DSP-0390 for Brain Tumors

Recruiting at 1 trial location

Overseen By Omar H Butt, M.D., Ph.D.

Age: 18+

Sex: Any

Trial Phase: Phase < 1

Sponsor: Washington University School of Medicine

Must be taking: Antiepileptics, Corticosteroids

Must not be taking: CYP3A4 inhibitors, CYP2D6 inhibitors

Disqualifiers: Multifocal disease, Cardiovascular disease, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 2 weeks

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, DSP-0390, for treating certain brain tumors called IDH-mutant gliomas. Researchers aim to determine how the drug behaves in the body and whether it accumulates in the tumor at levels that can kill cancer cells. Participants will take DSP-0390 for about two weeks before undergoing surgery to remove the tumor. Individuals with IDH-mutant grade II or III gliomas who are already planning surgery might be suitable for this study. As an Early Phase 1 trial, this research focuses on understanding how DSP-0390 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 protocol does not specify if you must stop all current medications, but certain medications are prohibited. You cannot take strong or moderate CYP3A4 inhibitors or inducers, strong CYP2D6 inhibitors, or certain antiepileptic drugs like carbamazepine, phenytoin, and phenobarbital. If you are on antiepileptic medication or corticosteroids, you must be on a stable dose for a specified period before starting the trial.

Is there any evidence suggesting that DSP-0390 is likely to be safe for humans?

Previous studies have tested DSP-0390 in patients with brain tumors to assess its safety. So far, lab tests have shown promising results in fighting tumors. However, since this trial is in an early stage, complete safety information for humans is not yet available. This phase of the trial focuses on determining the right dose and identifying any side effects. While early signs are positive, researchers are still learning about its safety in people. Always consult a healthcare provider to understand what joining a trial might mean. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about DSP-0390 because it offers a novel approach for treating brain tumors. Unlike traditional treatments like surgery, radiation, and chemotherapy, DSP-0390 is an oral medication that patients take once daily. This drug might work by targeting cancer cells more precisely, potentially leading to fewer side effects and improved outcomes. In addition, the treatment is designed to be administered shortly before surgery, providing a unique opportunity to shrink tumors and make surgical removal more effective.

What evidence suggests that DSP-0390 might be an effective treatment for brain tumors?

Research has shown that DSP-0390 targets changes in cholesterol processing in certain brain tumors with the IDH mutation, potentially leading to tumor cell death. In lab tests, DSP-0390 effectively fights tumor cells. This trial will test DSP-0390 at two dosages, 120 mg and 240 mg, to ensure the drug reaches the brain tumor in the right amounts. Although information from human studies remains limited, these findings suggest that DSP-0390 could be a promising treatment for brain tumors with the IDH mutation. ^{2 3 4 6 7}

Who Is on the Research Team?

Omar H Butt, M.D., Ph.D.

Principal Investigator

Washington University School of Medicine

Are You a Good Fit for This Trial?

This trial is for patients with IDH-mutant WHO grade II or III glioma, a type of brain tumor. Participants must be scheduled for tumor resection surgery and willing to have tissue collected during the procedure. They should also agree to blood draws over two weeks of treatment.

Inclusion Criteria

Ability to understand and sign an IRB approved written informed consent document

Agreement to use adequate contraception by women of childbearing potential and men

I am eligible for surgery to remove a tumor larger than 8 cc.

Exclusion Criteria

Receiving other investigational agents, allergic reactions to compounds similar to DSP-0390

Pregnancy or breastfeeding, HIV with specific criteria, detectable viral load for hepatitis C or evidence of hepatitis B infection

My cancer has spread to multiple areas, including the brain's lining or beyond.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive DSP-0390 once daily by mouth for 2 weeks prior to surgical resection, with the final dose administered the morning of surgery. Blood samples are taken at various time points.

2 weeks

Surgical Resection

Tissue is collected during surgical resection to determine the pharmacokinetic and pharmacodynamic effect of DSP-0390 in brain and blood.

1 day

Follow-up

Participants are monitored for safety and effectiveness after treatment, including the incidence of adverse events.

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• DSP-0390

Trial Overview The study tests DSP-0390's effects in the brain and blood of glioma patients. It aims to see if this drug reaches effective levels in brain tumors and whether it can cause cancer cells to die by targeting cholesterol metabolism changes caused by mutant IDH.

How Is the Trial Designed?

Group I: DSP-0390 240 mg Experimental Treatment 1 Intervention

The next 10 patients will be assigned to DSP-0390 240 mg once daily by mouth for approximately 2 weeks and up to 17 days prior to surgical resection, with the final dose being administered the morning of surgery.

Group II: DSP-0390 120 mg Experimental Treatment 1 Intervention

The first 10 patients will be assigned to DSP-0390 120 mg once daily by mouth for approximately 2 weeks and up to 17 days prior to surgical resection, with the final dose being administered the morning of surgery.

Who Is Running the Clinical Trial?

Washington University School of Medicine

Lead Sponsor

Trials

2,027

Recruited

2,353,000+

Sumitomo Pharmaceuticals America

Industry Sponsor

Trials

5

Recruited

300+

Published Research Related to This Trial

Adding nitrosourea-based chemotherapy to radiotherapy has been shown to increase progression-free survival in patients with grade II and III gliomas, although it does not improve overall survival.

Upcoming phase III trials will investigate whether adding temozolomide to radiotherapy can enhance overall survival in grade II/III gliomas, while also assessing cognitive function and quality of life for better patient outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Temozolomide and radiation in low-grade and anaplastic gliomas: temoradiation. Schiff, D.[2018]

In a study of 101 patients with malignant glioma, only 27% were eligible for adjuvant stereotactic radiosurgery, and these eligible patients had significantly longer median survival times (23.4 months) compared to ineligible patients (8.6 months).

The findings suggest a substantial selection bias in uncontrolled trials of stereotactic radiosurgery, indicating that a phase III randomized study may not be necessary as it is unlikely to show positive results.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Measuring bias in uncontrolled brain tumor trials--to randomize or not to randomize? Irish, WD., Macdonald, DR., Cairncross, JG.[2019]

Malignant gliomas are challenging to treat, with surgery and radiation providing the most significant survival benefits, while chemotherapy offers only modest improvements.

The paper discusses the limited effectiveness of current chemotherapy agents and suggests that future treatments may rely on combination therapies that include both targeted biological compounds and traditional chemotherapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Chemotherapy in the treatment of malignant gliomas. Stern, JI., Raizer, JJ.[2007]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06636162

Window of Opportunity Study of DSP-0390 in Gliomas This study focuses on determining the pharmacokinetic and pharmacodynamic effect of DSP-0390 in brain and blood from patients with IDH-mutant WHO grade II ...

2. ascopubs.org ascopubs.org/doi/10.1200/JCO.2022.40.16_suppl.TPS2077

DSP-0390, an oral emopamil binding protein (EBP) ... DSP-0390 has shown significant antitumor activity in orthotopic xenograft models of human GBM (data on file). Methods: DSP-0390 will be ...

3. withpower.com withpower.com/trial/dsp-0390-for-brain-tumors-70f8f

DSP-0390 for Brain Tumors It aims to see if this drug reaches effective levels in brain tumors and whether it can cause cancer cells to die by targeting cholesterol metabolism changes ...

4. targetedonc.com targetedonc.com/view/fda-grants-orphan-drug-designation-to-dsp-0390-in-brain-cancer

FDA Grants Orphan Drug Designation to DSP-0390 in ... Currently, a phase 1 clinical trial (NCT05023551) is evaluating the safety and efficacy of DSP-0390 in patients with recurrent, high-grade ...

5. trials.braintumor.org trials.braintumor.org/trials/NCT05023551

Study of DSP-0390 in Patients With Recurrent High-Grade ... This study will evaluate the safety and efficacy of DSP-0390 in patients with recurrent high grade glioma. Arms & Interventions. Arms. Experimental: Single arm ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05023551

Study of DSP-0390 in Patients With Recurrent High-Grade ... This is a Phase 1 dose-escalation study with a Part 2 expansion to evaluate the safety, PK, PD, and preliminary antitumor activity of orally administered DSP ...

7. academic.oup.com academic.oup.com/neuro-oncology/article/24/Supplement_7/vii249/6826797

RTID-03. DSP-0390, AN ORAL EMOPAMIL BINDING ... DSP-0390 has shown significant antitumor activity in orthotopic xenograft models of human GBM (data on file). ... Global survival trends for brain ...