Palovarotene for Liver Disease

Recruiting at 73 trial locations

Overseen By Ipsen Clinical Study Enquiries

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Ipsen

Must not be taking: CYP3A4 inhibitors, CYP3A4 inducers

Disqualifiers: Cardiovascular, Respiratory, Renal, others

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

Compensation: Varies

Number of Visits: 15

Duration: 5 days

32 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to understand how the body processes a single dose of palovarotene, a potential treatment, in individuals with varying liver function levels. Researchers will compare those with moderate and severe liver issues to healthy individuals without liver problems. Ensuring the drug's safety for all participants is also a priority. Suitable candidates will have had stable chronic liver disease for at least three months without recent flare-ups. Participants must stay in the clinic for five days and return for a follow-up visit on the tenth day. 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 treatment.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but it mentions that participants with liver impairment may take other medications if approved by the investigator. It's best to discuss your specific medications with the trial team.

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

Research has shown limited information on the safety of palovarotene for individuals with moderate to severe liver disease. This drug is usually not recommended for these patients because its effects remain insufficiently studied. Some reports suggest that using palovarotene in individuals with liver problems could be risky, potentially worsening their condition.

For those with normal liver function, palovarotene has been used and can cause side effects, which vary among individuals. The current clinical trial aims to learn more about the safety and tolerability of the treatment for those with liver issues. As this is an early-phase study, the primary goal is to understand safety, so participants receive close monitoring. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for liver disease, which often focus on managing symptoms or slowing disease progression, Palovarotene is unique because it targets the underlying mechanisms of liver function directly. This drug is a retinoic acid receptor gamma agonist, which means it works by regulating gene expression involved in cell growth and differentiation, potentially offering a new approach to treating liver impairment. Researchers are excited about Palovarotene because it could provide more targeted therapy for patients with varying degrees of liver impairment, offering hope for better outcomes compared to current options.

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

Research shows that the liver mainly processes palovarotene. It is usually not recommended for individuals with moderate or severe liver problems. This trial will evaluate the pharmacokinetics of palovarotene in various participant groups, including those with moderate and severe hepatic impairment, as well as healthy participants. Studies have found that palovarotene can cause liver-related side effects, prompting close examination of its effects on individuals with liver issues. Early findings suggest that even with liver problems, the body still processes the drug, although the effects might differ. The goal is to understand how different levels of liver function affect the drug's safety and processing. ^{6 7 8 9 10}

Who Is on the Research Team?

Ipsen Medical Director

Principal Investigator

Ipsen

Are You a Good Fit for This Trial?

This trial is for adults with moderate or severe liver impairment, and healthy adults with normal liver function. Participants must be able to stay in the clinical unit until Day 5 and return on Day 10. Specific details about who can join are not provided here.

Inclusion Criteria

Contraceptive use by men or women should be consistent with local regulations regarding methods of contraception for those participating in clinical trials

A stable medication regimen, defined as not starting new therapy(ies) or significantly changing dosage(s) within 30 days prior to dosing

My weight is at least 60 kg, and my BMI is between 18 and 36.

Exclusion Criteria

I don't have conditions that affect how drugs work in my body.

I have had hepatitis B or C for less than 6 months, or I have stable chronic HCV.

I have been diagnosed with a liver or bile duct condition.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive a single dose of palovarotene and are monitored for pharmacokinetics and safety

5 days

In-patient stay until Day 5

Follow-up

Participants are monitored for safety and effectiveness after treatment

5 days

Final visit on Day 10

What Are the Treatments Tested in This Trial?

Interventions

• Palovarotene

Trial Overview The study tests how a single dose of palovarotene (up to 10 mg) is processed by people with different levels of liver health. It's divided into three groups: one for those with normal liver function, another for moderate impairment, and a third for severe impairment if it's safe.

How Is the Trial Designed?

Group I: Group 3 Severe Hepatic impaired participants Experimental Treatment 1 Intervention

Evaluation of the pharmacokinetics of IPN60120 in Severe Hepatic impaired participants (Child-Pugh class C)

Group II: Group 2 Moderate Hepatic impaired participants Experimental Treatment 1 Intervention

Evaluation of the pharmacokinetics of IPN60120 in Moderate Hepatic impaired participants (Child-Pugh class B)

Group III: Group 1 Healthy participants Experimental Treatment 1 Intervention

Evaluation of the pharmacokinetics of IPN60120 in control healthy participants matching the age, sex and weight with hepatic impaired participants

Who Is Running the Clinical Trial?

Ipsen

Lead Sponsor

Trials

358

Recruited

74,600+

David Loew

Ipsen

Chief Executive Officer since 2020

BA in Business Administration and MBA from the University of St. Gallen, Switzerland

Sandra Silvestri

Ipsen

Chief Medical Officer since 2023

MD, PhD

Citations

1. tipranks.com tipranks.com/news/company-announcements/ipsens-new-study-on-palovarotene-implications-for-liver-impaired-patients

Ipsen's New Study on Palovarotene: Implications for Liver- ... The study aims to understand how liver impairment affects the processing of palovarotene, a drug intended for specific medical conditions, ...

2. ipsen.com ipsen.com/websites/ipsen_com_v2/wp-content/uploads/sites/16/2024/12/02164310/PM-Sohonos-EN-20Nov2024.pdf

PRODUCT MONOGRAPH INCLUDING PATIENT ... Palovarotene is not recommended in patients with moderate and severe hepatic impairment. Hepatotoxicity. Retinoids have been associated with ...

3. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2023/215559Orig1s000IntegratedR.pdf

215559Orig1s000 INTEGRATED REVIEW - accessdata.fda.gov The available safety data show that the risks of palovarotene are consistent with the known effects of ... hepatic impairment on the. PK of.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10624720/

The Pharmacokinetic Profile of Palovarotene These results show that palovarotene should be taken after a meal, either as a whole capsule or sprinkled on food.

5. researchgate.net researchgate.net/publication/359776466_Palovarotene_First_Approval

Palovarotene: First Approval Palovarotene is extensively metabolized in the liver. Palovarotene use is not recommended in patients with moderate or severe hepatic failure ...

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2025/215559s002lbl.pdf

sohonos - accessdata.fda.gov 8.7 Hepatic Impairment. The effect of moderate or severe hepatic impairment on the pharmacokinetics of palovarotene has not been evaluated. SOHONOS undergoes ...

7. ipsen.com ipsen.com/websites/Ipsen_Online/wp-content/uploads/sites/61/2024/01/05103949/PM-Sohonos-EN-17Nov2023.pdf

palovarotene capsules There are no clinical data in patients with moderate and severe hepatic impairment. Based on a population PK analysis, there was no evidence ...

8. mayoclinic.org mayoclinic.org/drugs-supplements/palovarotene-oral-route/description/drg-20555967

Palovarotene (oral route) - Side effects & dosage May make these conditions worse. Kidney disease, severe or; Liver disease, moderate or severe—Use is not recommended in patients with these conditions. Back ...

9. drugs.com drugs.com/cons/palovarotene.html

Palovarotene Advanced Patient Information Liver disease, moderate or severe—Use is not recommended in patients with these conditions. Proper use of palovarotene. Take this medicine ...

10. rxlist.com rxlist.com/sohonos-drug.htm

Sohonos (Palovarotene): Side Effects, Uses, Dosage ... Hepatic Impairment. The effect of moderate or severe hepatic impairment on the pharmacokinetics of palovarotene has not been evaluated. SOHONOS undergoes ...