Volixibat for Intrahepatic Cholestasis of Pregnancy

(OHANA Trial)

No longer recruiting at 28 trial locations

Overseen By Clinical Trials Mirum

Age: 18 - 65

Sex: Female

Trial Phase: Phase 2

Sponsor: Mirum Pharmaceuticals, Inc.

Disqualifiers: Non-reassuring fetal status, others

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

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a treatment called volixibat for women with Intrahepatic Cholestasis of Pregnancy (ICP), a liver condition during pregnancy that causes high bile acid levels and intense itching. The trial consists of two parts: the first tests two different doses of volixibat to assess safety and tolerability, while the second compares a chosen dose against a placebo (a capsule without the active drug) to evaluate effectiveness. It suits pregnant women diagnosed with ICP who experience significant itching. Participants should not plan to deliver within a week of the trial's start. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Do I need to stop my current medications for this trial?

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

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

Previous studies have tested volixibat for safety in adults with nonalcoholic steatohepatitis (NASH). The research found that volixibat is generally well-tolerated. Some participants experienced side effects, mostly mild, such as stomach-related discomforts like diarrhea. No serious health problems were directly linked to the drug. Since this trial focuses on safety, it suggests that volixibat has shown a reasonable safety profile in earlier research. While some minor side effects might occur, the treatment is not expected to cause significant harm. ^{1 2 3 4 5}

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

Researchers are excited about Volixibat because it offers a fresh approach to treating intrahepatic cholestasis of pregnancy (ICP), a condition typically managed with medications like ursodeoxycholic acid. Unlike these existing treatments, Volixibat works by inhibiting the ileal bile acid transporter, which is believed to help reduce the accumulation of bile acids in the serum. This mechanism targets the root cause of the problem more directly, potentially leading to more effective management of symptoms. Additionally, Volixibat is administered orally, making it a convenient option for patients.

What evidence suggests that volixibat might be an effective treatment for Intrahepatic Cholestasis of Pregnancy?

Studies have shown that volixibat blocks a protein that moves bile acids in the body. This action can lower bile acid levels, which benefits conditions with high bile acids, such as Intrahepatic Cholestasis of Pregnancy (ICP). Research on volixibat in other conditions, like nonalcoholic steatohepatitis, has demonstrated its ability to reduce disease markers effectively without serious side effects. Specifically, patients in previous studies experienced improvements in liver-related symptoms. These findings suggest that volixibat could help manage ICP symptoms by safely lowering bile acid levels. Participants in this trial will receive different dosages of volixibat or a placebo to evaluate its effectiveness and safety. ^{4 6 7 8 9}

Are You a Good Fit for This Trial?

Inclusion Criteria

Provide signed informed consent as described in the protocol and willing to comply with all study visits and requirements.

Diagnosis of ICP.

(Part 2 only) Qualified level of pruritus associated with ICP, during screening.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part 1

Open-label randomized study of volixibat to evaluate safety and tolerability of two doses

Up to 21 weeks

Treatment Part 2

Double-blind, placebo-controlled study to evaluate the safety and efficacy of a selected volixibat dose

Up to 21 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• Volixibat

How Is the Trial Designed?

Group I: Part 2 Arm 1 - Volixibat Selected Dose mg Experimental Treatment 2 Interventions

Participants randomized to this arm will receive volixibat selected dose (mg) twice daily.

Group II: Part 1 Arm 2 - Volixibat 80mg Experimental Treatment 1 Intervention

Participants randomized to this arm will receive volixibat 80mg twice daily.

Group III: Part 1 Arm 1 - Volixibat 20mg Experimental Treatment 1 Intervention

Participants randomized to this arm will receive volixibat 20mg twice daily.

Group IV: Part 2 Arm 2 - Placebo (Placebo Comparator) Placebo Group 1 Intervention

Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.

Who Is Running the Clinical Trial?

Mirum Pharmaceuticals, Inc.

Lead Sponsor

Trials

32

Recruited

102,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT02787304

Study Details | NCT02787304 | Volixibat (SHP626) in the ... The purpose of this study is to determine if the investigational treatment volixibat (SHP626) is safe, tolerable and effective in adults with nonalcoholic ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT02787304?intr=VOLIXIBAT%20POTASSIUM&aggFilters=studyType:int&viewType=Table&rank=1

Volixibat (SHP626) in the Treatment of Adults With Nonalcoholic ... A type of clinical study in which participants are identified as belonging to study groups and are assessed for biomedical or health outcomes. Participants may ...

3. sciencedirect.com sciencedirect.com/science/article/pii/S0168827820301860

24-week interim analysis from a randomized, phase II study The primary endpoint was a ≥2-point reduction in non-alcoholic fatty liver disease activity score without worsening fibrosis at week 48. Results. Volixibat did ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5794849/

Volixibat in Healthy Men: Phase 1 Open-Label Study - PMC Volixibat is a potent inhibitor of the apical sodium-dependent bile acid transporter in development for the treatment of nonalcoholic steatohepatitis.

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5756385/

a randomised placebo-controlled trial This paper reports the results of a phase 1, placebo-controlled, dose-escalation study of the ASBT inhibitor volixibat in healthy adults and ...

6. go.drugbank.com go.drugbank.com/salts/DBSALT002576

Volixibat potassium Volixibat, also known as SHP626 or LUM002, is an investigational drug that will potentially be used for the treatment of Non-Alcoholic Steatohepatitis (NASH).

7. precision.fda.gov precision.fda.gov/ginas/app/ui/substances/7d9b2e25-2e0f-4af1-baee-e8b6d1048761

VOLIXIBAT POTASSIUM VOLIXIBAT POTASSIUM · overview · Substance Hierarchy · Chemical Structure · Chemical Moieties · Names and Synonyms · Codes - Classifications · Codes - Identifiers.

8. orpha.net orpha.net/en/drug/substance/689608?name=

Volixibat The documents contained in this website are presented for information purposes only. The material is in no way intended to replace professional medical care by ...

9. probechem.com probechem.com/products_Volixibatpotassium.html

Volixibat potassium (SHP-626;SAR-548304;LUM-002) Catalog No.: PC-61419Not For Human Use, Lab Use Only. A highly potent and selective inhibitor of the apical sodium-dependent bile acid transporter (ASBT) in ...