Quizartinib for Liver Disease

Recruiting at 3 trial locations

Overseen By Daiichi Sankyo Contact for Clinical Trial Information

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Daiichi Sankyo

Disqualifiers: Primary biliary cirrhosis, Gilbert's syndrome, 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: 1 day

12 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called quizartinib (also known as Vanflyta) to compare its behavior in individuals with severe liver problems to those with healthy liver function. The goal is to understand the drug's pharmacokinetics, or how it moves through the body, in these two groups. Participants will receive a single oral dose of the medication. This trial may suit individuals with severe liver issues who do not have other serious health conditions and are open to trying a new treatment. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new medication.

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 coordinator or your doctor.

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

Research has shown that quizartinib is generally safe and well-tolerated. Since its approval in the United States, no reports have indicated serious liver problems caused by this drug. This is encouraging for those considering participation in a clinical trial, especially one focused on liver health.

However, quizartinib has not been specifically tested in individuals with severe liver problems. While the drug is generally safe, this trial aims to provide more information about its effects on people with serious liver issues.

In previous studies, some serious side effects, such as infections and brain swelling, occurred when quizartinib was combined with other treatments. When used alone, quizartinib has maintained a reassuring safety record. This trial will further assess its safety in individuals with severe liver conditions. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for liver disease?

Quizartinib is unique because it brings a fresh approach to tackling liver disease. Unlike traditional treatments that often focus on managing symptoms or slowing disease progression, quizartinib targets specific molecular pathways, potentially offering more precise and effective results. Researchers are particularly excited about its ability to directly target cancer cells, thanks to its action as a FLT3 inhibitor, which is different from most liver disease treatments. This novel mechanism offers hope for improved outcomes, especially for patients who have limited options with current therapies.

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

Quizartinib has mainly been studied in people with acute myeloid leukemia (AML), a type of blood cancer. Research shows it can help treat AML by targeting certain cancer cells. However, no information exists yet on its effectiveness for liver disease, especially in people with serious liver problems. In this trial, participants with severe hepatic impairment will receive a single oral dose of 30 mg quizartinib, while a control group of healthy participants will receive the same dose. The study aims to learn how quizartinib acts in the body when the liver isn't working properly. This could provide insights into its possible use for liver issues, but its effectiveness for these conditions remains unknown. ^{1 2 3 6 7}

Who Is on the Research Team?

Global Clinical Leader

Principal Investigator

Daiichi Sankyo

Are You a Good Fit for This Trial?

This trial is for men and women aged 18-75 with severe liver impairment, a BMI of 18-37, and at least 40 kg in weight. Women must not undergo egg retrieval or donate eggs during the study and for seven months after. Men must use contraception or be surgically sterilized. Participants cannot have certain liver diseases, serious medical conditions that could affect the study's safety or results, recent significant illness, known allergies to Quizartinib ingredients, or a history of digestive surgery affecting drug absorption.

Inclusion Criteria

In females, agreement to not retrieve eggs/ova via assisted reproductive technology (ART) either for their own use or donation while on the study or for 7 months after the last dose of study drug, whichever is later

Voluntarily consents to participate in this study and provides written informed consent before the start of any study-specific procedures

I am a male and have been sterilized, practice abstinence, or agree to use contraception during and up to 4 months after treatment.

Exclusion Criteria

I have been diagnosed with primary biliary cirrhosis or primary sclerosing cholangitis.

Any serious and/or unstable pre-existing medical, psychiatric disorder, or other conditions (including lab abnormality except hepatic impairment) that could interfere with safety, obtaining informed consent, compliance to the study procedures, or the validity of the study results

Presence or history of clinically severe adverse reaction to any drug or known hypersensitivity to any of the ingredients (including inactive ingredients) of quizartinib

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 30 mg quizartinib

1 day

1 visit (in-person)

Follow-up

Participants are monitored for pharmacokinetics, safety, and tolerability

30 days

Multiple visits (in-person and/or virtual)

What Are the Treatments Tested in This Trial?

Interventions

• Quizartinib

Trial Overview The trial is testing Quizartinib pharmacokinetics (how the drug moves through the body) in subjects with severe hepatic impairment compared to healthy individuals. It aims to understand how liver disease affects the processing of this medication.

How Is the Trial Designed?

Group I: Severe HI Experimental Treatment 1 Intervention

Participants will receive a single oral dose of 30 mg quizartinib

Group II: Control Group Experimental Treatment 1 Intervention

Healthy participants will receive a single oral dose of 30 mg quizartinib

Quizartinib is already approved in United States, Japan for the following indications:

Approved in United States as Vanflyta for:

• Acute myeloid leukemia (AML)

Approved in Japan as Vanflyta for:

• Acute myeloid leukemia (AML)

Who Is Running the Clinical Trial?

Daiichi Sankyo

Lead Sponsor

Trials

443

Recruited

493,000+

Hiroyuki Okuzawa

Daiichi Sankyo

Chief Executive Officer

Degree in Social Sciences from Hitotsubashi University

Yuki Abe

Daiichi Sankyo

Chief Medical Officer since 2023

MD

Citations

1. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK605695/

Quizartinib - LiverTox - NCBI Bookshelf In the prelicensure clinical trials of quizartinib in patients with AML, ALT elevations were arose in 10% to 16% of patients and were above 5 ...

2. vanflytahcp.com vanflytahcp.com/en/efficacy

Efficacy | VANFLYTA® (quizartinib) | HCP VANFLYTA has not been studied in patients with severe hepatic impairment. To report SUSPECTED ADVERSE REACTIONS, contact Daiichi Sankyo, Inc, at 1-877-437-7763 ...

3. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2023/216993Orig1s000MultidisciplineR.pdf

center for drug evaluation and research - accessdata.fda.gov Quizartinib was previously evaluated under NDA 212166 for the treatment of ... Efficacy of quizartinib was supplemented by data from Study QuANTUM-R.

4. ema.europa.eu ema.europa.eu/en/documents/assessment-report/vanflyta-epar-refusal-public-assessment-report_en.pdf

Vanflyta - European Medicines Agency See appendix 1. Outcome. Based on the CHMP review of data on quality, safety and efficacy for Vanflyta in the treatment of adult patients ...

5. theglobeandmail.com theglobeandmail.com/investing/markets/stocks/DSKYF/pressreleases/35744364/daiichi-sankyos-quizartinib-study-a-potential-game-changer-for-hepatic-impairment-treatment/

Daiichi Sankyo's Quizartinib Study: A Potential Game- ... ' The study aims to evaluate the pharmacokinetics (PK), safety, and tolerability of Quizartinib in individuals with severe hepatic impairment ...

6. daiichisankyo.us daiichisankyo.us/prescribing-information-portlet/getPIContent?productName=Vanflyta&inline=true

highlights of prescribing information Fatal adverse reactions occurred in 10% of patients who received VANFLYTA plus chemotherapy, including sepsis (5%), fungal infections (0.8%), brain edema (0.8% ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT04473664

A Study of Quizartinib Pharmacokinetics in Participants ... The primary objective of this study is to determine the plasma pharmacokinetics (PK) of quizartinib and its pharmacologically active metabolite AC886 after a ...