Nanvuranlat for Biliary Tract Cancer

(Beacon-BTC Trial)

Not yet recruiting at 18 trial locations

Overseen By Kazuo Sekiguchi

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: J-Pharma Co., Ltd.

Must not be taking: Strong inhibitors, Sensitive substrates

Disqualifiers: Active CNS metastases, Cardiovascular disease, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: 28

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called nanvuranlat for people with biliary tract cancer (BTC), which affects the bile ducts, gallbladder, or related areas. The trial aims to determine the best dosage and assess its effectiveness compared to standard treatments. Participants will receive either nanvuranlat or a treatment chosen by their doctor and will be monitored closely. This trial suits those with advanced BTC who have tried at least one previous platinum-based therapy without success. As a Phase 3 trial, this treatment is in the final step before FDA approval, offering participants access to a potentially groundbreaking therapy.

Will I have to stop taking my current medications?

The trial requires a washout period (time without taking certain medications) for some treatments before starting the study. If you are on strong inhibitors or sensitive substrates of specific transporters, you may need to switch to other medications. It's best to discuss your current medications with the study team to see if any changes are needed.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that nanvuranlat has a good safety record from previous studies. These studies found that nanvuranlat, which blocks a protein called LAT1, was well-tolerated by patients with advanced biliary tract cancer. Specifically, one study found that nanvuranlat helped patients live longer without their cancer worsening compared to those who took a placebo. It also maintained an acceptable safety level for participants.

While some side effects occurred, they were generally manageable, and serious side effects were rare. This suggests that nanvuranlat could be a promising option for patients, as earlier research supports its safety. Prospective participants in a clinical trial for nanvuranlat might find these findings reassuring regarding its safety in humans. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Nanvuranlat is unique because it offers a novel way to treat biliary tract cancer by focusing on different dosing strategies and delivery methods compared to current standards like FOLFOX and FOLFIRI regimens. While standard treatments involve a combination of chemotherapeutic agents, Nanvuranlat simplifies the approach with single-agent therapy delivered through varying infusion schedules. This drug's different dosing options, including a 46-hour continuous infusion, could potentially improve convenience and efficacy for patients. Researchers are excited because these features might enhance treatment outcomes and reduce side effects, offering a fresh hope for those battling this challenging cancer.

What evidence suggests that this trial's treatments could be effective for biliary tract cancer?

Research shows that nanvuranlat may help treat advanced biliary tract cancer. This trial will administer different dosages of nanvuranlat—50mg, 75mg, and 375mg—to evaluate its effectiveness. Studies have found that nanvuranlat can significantly delay cancer progression, improving progression-free survival (PFS). In earlier trials, nanvuranlat, compared to a placebo, positively affected patients who had already tried other treatments. It blocks certain proteins in cancer cells, potentially stopping cancer growth. These findings suggest nanvuranlat could be a promising option for people with this difficult-to-treat cancer. ^{1 2 6 7 8}

Who Is on the Research Team?

Eric K Rowinsky, MD

Principal Investigator

Uniphar Development, LLC

Ghassan Abou-Alfa, MD

Principal Investigator

Memorial Sloan Kettering Cancer Center

Are You a Good Fit for This Trial?

Adults aged 18 or older with biliary tract cancer, including bile duct and gallbladder cancers, are eligible for this trial. Participants will be chosen based on specific health criteria not detailed here.

Inclusion Criteria

Expected life expectancy of at least 90 days after the first day of treatment

Must have at least 1 measurable lesion by RECIST v1.1 based on imaging

Must be able and willing to comply with scheduled visits, treatment plans, procedures, and laboratory tests

Exclusion Criteria

Previously developed shock, anaphylaxis, or renal disorder due to SBECD

Known reaction or contraindication to any component of study intervention

Resting QTcF > 470 msec at screening

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Part A: Dose Regimen Selection

Participants are randomized to receive one of three nanvuranlat dose regimens or Physician's Best Choice (PBC).

14 days per cycle, ongoing until safety issues or disease progression

Visits every 14 days

Part B: Efficacy Evaluation

Participants receive the selected nanvuranlat dose regimen or PBC to evaluate efficacy and safety.

14 days per cycle, ongoing until safety issues or disease progression

Visits every 14 days

Follow-up

Participants are monitored for safety and effectiveness after treatment

Approx. 36 months

What Are the Treatments Tested in This Trial?

Interventions

• Nanvuranlat

Trial Overview The study is testing different doses of Nanvuranlat compared to the Physician's Best Choice (PBC) treatments like FOLFOX, FOLFIRI, or supportive care alone in two parts: dose selection (Part A) and evaluating overall survival (Part B).

How Is the Trial Designed?

Group I: Physicians Best Choice (PBC) Experimental Treatment 1 Intervention

1. FOLFOX regimen (Leucovorin, 5-Flurouracil and Oxaliplatin) administered on Day 1 of each 14 day cycle, or 2. FOLFIRI regimen (Leucovorin, 5-Flurouracil and Irinotecan) administered once during 14 day cycle, or 3. Best Supportive Care (BSC) including symptomatic therapeutics, palliative radiation for pre-existing metastases and transfusion of blood products administered at discretion of Investigator.

Group II: Navuranlat 75mg Experimental Treatment 1 Intervention

Nanvuranlat 75mg administered via a 90-minute intravenous infusion once daily for 5 consecutive days, followed by 9 days off treatment

Group III: Navuranlat 375mg Experimental Treatment 1 Intervention

Nanvuranlat 375mg administered via a 46-hour continuous intravenous infusion once every 14 days

Group IV: Nanvuranlat 50mg Experimental Treatment 1 Intervention

Nanvuranlat 50mg administered via a 90-minute intravenous infusion once daily for 5 consecutive days, followed by 9 days off treatment

Who Is Running the Clinical Trial?

J-Pharma Co., Ltd.

Lead Sponsor

Uniphar Development, LLC

Collaborator

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39058429/

A Phase II Placebo-Controlled Study of the Effect and ... Compared with placebo, nanvuranlat improved PFS in patients with advanced and refractory biliary tract cancer with an acceptable safety profile.

2. ascopubs.org ascopubs.org/doi/10.1200/JCO.2023.41.4_suppl.494

Nanvuranlat, an L-type amino acid transporter (LAT1) ... LAT1 inhibitor monotherapy with nanvuranlat demonstrated useful clinical efficacy in patients with four different subtypes of pre-treated, advanced, refractory ...

3. onclive.com onclive.com/view/nanvuranlat-significantly-improves-pfs-in-pretreated-advanced-biliary-tract-cancer

Nanvuranlat Significantly Improves PFS in Pretreated, ... Nanvuranlat, a first-in-class LAT1 inhibitor, induced a statistically significant improvement in progression-free survival compared with placebo.

4. aacrjournals.org aacrjournals.org/clincancerres/article/30/18/3990/747458/A-Phase-II-Placebo-Controlled-Study-of-the-Effect

A Phase II Placebo-Controlled Study of the Effect and Safety of ... Nanvuranlat demonstrated clinical efficacy with a statistically significant improvement in progression-free survival (PFS) with a favorable safety profile in ...

5. asco.org asco.org/abstracts-presentations/ABSTRACT410084

Subgroup analysis of double-blind, placebo ... Subgroup analysis of double-blind, placebo-controlled Ph. 2 study of nanvuranlat in treatment of pre-treated, advanced, refractory biliary tract cancer (BTC): ...

6. ascopubs.org ascopubs.org/doi/10.1200/JCO.2023.41.16_suppl.4011

Subgroup analysis of double-blind, placebo-controlled Ph. ... This subgroup analysis indicates that the efficacy of nanvuranlat in PFS and OS is enhanced in BTC patients with high LAT1 expression, when compared with ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT07265674

NCT07265674 | A Study to Select a Dose Regimen (Part A) ... Also called a data safety and monitoring board ... Safety of Nanvuranlat in Patients With Previously Treated Advanced Biliary Tract Cancer.

8. j-pharma.com j-pharma.com/en/archives/1735

J-Pharma presented Japanese Phase II Trial of ... Furuse said “Nanvuranlat, LAT1 Inhibitor, demonstrated a statistically significant progression-free survival with a good safety profile for ...