Bintrafusp Alfa for Olfactory Neuroblastoma

Overseen By Charalampos Floudas, M.D.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: National Cancer Institute (NCI)

Must not be taking: Immunosuppressants

Disqualifiers: Autoimmune diseases, Cardiovascular disease, others

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

Prior Safety Data This treatment has passed at least one previous human trial

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

Compensation: Varies

Number of Visits: 28

Duration: 52 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if the immunotherapy drug bintrafusp alfa can shrink or eliminate olfactory neuroblastoma (ONB), a rare nasal cavity cancer that often spreads to the neck, lungs, and bones. Researchers are exploring a new treatment approach for ONB, particularly when it resists standard therapies. The trial seeks participants with ONB that has recurred or spread after initial treatments. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Do I have to stop taking my current medications for the trial?

The trial protocol does not specify if you need to stop taking your current medications. However, you cannot use immunosuppressive medications within 14 days before starting the study drug, except for certain types like inhaled or topical glucocorticoids. It's best to discuss your specific medications with the trial team.

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

Research has shown that bintrafusp alfa effectively targets tumors and is generally safe, according to previous studies. In earlier research, about 34.8% of patients experienced serious side effects, classified as grade 3 or higher adverse events. However, no life-threatening side effects, known as grade 5, occurred. This indicates that while some individuals had significant reactions, the most severe outcomes did not happen. Bintrafusp alfa is currently being tested for its potential to shrink or eliminate tumors in people with olfactory neuroblastoma. It is important to consult a healthcare provider to determine if joining a trial is appropriate. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard chemotherapy and radiation options for olfactory neuroblastoma, Bintrafusp Alfa offers a unique dual-action approach. This innovative treatment combines the blocking of PD-L1, a protein that tumors use to hide from the immune system, with the inhibition of TGF-beta, a molecule that can suppress immune responses and promote tumor growth. Researchers are excited because this dual mechanism could enhance the body's immune response against cancer cells more effectively than current treatments, potentially improving outcomes for patients with this rare cancer.

What evidence suggests that bintrafusp alfa might be an effective treatment for olfactory neuroblastoma?

Research has shown that bintrafusp alfa, a type of immunotherapy, may help treat certain cancers by blocking proteins that allow tumors to hide from the immune system. Early results suggest this drug can slow down or shrink tumors in some patients. In past studies, patients who received bintrafusp alfa lived for an average of 11.9 months. Some side effects occurred, but serious ones were uncommon. Although limited information exists on its use for olfactory neuroblastoma, the drug's action and early success in other cancers provide hope for treating this rare nasal cancer. Participants in this trial will receive bintrafusp alfa to evaluate its effectiveness for olfactory neuroblastoma. ^{1 2 3 5 6}

Who Is on the Research Team?

Charalampos Floudas, M.D.

Principal Investigator

National Cancer Institute (NCI)

Are You a Good Fit for This Trial?

Adults over 18 with recurrent or metastatic Olfactory Neuroblastoma (ONB) that's resistant to standard treatments, including a platinum agent. They must have good organ and marrow function, an ECOG performance status of <=2, and be able to consent. HIV-positive participants are eligible if stable on antiretroviral therapy with no recent opportunistic infections.

Inclusion Criteria

I can take care of myself but might not be able to do heavy physical work.

Ability of participant to understand and the willingness to sign a written informed consent document

Participants must use effective methods of contraception

Exclusion Criteria

I have never been removed from immunotherapy due to severe side effects.

I am not pregnant or breastfeeding.

I have not received any live vaccines in the last 4 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive bintrafusp alfa intravenously every 2 weeks for 26 doses

52 weeks

26 visits (in-person)

End of Treatment

Participants have an end of treatment visit within 7 days after stopping the study drug

1 week

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with visits every 3 months for the first year, then every 6 months for years 2-5, and then once a year after that

Long-term

Regular visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Bintrafusp Alfa

Trial Overview The trial is testing Bintrafusp alfa, an immunotherapy drug given every two weeks for up to 26 doses. It aims to see if this treatment can shrink or eliminate ONB tumors. Participants will undergo various tests and may opt for tumor biopsies during the study.

How Is the Trial Designed?

Group I: 1/Arm 1: Treatment with Bintrafusp Alfa Experimental Treatment 10 Interventions

Treatment with Bintrafusp alfa

Bintrafusp Alfa is already approved in United States, European Union for the following indications:

Approved in United States as Bintrafusp Alfa for:

• Non-small cell lung cancer (NSCLC)

Approved in European Union as Bintrafusp Alfa for:

• Biliary tract cancer

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials

14,080

Recruited

41,180,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05012098

NCT05012098 | Phase 2 Study of Bintrafusp Alfa in ... Olfactory neuroblastoma (ONB) is a rare cancer of the nasal cavity. At diagnosis, it is usually locally advanced. It tends to spread to the neck. Sometimes it ...

2. clinicalstudies.info.nih.gov clinicalstudies.info.nih.gov/protocoldetails.aspx?id=000030-C&&query=Nasal%20Cavity%20Olfactory%20Neuroblastoma

Phase 2 Study of Bintrafusp alfa in Recurrent/Metastatic ... Participants will receive bintrafusp alfa once every 2 weeks for 26 doses. They will get it intravenously over 60 minutes. They may get other medicines to ...

3. withpower.com withpower.com/trial/phase-3-esthesioneuroblastoma-olfactory-9-2021-42cb9

Bintrafusp Alfa for Olfactory Neuroblastoma This trial is testing bintrafusp alfa, an immunotherapy drug, in adults with a rare nasal cancer called olfactory neuroblastoma that hasn't improved with ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S2772610X23001344

Olfactory neuroblastoma in children and adolescents A clinical trial to assess the effectiveness of bintrafusp alfa (NCT05012098) is currently open for adult patients with recurrent or metastatic ON. 5 ...

5. synapse.patsnap.com synapse.patsnap.com/drug/8628322022fc41429fa3da8a4f6098f5

Bintrafusp alfa - Drug Targets, Indications, Patents The median OS was 11.9 months (95 % CI, 5.8-15.7 months). Grade 3 or higher adverse events were observed in 34.8 % of patients and no grade 5 adverse event was ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8156979/

Bintrafusp alfa (M7824), a bifunctional fusion protein targeting ... Bintrafusp alfa has demonstrated antitumor activity and a manageable safety profile in the dose-escalation part and multiple expansion cohorts of this phase I ...