The OHANA Study is being conducted with both the mother and baby’s well-being in mind.
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 medication, volixibat, as a treatment for expectant mothers with Intrahepatic Cholestasis of Pregnancy (ICP).
Are female and between the ages of 18 and 45
Have a viable intrauterine pregnancy of at least 20 weeks
Have onset of itch (also known as pruritus) during pregnancy due to a diagnosis of ICP
Are not already scheduled to deliver your baby within the next 7 days, for any reason
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.
To learn more about ICP, please click here to view an interview with ICP experts, Professor Catherine Williamson (King’s College, London) and Dr. Katherine Kohari (Yale School of Medicine).
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, 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 expectant mothers to learn if volixibat, the investigational medication 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. Volixibat is being studied to assess if it may improve maternal itching as well as reduce the risk of adverse outcomes for the baby by reducing bile acids.
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.
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.
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.
The baseline visit will be followed by a treatment period. This treatment period includes:
The treatment period ends when your baby is born.
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.
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 give you more information to see if you or your loved one qualifies.
Additional study requirements and exclusions apply. A study representative will discuss these with you during the screening period.
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.
Investigational medications are not yet approved by any government health authorities. A clinical research study is used to assess the safety and effectiveness of an investigational 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 development study (Phase 2).
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. Volixibat is being studied to assess if it may improve maternal itching as well as reduce the risk of adverse outcomes for the baby by reducing bile acids.
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, volixibat has been designed to stay out of the mother’s bloodstream. This means your baby is unlikely to be exposed to volixibat.
This study is recruiting expectant mothers 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.
Participants may be eligible for a stipend. You may be reimbursed for study-related expenses like travel. Study treatment and assessments are provided at no cost to participants and their families.
Yes! While it is ideal 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.
For more information on study center locations, please refer to the site location map below or visit the OHANA Study page on clinicaltrials.gov.
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.
This initial appointment is an opportunity for you to:
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.
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.
To learn more, please talk to your doctor for more information. You can also visit clinicaltrials.gov using the study ID: NCT04718961.
GREEN = ACTIVE Location is currently enrolling patients for this study. Study center contact details can be found by visiting clinicaltrials.gov:NCT04718961
RED = PLANNED Location is planned to open but is not currently enrolling patients.
Cookie | Duration | Description |
---|---|---|
cookielawinfo-checkbox-analytics | 11 months | This cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Analytics". |
cookielawinfo-checkbox-functional | 11 months | The cookie is set by GDPR cookie consent to record the user consent for the cookies in the category "Functional". |
cookielawinfo-checkbox-necessary | 11 months | This cookie is set by GDPR Cookie Consent plugin. The cookies is used to store the user consent for the cookies in the category "Necessary". |
cookielawinfo-checkbox-others | 11 months | This cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Other. |
cookielawinfo-checkbox-performance | 11 months | This cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Performance". |
viewed_cookie_policy | 11 months | The cookie is set by the GDPR Cookie Consent plugin and is used to store whether or not user has consented to the use of cookies. It does not store any personal data. |