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The OHANA Study is being conducted with both the mother and baby’s well-being in mind.

The primary objective of this study is to evaluate the safety and effectiveness of the investigational drug, volixibat, as a treatment for expectant mothers with Intrahepatic Cholestasis of Pregnancy (ICP).

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You or a loved one may qualify to participate if you:

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Are female and between the ages of 18 and 45

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Have a viable intrauterine pregnancy between 20 to 34 weeks

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Have onset of itch (also known as pruritus) during pregnancy due to a diagnosis of ICP

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Are not already scheduled to deliver your baby within the next 7 days, for any reason

Qualified Participants in The OHANA Study Will:

  • Help advance ICP research! The OHANA Study will give medical professionals the information they need to potentially help you and other expectant mothers suffering from ICP
  • Receive study-related medical care at no cost from an ICP expert

About ICP

Intrahepatic Cholestasis of Pregnancy (ICP)

Intrahepatic Cholestasis of Pregnancy (ICP) is a disease of the liver that occurs in women during pregnancy. The signs and symptoms of ICP typically begin in the second and third trimester.

 

ICP is associated with elevated levels of bile acids in the expectant mother. Research has shown that bile acids can cross the placenta into the baby’s system and can cause adverse outcomes such as spontaneous preterm birth, fetal distress, and in very severe cases, stillbirth.

 

In addition to adverse outcomes for babies, ICP also often causes severe itch in the expectant mother which may lead to a very low quality of life until the itch improves after the birth of the baby.

About the

OHANA Study

The OHANA Study is being conducted with both the expectant mother and baby’s well-being in mind. In contrast to most existing treatments for ICP, including ursodeoxycholic acid (UDCA), volixibat has been designed to stay in the intestine and not enter mother’s bloodstream. This means your baby is unlikely to be exposed to volixibat.

Increased serum bile acids are often associated with severe itching in ICP and similar diseases; in ICP, they can also be associated with adverse outcomes such as preterm birth, fetal distress, and in very severe cases, stillbirth. There are no existing treatments for ICP which lower serum bile acids adequately and none that address the adverse outcomes in the mother or in the baby.

Because of this unmet medical need, we are enrolling pregnant women to learn if volixibat, the investigational drug we are studying in OHANA, is a safe and effective treatment option for ICP. Volixibat has been shown in previous clinical studies to reduce serum bile acid levels in the blood by increasing the excretion of bile acids via feces. By reducing bile acids in the blood, volixibat is anticipated to improve maternal itching as well as reduce the risk of morbidity and mortality in the baby.

Your study team will be here to closely monitor your health over the course of the study. The safety and well-being of you and your baby are our absolute priority. OHANA means “family” in Hawaiian, and we want you to feel supported and informed every step of the way like you are family.

What will happen during this study?

The study consists of a screening period, a treatment period, and a follow-up period. The study will last until four weeks after your baby is born, but the treatment period ends on the day your baby is born.

Screening Period

First, at the screening visit, the study will be explained in detail and you will be able to ask any questions you may have. If you choose to participate, you will review an informed consent form. Once you sign the informed consent form, the study doctor will order some tests to help determine if you will qualify for the study.

If you qualify for the study, you will be scheduled for a baseline visit. At the baseline visit you will have some additional tests done and you will receive your first dose of the study treatment (either volixibat or placebo). You and your baby will be monitored closely, before and after you take the first dose of study treatment in the clinic.

Study Treatment Period

The baseline visit will be followed by a treatment period. This treatment period includes:

  • Standard medical care for your ICP from an experienced medical team, plus oral, twice-daily dosing with the study treatment.
  • Weekly in-person visits and additional check-ins when needed, with the study staff during the study.
  • Once-daily completion of a question in an eDiary which will provide information on your itch.

The treatment period ends when your baby is born.

Follow-Up Period

After the treatment period ends, you will have a telephone appointment two weeks after your baby arrives and a clinic visit four weeks after your baby arrives to check in on your health and the health of your baby.

Am I Eligible?

To participate in this clinical research study, you must meet a set of qualifying criteria. Some are listed here. A study representative will be able to explain more information to see if you or your loved one qualifies.

You or a loved one may qualify if you:

Are female and between the ages of 18 and 45

Have a viable intrauterine pregnancy between 20 to 34 weeks

Have onset of itch (also known as pruritus) during pregnancy due to a diagnosis of ICP

 

Are not scheduled to deliver your baby within the next 7 days, for any reason

 

Additional study requirements and exclusions may apply. A study representative will discuss these with you during the screening period.

Clinical Research

Clinical research studies aim to advance treatment options for patients.

Through clinical research, scientists and doctors are able to determine whether a new medical strategy, drug, or device is safe and effective for people. New treatments are approved only through conducting thorough clinical studies, which rely on potential study participants like you to evaluate their safety and efficacy. Participation in clinical research studies is voluntary. Study participants may cease participation at any time.

What is investigational medication?

Investigational medications are not yet approved by any government health authorities such as the FDA. A clinical research study is used to assess the safety and effectiveness of an investigational medication.

What are the stages of development for a new medication?

Clinical trials are important to help determine if new medications are both safe and effective. Typically, investigational medications must pass through three stages or phases of development in clinical trials: Phase 1, Phase 2, and Phase 3. Results from each phase must be properly assessed before medications can be approved for use by the general population.

Volixibat has already been studied in more than 400 patients in earlier Phase 1 and Phase 2 studies, in patients with conditions other than ICP. The OHANA Study is a Phase 2 study.

Frequently Asked Questions

Why is this clinical research study being conducted?

Increased serum bile acids are often associated with severe itching in ICP and similar diseases; in ICP, they can also be associated with adverse outcomes such as preterm birth, fetal distress, and in very severe cases, stillbirth. There are no existing treatments for ICP which lower serum bile acids adequately and none that address the adverse outcomes for the mother or for the baby.

 

Because of this unmet medical need, we are enrolling pregnant women to learn if volixibat, the investigational drug we are studying in OHANA, is a safe and effective treatment option for ICP.  Volixibat has been shown in previous clinical studies to reduce serum bile acid levels in the blood by increasing the excretion of bile acids via feces.  By reducing bile acids in the blood, volixibat is anticipated to improve maternal itching as well as reduce the risk of morbidity and mortality in the baby.

 

The OHANA Study is being conducted with both the expectant mother and baby’s well-being in mind. In contrast to most existing treatments for ICP, including ursodeoxycholic acid, volixibat has been designed to stay out of the mother’s bloodstream.  This means your baby is unlikely to be exposed to volixibat.

Why is the OHANA Study recruiting pregnant women?

This study is recruiting pregnant women because they are the population affected by ICP. The signs and symptoms of ICP typically begin in the second and third trimester but can also occur earlier in pregnancy.  Your study team will be here to closely monitor your health over the course of the study. The safety and well-being of you and your baby are our absolute priority. OHANA means “family” in Hawaiian, and we want you to feel supported and informed every step of the way like you are family.

Will compensation be provided?

No, unless otherwise specified by your study site. However, you may be reimbursed for study-related expenses, such as travel for study participants and up to two companions. Study drug and assessments are provided at no cost to participants or their families.

Is study participation voluntary?

Yes! While it is important to remain in a clinical research study after enrollment, you have the right to cease your participation at any time for any reason. If you do decide to leave the study, speak with the study doctor first to discuss how leaving the clinical research study may affect your health, along with other treatment options.

Where are the study centers located?

The OHANA Study centers are located throughout the United States. For more information on study center locations, please visit the OHANA Study page on clinicaltrials.gov.

How long is the study and how many study center visits are required?

The study consists of a screening period, a treatment period, and a follow-up period. The study will last until four weeks after your baby is born, but the treatment period ends on the day your baby is born. Weekly in-person visits and additional check-ins via telephone or email with the study staff will be required.

What can I expect on my first visit to the study center?

This initial appointment is an opportunity for you to:

  • Learn more about the OHANA Study. You’ll be speaking with the study team at the study center to learn more about participation in the study and to see if you might be a good candidate.
  • Ask any important questions. These can be any questions you may have about the OHANA Study or clinical research in general.
  • Determine if the OHANA Study may be right for you. If you agree to be considered for the study, the center will ask you to review and sign a consent form. After you speak with the study team and learn details about the study, they will review some additional study criteria to determine if you are eligible to participate in the study.

Who is conducting the OHANA Study?

Mirum Pharmaceuticals, Inc., based in Foster City, California, is a biopharmaceutical company focused on the development and commercialization of therapies to treat debilitating liver diseases. Mirum is developing two apical sodium bile transporter (ASBT) inhibitors for the treatment of cholestatic liver diseases, including maralixibat for pediatric liver diseases, and volixibat for adult liver diseases.

 

In addition to ICP, volixibat is being evaluated in primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC).

 

Maralixibat is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA).

 

Mirum works closely with advocates and the patient communities to understand and address the most critical aspects affecting patients and families living with rare liver diseases.

 

For more information about Mirum, please visit MirumPharma.com.

How can I learn more?

To learn more, please talk to your doctor for more information. You can also visit clinicaltrials.gov using the study ID: NCT04718961.

Study Locations

For more information on study center locations, please visit the OHANA Study page on clinicaltrials.gov.

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