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Research

We participate in a number of multi-center studies and clinical trials. We also are enrolling subjects for some of our clinical trials.

PROBE: PROspective study of Biliary atresia Epidemiology: A Prospective Database of Infants with Cholestasis
A protocol of the Childhood Liver Disease Research and Education Network (ChiLDREN) 

  • Principal Investigator: Dr. Kathleen B. Schwarz 
  • Contact Kim Pfeifer, RN 410.614.8583 kfehily2@jhmi.edu 
  • Description: This research study is being conducted for two reasons: The first is to collect medical information about children born with cholestatic liver disease and store it in a national database. The second is to develop a repository (long-term storage) of samples of blood, urine, and tissue from children born with cholestatic liver disease.

BASIC: Biliary Atresia Study in Infants, Children and Adults
A protocol of the Childhood Liver Disease Research and Education Network (ChiLDREN) 

  • Principal Investigator: Dr. Kathleen B. Schwarz 
  • Contact Kim Pfeifer, RN 410.614.8583 kfehily2@jhmi.edu 
  • Description: This research study is being conducted to learn more about the natural history and causes of Biliary Atresia in children. This study is also being done to develop a repository (long-term storage) of samples of blood and urine from older children with Biliary Atresia to be used in research about liver disease.

LOGIC: Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis
A protocol of the Childhood Liver Disease Research and Education Network (ChiLDREN)

  • Principal Investigator: Dr. Kathleen B. Schwarz
  • Contact: Kim Pfeifer, RN 410.614.8583 kfehily2@jhmi.edu 
  • Description: This research study collects medical information, blood, urine, and tissue samples from children and young adults with the following liver diseases: Alagille syndrome, Alpha-1-Antitrypsin deficiency, Progressive familial intrahepatic cholestasis (PFIC), Bile acid synthesis defects. The information and samples will be part of a database. This information will help the researchers to learn more about these important diseases.

MITOHEP: Longitudinal Study of Mitochondrial Hepatopathies: A protocol of the Childhood Liver Disease Research and Education Network (ChiLDREN) 

  • Principal Investigator: Dr. Kathleen B. Schwarz 
  • Contact: Kim Pfeifer, RN 410.614.8583 kfehily2@jhmi.edu 
  • Description: The specific aims of this study are (1) to determine the clinical phenotypes and natural history of hepatic respiratory chain and fatty acid oxidation disorders, (2) to determine the correlation between genotype and phenotype, (3) to determine if circulating biomarkers reflect diagnosis and predict liver disease progression and survival with the native liver, (4) to determine the clinical outcome of these disorders following liver transplantation, and (5) to develop a repository of serum, plasma, urine, tissue and DNA specimens that will be used in ancillary studies.

Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH): A protocol of the Childhood Liver Disease Research and Education Network (ChiLDREN) 

  • Principal Investigator: Dr. Shruti Paranjape 
  • Contact: Kim Pfeifer, RN 410.614.8583 kfehily2@jhmi.edu 
  • Description: The specific aims of this study are: (1) To determine if sonographic findings predict the risk of progression of liver disease to cirrhosis by comparing cystic fibrosis subjects with heterogeneous echogenicity pattern on ultrasound to those with normal echogenicity pattern on ultrasound (2) To develop a database and biorepository of serum, plasma, urine and DNA to aid the investigations in ascertaining the mechanisms, consequences, genetic risk factors and biomarkers for the development of cirrhosis (3) To determine if there are differences in health related quality of life, pulmonary or nutritional status in children with cystic fibrosis who have a heterogeneous echo pattern on ultrasound compared to those who have a normal echo pattern on ultrasound (4) To determine if Doppler velocity measurements of hepatic and splenic vessels predict an increased risk for the development of cirrhosis. (5) To determine if cirrhosis on ultrasound progresses to portal hypertension during the study period (6) To determine if homogeneous liver progresses to either cirrhosis or heterogeneous liver.

Hepatitis B Research Network Pediatric Cohort Study (HBRN) 

  • Principal Investigator: Dr. Kathleen B. Schwarz
  • Contact: Kim Pfeifer, RN 410.614.8583 kfehily2@jhmi.edu 
  • Description: The purpose of this study is to describe participants 6 months to <18 years of age with hepatitis B virus (HBV) infection in a prospective cohort in the United States (US) and Canada and identify predictors of disease activation and progression.

Entecavir/Pegylated Interferon in Immune Tolerant Children With Chronic Hepatitis B Virus (HBV) Infection: A protocol of the Hepatitis B Research Network (HBRN)

  • Principal Investigator: Dr. Kathleen B. Schwarz 
  • Contact: Kim Pfeifer, RN 410.614.8583 kfehily2@jhmi.edu
  • Description: The purpose of this study is to determine the effectiveness of treatment using a combination of drugs (entecavir and pegylated interferon) vs. no treatment in children ages 3-<18 years old with immunotolerant chronic hepatitis B.

A Phase III Study of the Safety and Efficacy of Entecavir in Pediatric Patients with Chronic HBV-Infection 

  • Principal Investigator: Dr. Kathleen B. Schwarz 
  • Contact: Kim Pfeifer, RN 410.614.8583 kfehily2@jhmi.edu 
  • Description: The purpose of this study is to determine the effectiveness of treatment using a entecavir to treat children ages 2-17 years with immune active chronic hepatitis B.