younger patients with recurrent or refractory solid tumors or brain tumors. The goal is to assess how well selinexor stops tumor growth by killing tumor cells, preventing their division, or stopping their spread. Young patients with these tumors who have not found success with other treatments might be suitable candidates. As a Phase 1 trial, this research aims to understand how selinexor works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial requires that you stop taking any other anti-cancer medications and investigational drugs before participating. If you are on corticosteroids, you need to be on a stable or decreasing dose for at least 7 days before joining the trial.

Is there any evidence suggesting that selinexor is likely to be safe for humans?

Research shows that selinexor is under study to determine its safety for treating solid tumors and brain tumors in children. Earlier studies in similar conditions have shown that selinexor can be effective, but assessing how well children tolerate it is crucial.

Selinexor is administered orally and has been tested in various types of childhood cancers. These early studies focused on determining the right dose and monitoring side effects. Some participants experienced side effects such as nausea, fatigue, and low blood counts, which are common with many cancer treatments. However, the studies aimed to manage these effects and establish a safe dosage.

As this is an early phase trial, the primary goal is to understand safety. Researchers closely monitor any side effects to ensure the treatment is as safe as possible for young patients. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for pediatric solid tumors or brain tumors, which often involve surgery, radiation, and chemotherapy, selinexor works by a unique mechanism. It targets a protein called XPO1, which plays a key role in transporting various molecules out of the cell nucleus. By inhibiting this protein, selinexor can potentially prevent cancer cells from growing and spreading. Researchers are excited about selinexor because this approach could offer a new way to tackle tumors that are resistant to traditional therapies. Plus, selinexor is taken orally, which might make it easier for young patients to undergo treatment compared to more invasive options.

What evidence suggests that selinexor might be an effective treatment for pediatric solid tumors or brain tumors?

Research has shown that selinexor, which participants in this trial will receive, may effectively treat difficult tumors. In earlier studies, selinexor slowed or even reduced tumor size in various solid tumors and leukemia cases. It was particularly effective in stopping the growth of some brain tumors. This suggests that selinexor might help control tumor growth by preventing cancer cells from multiplying or spreading. While more research is needed, these findings offer hope for those dealing with stubborn tumors. ^{1 2 3 6 7}

Who Is on the Research Team?

Julia Glade-Bender

Principal Investigator

COG Phase I Consortium

Are You a Good Fit for This Trial?

This trial is for young patients with solid tumors or high-grade gliomas that have returned or are not responding to treatment. They must be able to swallow tablets, have a body surface area (BSA) >= 0.84 m^2, and meet specific blood count and organ function criteria. Not eligible if they've had selinexor before, are pregnant/breastfeeding, on investigational drugs, have uncontrolled infections or certain medical conditions.

Inclusion Criteria

My condition is a high-grade, aggressive brain tumor that has returned or didn't respond to treatment.

Patients must meet specific blood count criteria

Patients must be able to swallow tablets whole

Exclusion Criteria

Patients with prior solid organ transplantation

I have severe unsteadiness or movement disorders.

I have macular degeneration, uncontrolled glaucoma, or cataracts.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive selinexor orally on a twice weekly or once weekly schedule. Treatment repeats every 28 days for up to 24 cycles.

Up to 24 months

Visits on days 1, 3, 8, 10, 15, 17 or days 1, 8, 15, 22 of each 28-day cycle

Follow-up

Participants are monitored for safety and effectiveness after treatment completion

30 days

What Are the Treatments Tested in This Trial?

Interventions

• Selinexor

Trial Overview The trial is testing the safety and optimal dosage of a chemotherapy drug called Selinexor in younger patients. This medication aims to stop tumor growth by killing cells or preventing them from dividing and spreading.

How Is the Trial Designed?

Group I: Treatment (selinexor) Experimental Treatment 2 Interventions

Patients receive selinexor PO on either a twice weekly (days 1, 3, 8, 10, 15, 17) or once weekly (days 1, 8, 15, and 22) schedule. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity.

Selinexor is already approved in United States, Canada for the following indications:

Approved in United States as Xpovio for:

• Multiple myeloma
• Diffuse large B-cell lymphoma

Approved in Canada as Xpovio for:

• Multiple myeloma

Who Is Running the Clinical Trial?

Children's Oncology Group

Lead Sponsor

Trials

467

Recruited

241,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12045713/

Phase 1 trial of selinexor in pediatric recurrent/refractory solid ... While outcomes have improved for most childhood cancers, treatment gains have been limited for a subset of children with aggressive non-hematologic malignancies ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4722540/

a report from the Pediatric Preclinical Testing Program - PMC Conclusions. Selinexor induced regression against several solid tumor and ALL xenografts and slowed tumor growth in a larger number of models. Pharmacodynamic ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT02323880

Study Details | NCT02323880 | Selinexor in Treating ... This phase I trial studies the side effects and best dose of selinexor in treating younger patients with solid tumors or central nervous system (CNS) tumors ...

4. scholars.uky.edu scholars.uky.edu/en/projects/acns1821-a-phase-12-trial-of-selinexor-kpt-330-and-radiation-ther

A Phase 1/2 Trial of Selinexor (KPT-330) and Radiation ... Abstract Pediatric oncology cure rates have risen dramatically over the last generation, but those for brain tumors still lag behind.

5. ascopubs.org ascopubs.org/doi/10.1200/JCO.2018.36.15_suppl.10552

Preclinical effect of selinexor (KPT-330), a selective ... In vivo treatment of two MRT PDXs with oral selinexor for 15 days significantly inhibited tumor growth in both models (p < 0.0001 and p = 0.0002). Conclusions: ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05985161

NCT05985161 | A Study of Selinexor in People With Wilms ... The purpose of this study is to find out whether selinexor is an effective treatment for people who have a relapsed/refractory Wilms tumor, rhabdoid tumor, ...

7. aacrjournals.org aacrjournals.org/clincancerres/article-pdf/doi/10.1158/1078-0432.CCR-21-2225/3019392/ccr-21-2225.pdf

A Phase II Study of the Efficacy and Safety of Oral Selinexor in ... It is overexpressed in many solid tumors, including gliomas, where its increased expression is associated with poor outcome (4–6). Selinexor is a novel, oral.