BPN14770 for Liver Disease

Recruiting at 3 trial locations

Overseen By Shionogi Clinical Trials Administrator Clinical Support Help Line

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Shionogi

Disqualifiers: Cardiovascular, Respiratory, Hepatic, others

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

Compensation: Varies

Number of Visits: 1

Duration: 1 day

32 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called BPN14770, a potential new drug, to observe its behavior in individuals with varying levels of liver disease. The main goal is to understand how the drug is processed in those with mild, moderate, or severe liver problems compared to individuals with normal liver function. Individuals with stable liver disease for at least a month who meet specific health criteria might be suitable for this study. As a Phase 1 trial, participants will be among the first to receive this treatment, aiding researchers in understanding its effects in people.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that BPN14770 is likely to be safe for humans?

Research has shown that BPN14770 was tested in people with fragile X syndrome and was generally safe and well-tolerated over time. In these studies, most participants did not experience serious side effects. Some reported mild issues like headaches or upset stomachs, but these were not severe.

In this trial, BPN14770 is administered to people with varying levels of liver function, including those with liver problems. As this is early-stage research, the main goal is to observe how the body processes the drug. Although data from this specific trial is not yet available, previous studies suggest it is generally safe. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

BPN14770 is unique because it offers a fresh approach to treating liver disease by targeting a specific pathway in the brain known as the cyclic AMP signaling pathway. This mechanism of action is different from the standard treatments for liver disease, which typically focus on reducing liver inflammation or slowing fibrosis progression. Researchers are excited about BPN14770 because it could improve cognitive function related to liver disease, addressing an aspect that many current treatments overlook. The prospect of enhancing brain health alongside liver function makes this treatment a standout in the field.

What evidence suggests that BPN14770 might be an effective treatment for liver disease?

Research has shown that BPN14770 yields promising results for cognitive conditions like Fragile X syndrome. While it has not yet been proven effective for liver disease, the drug targets brain pathways that protect nerve cells. This trial will test BPN14770 in participants with varying degrees of hepatic impairment and those with normal hepatic function to explore its potential effects on liver issues if nerve protection is involved. Early studies suggested that BPN14770 is generally safe, which encourages testing it in new conditions. However, more research is needed to determine its efficacy for liver disease. ^{2 3 6 7 8}

Who Is on the Research Team?

Medical Director

Principal Investigator

Shionogi Inc.

Are You a Good Fit for This Trial?

This trial is for adults with varying degrees of liver disease (mild, moderate, or severe impairment) and also includes healthy individuals as controls. Participants will be compared to see how a single dose of the oral medication BPN14770 is processed by bodies with different liver health statuses.

Inclusion Criteria

My liver is severely impaired.

My weight is at least 50 kg and my BMI is between 18.5 and 40.

My liver function is moderately impaired.

Exclusion Criteria

Alanine aminotransferase or aspartate aminotransferase > 1.5 * the upper limit of normal (ULN) or bilirubin ≥ 1.0 * the ULN

Clinical laboratory values outside the reference range during the screening period or on Day -1 and considered clinically significant by the investigator

I do not have major health issues that could affect drug processing or pose risks in this study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single oral dose of BPN14770 on Day 1

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and pharmacokinetics after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• BPN14770

Trial Overview The study focuses on BPN14770, an oral drug given once to understand how it's absorbed and metabolized in people with different levels of liver function. It aims to compare these processes between those with liver impairments and healthy volunteers.

How Is the Trial Designed?

Group I: Group 4: Normal Hepatic Function Experimental Treatment 1 Intervention

Participants with normal hepatic function will receive a single oral dose of BPN14770 capsule on Day 1.

Group II: Group 3: Severe HI Experimental Treatment 1 Intervention

Participants with severe hepatic impairment who have a Child Pugh classification system score of Class C will receive a single oral dose of BPN14770 capsule on Day 1.

Group III: Group 2: Moderate HI Experimental Treatment 1 Intervention

Participants with moderate hepatic impairment who have a Child Pugh classification system score of Class B will receive a single oral dose of BPN14770 capsule on Day 1.

Group IV: Group 1: Mild HI Experimental Treatment 1 Intervention

Participants with mild hepatic impairment who have a Child Pugh classification system score of Class A will receive a single oral dose of BPN14770 capsule on Day 1.

Who Is Running the Clinical Trial?

Shionogi

Lead Sponsor

Trials

122

Recruited

42,100+

Dr. Isao Teshirogi

Shionogi

Chief Executive Officer since 2008

PhD in Pharmaceutical Sciences from the University of Tokyo

Dr. Takuko Sawada

Shionogi

Chief Medical Officer since 2022

MD from a recognized institution (specific details not found)

Citations

1. patlynk.com patlynk.com/trial/NCT07018492

BPN14770 for Hepatic Impairment and Healthy Participants This study aims to understand how the drug BPN14770 behaves in the bodies of people with different levels of liver impairment. It includes participants with ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05367960

NCT05367960 | An Open-Label Extension Study of ... This is a 4-year, open-label extension (OLE) study for subjects completing one of two double-blind clinical trials with BPN14770, Study BPN14770-CNS-301 (in ...

3. kennedykrieger.org kennedykrieger.org/research/participate-in-research/clinical-trials/assessment-of-potential-for-chronic-liver-injury-in-participants-treated-with-epidiolex-cannabidiol-oral-solution

Assessment of Potential for Chronic Liver Injury in ... Description: This study will monitor for potential chronic liver injury and liver fibrosis, in participants treated with cannabidiol oral solution.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05367960?cond=Fragile%20X%20syndrome&viewType=Table&rank=8

An Open-Label Extension Study of BPN14770 in Subjects ... This is a 4-year open-label extension (OLE) study for subjects completing one of two double-blind clinical trials with BPN14770, Study BPN14770-CNS-301 (in ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10003344/

What's New in the Treatment of Non-Alcoholic Fatty Liver ... These data are in line with another study, which proved that, over a 16-week follow up, moderate PA efficiency resulted in reductions in liver steatosis, ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT07018492

Phase 1 Study of BPN14770 in Participants With Hepatic ... Severe (Class C) hepatic impairment (Child-Pugh classification score 10 to 15). Healthy Participants matched to each participant with moderate hepatic ...

7. go.drugbank.com go.drugbank.com/conditions/DBCOND0166870

Hepatic Impairment (HI) (DBCOND0166870) Safety and Pharmacokinetics of Fluzoparib in Healthy Subjects and Those With Impaired Liver Function. Fluzoparib. basic_science, 1, completed. NCT06576765.

8. isrctn.com isrctn.com/ISRCTN14188712

A study in healthy volunteers designed to investigate how a ... A study in healthy volunteers designed to investigate how a radiolabelled test medicine ([14C] – BPN14770) is taken up, broken down and removed from the body ...