The BOLD research study is the first pivotal Phase 3 study to evaluate the safety and efficacy of odevixibat in babies with biliary atresia who have undergone a Kasai hepatoportoenterostomy (HPE).
A clinical trial is a scientific study where researchers learn if and how a medication works. The BOLD study has been initiated by Albireo Pharma, a clinical-stage biopharmaceutical company focused on rare pediatric liver diseases and disorders.
What is biliary atresia?
Biliary atresia is a rare pediatric liver disease where damaged or absent bile ducts outside the liver result in bile and bile acids being trapped inside the liver. This may result in cirrhosis, and even liver failure. Symptoms typically develop two to six weeks after birth.
There are no approved medications for biliary atresia.
What is the BOLD study?
The BOLD (Biliary Atresia and the Use of Odevixibat in Treating Liver Disease) study is the first pivotal Phase 3 clinical trial for infants with biliary atresia. During a two-year period, the double-blind, randomized study will investigate how well and safely the investigational* drug odevixibat works. The trial will evaluate if a child with biliary atresia who has undergone a Kasai HPE can delay or avoid liver transplant by taking odevixibat.
The results from the trial will be shared with regulatory authorities that assess if odevixibat can be approved to treat biliary atresia.
*The term "investigational" means that odevixibat is not approved for use in biliary atresia or any other conditions anywhere in the world.
What is odevixibat?
The investigational medicine of the BOLD study, odevixibat, is a potent, non-systemic ileal bile acid transport inhibitor (IBATi) designed to treat rare pediatric cholestatic liver diseases such as biliary atresia. Odevixibat is a once-daily oral medication which may be emptied into breastmilk, formula, or certain soft foods.
Babies with a clinical diagnosis of biliary atresia, aged 90 days or less at the time of Kasai HPE, and who are able to start the study treatment within 3 weeks of their Kasai, may be eligible to participate.
There is a very small window of time where babies may be eligible, so it is important to talk to your baby's doctor right away.
More information may be found here: https://clinicaltrials.gov/ct2/show/NCT04336722.
Read the research study flyer
If you are a parent or guardian who would like to learn more about your child’s participation in the BOLD clinical trial, discuss it with your baby’s doctor. We’ve prepared a brochure that has information about the trial that you can give to your doctor.
For more information
Ask your baby's doctor to call or email Albireo to learn
about the BOLD Study.
Phone: +1 (857) 378-2035
Email: [email protected]
In order to be included in the clinical trial, the patient must be:
- A male or female with a clinical diagnosis of biliary atresia
- Aged 90 days or less at time of Kasai HPE
- Eligible to start study treatment within 3 weeks post-Kasai HPE
Patients may be excluded from the trial if they:
- Have intractable ascites
- Had Ileal resection surgery
- Have an ALT ≥10× upper limit of normal (ULN) at screening
- Are on total parenteral nutrition at randomization
- Have acute ascending cholangitis (patients may be randomized after resolution of acute ascending cholangitis)
- Have choledochal cystic disease
Note: Your doctor will be able to determine if your child is able to participate in the BOLD study.
United States, California
Stanford Children's Health
Palo Alto, California, United States, 94304
Contact: Amrita Narang, MD
UCSF Benioff Children's Hospital San Francisco
San Francisco, California, United States, 94158
Contact: Phillip Rosenthal, MD
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Contact: Cara Mack, MD
United States, Delaware
Nemours/Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States, 19803
Contact: Adebowale Adeyemi, MD
United States, Florida
University of Miami
Miami, Florida, United States, 33146
Contact: Tamir Miloh, MD
United States, Georgia
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, United States, 30322
Contact: Saul Karpen, MD, PhD
United States, Indiana
Indiana University school of Medicine
Indianapolis, Indiana, United States, 46202
Contact: Jean Molleston, MD
United States, Ohio
Cleveland, Ohio, United States, 44195
Contact: Vera Hupertz, MD
United States, Maryland
Johns Hopkins Children's Center
Baltimore, Maryland, United States, 21287
Contact: Wikrom Karnsakul, MD
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Contact: Andrew Wehrman, MD
United States, Missouri
Children's Mercy Hospital
Kansas City, Missouri, United States, 64108
Contact: Ryan Fischer, MD
United States, New York
The Children's Hospital at Montefiore
Bronx, New York, United States, 10467
Contact: Nadia Ovchinsky, MD
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029
Contact: Jaime Chu, MD
NewYork-Presbyterian Morgan Stanley Children's Hospital
New York, New York, United States, 10032
Contact: Jennifer Vittorio, MD
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
Contact: Henry Lin, MD
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Contat: Kathleen Loomes, MD
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Contact: Patrick McKiernan, MD
United States, Texas
UT Southwestern Medical Center
Dallas, Texas, United States, 75390
Contact: Amal Aqul, MD
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Contact: Simon Horslen, MB ChB
United States, Wisconsin
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Contact: Bernadette Vitola, MD
The Children's Hospital at Westmead
Contact: Michael Stormon, MD
Royal Children's Hospital
Contact: Winita Hardikar, PhD
Women's Children's Hospital
North Adelaide, Australia
Contact: Richard Couper, MD
Cliniques universitaires Saint-Luc
Contact: Etienne Sokal, MD, PhD
Contact: Ruth De Bruyne, MD, PhD
Contact: Fernando Alvarez, MD
The Hospital for Sick Children
Contact: Vicky Ng, MD
Le Kremlin-Bicêtre, France
Contact: Emmanuel Gonzales, MD, PhD
Hôpital Femme Mère Enfant
Contact: Mathias Ruiz, MD
Jeanne de Flandre Hospital
Contact: Madeleine Aumar, MD
Necker University Hospital - Enfants malades
Contact: Florence Lacaille, MD
Hannover Medical School
Contact: Ulrich Baumann, MD
University Children´s Hospital Tuebingen
Contact: Ekkehard Sturm, MD, PhD
Semmelweis Egyetem I.sz Gyermekgyógyászati Klinika
Contact: Antal Dezsöfi, MD, PhD
Shaare Zedek Medical Center
Contact: Eyal Shteyer, MD
Schneider Children´s Medical Center of Israel
Petah tikva, Israel
Contact: Orith Waisbourd-Zinman, MD
ASST Papa Giovanni XXIII
Contact: Lorenzo D'Antiga, MD
Meyer Children´s University Hospital
Contact: Giuseppe Indolfi, MD, PhD
Regina Margherita Children's Hospital
Contact: Pier Luigi Calvo, MD
University Hospital of Padova
Contact: Mara Cananzi, MD, PhD
Korea, Republic of
Samsung Medical Center
Seoul, Korea, Republic of
Contact: Yon Ho Choe, MD
Seoul National University Children's Hospital
Seoul, Korea, Republic of
Contact: Hyun-Young Kim, PhD
Hospital Raja Perempuan Zainab II
Kota Bharu, Malaysia
Contact: Wan Mohd Ruzaimie Wan Mohamad Noor, MD
University of Malaya Medical Centre
Kuala Lumpur, Malaysia
Contact: Way Seah Lee, MD
University Medical Center Groningen
Contact: Hendrik Jan Verkade, MD, PhD
Starship Child Health
Auckland, New Zealand
Contact: Helen Evans, MD
Instytut Pomnik-Centrum Zdrowia Dziecka
Contact: Piotr Czubkowski, MD, PhD
Hospital Universitari Vall d'Hebron
Contact: Jesús Quintero, MD, PhD
National Taiwan University Hospital
Contact: Huey-Ling Chen, PhD, MD
Taipei Veterans General Hospital
Contact: Ching-Feng Huang, MD, PhD
Hacettepe University Ihsan Dogramaci Childrens Hospital
Contact: Hasan Özen, MD
Akdeniz University Medical Faculty
Contact: Reha Artan, MD
Istanbul University, Istanbul Medical Faculty
Contact: Ozlem Durmaz, MD
Biliary atresia is a rare pediatric liver disease that occurs in an estimated one out of every 15,000-20,000 live births in the United States and European Union. Though rare, it is the most common pediatric cholestatic liver disease and the leading cause of pediatric liver transplant across all diseases.
Symptoms typically develop about two to six weeks after birth due to damaged or missing bile ducts. With no pathway out of the liver, bile builds up and starts to destroy liver cells, which leads to scarring of the liver, called cirrhosis, and possibly liver failure.
There are currently no approved medications for biliary atresia. A Kasai HPE procedure can help reestablish bile flow from the liver to the gallbladder.
BOLD is a double-blind, randomized, placebo-controlled study.
- Double-blind: trial participants, their families, and clinicians involved in the research study do not know if a participant is taking the investigation medication or a placebo.
- Placebo: looks like the study drug but does not contain any of the medication. Researchers compare results from patients taking a placebo with those taking the study drug to see if the study drug works better.
- Randomized: investigators randomly assign half the trial participants to the experimental group, which receives the drug, and the other half to the control group, which receives the placebo.
A clinical trial is usually described by the phase it is in. Each phase usually has a different purpose, a larger group of participants, and a longer study period.
- Phase 1: Basic safety. Tests an experimental treatment on a small group of often healthy people to understand its safety and potential side effects, as well as to help find the correct dosage.
- Phase 2: Effectiveness. Focuses on whether the medicine works on a larger group of people who have a specific disease or condition. Researchers continue to study safety, including short-term side effects. This phase can last several years.
- Phase 3: Wider safety and effectiveness. Gathers more information about safety and effectiveness by studying much larger and more varied groups of people, often with different medication doses. A treatment may also be used in combination with other medications.
- Upon completion of Phase 3, a company can submit the investigational medicine for review by a regulatory body such as the Food & Drug Administration (FDA) in the United States. Once that body reviews the data and agrees that the Phase 3 trial had positive results, it may approve the investigational medicine for use by patients within the territory it governs.
- Phase 4: Long-term effects. After the FDA approves a medication for use, this phase examines a product in the largest set of patients to gauge long-term effectiveness of the treatment or intervention.
Albireo Pharma, Inc. is conducting the BOLD clinical trial. Learn more about Albireo’s commitment to families at www.albireopharma.com.
Phone: +1 (857) 378-2035
Email: [email protected]