Clinical Studies

This section provides information about ChiLDReN studies. A brief description of each study is provided, with a link to the clinicaltrials.gov website that offers detailed information that may help you consider study participation.

The ChiLDReN Network has several active studies focused on children that are diagnosed with cholestasis:

Most of the ChiLDReN studies are natural history studies aimed at acquiring information and data that will provide a better understanding of these rare conditions. Participants will be asked to allow study personnel to obtain information from medical records and an interview, and to collect blood, urine, and tissue samples when clinically indicated, in order to understand the causes of these diseases and to improve the diagnosis and treatment of children with these diseases. All of the information obtained in these studies is confidential and no names or identifying information are used in the study.

Natural History Studies

GENETIC COLLECTION: This study provides a means to collect a genetic biosample from participants previously enrolled into clinical research under the ChiLDReN-supported protocols of PROBE, BASIC, LOGIC, and MITOHEP. Genetic biosamples from the participants’ biological parents may be collected as well.
Eligibility: (Children) Previous enrollment in, but exited from, one of PROBE, BASIC, LOGIC, or MITOHEP; consented for DNA sample during enrollment in one of these studies but not previously collected; still followed at the clinical site. (Parents) Biological parent of previously consented child participant without collection of parental DNA biospecimen; child still followed at the site. Deceased participants, participants who cannot be contacted, and participants who exited prior study due to violating eligibility criteria are excluded from this study.
ClinicalTrials.gov Study NCT05272319
Current Enrollment:

PSC: Prospective Observational Study of Primary Sclerosing Cholangitis (PSC) in Children.
Eligibility: Age 2 – 25 at time of screening, diagnosis of either Large Duct PSC or Small Duct Only PSC, and absence of conditions predisposing to secondary sclerosing cholangitis, other liver disease, or pregnancy at time of enrollment. For the MRI portion of the study, aged 8 – 25 with no contraindication to MRI or skin condition that could be aggravated by magnetic resonance elastography.
ClinicalTrials.gov Study NCT04181138
Current Enrollment:

PROBE: A prospective study of infants and children with cholestasis.
Eligibility: Infants up to 6 months of age that have been diagnosed with cholestasis (direct hyperbilirubinemia).
ClinicalTrials.gov Study NCT00061828
Current Enrollment: 1475
 

BASIC: A prospective database study of older children with biliary atresia.
Eligibility: Children and adults age 6 months and older that have been diagnosed with biliary atresia, both before and after liver transplantation.
ClinicalTrials.gov Study NCT00345553
Current Enrollment: 1743
 

LOGIC: A longitudinal study of genetic causes of intrahepatic cholestasis.
Eligibility: Children and adults ages 6 months through 25 years diagnosed with Alagille Syndrome, alpha-1 antitrypsin deficiency, progressive familial intrahepatic cholestasis, or bile acid synthesis defects, both before and after liver transplantation.
ClinicalTrials.gov Study NCT00571272
Current Enrollment: 1410
 

MITOHEP: A longitudinal study of mitochondrial hepatopathies.
Eligibility: Children and adults through age 18 years that have been diagnosed with (or are strongly suspected to have) a mitochondrial liver disease.
ClinicalTrials.gov Study NCT01148550
Current Enrollment: 91
 

FORCE:A cross-sectional and longitudinal assessment of the utility of liver stiffness measurement (as assessed by elastography) in children with chronic cholestatic liver disease.
Eligibility: Children currently enrolled in BASIC, PROBE, or LOGIC with a diagnosis of biliary atresia, alpha-1 antitrypsin deficiency or Alagille syndrome.
ClinicalTrials.gov Study NCT02922751
Current Enrollment: 552
 

PUSH: A longitudinal study of the risk of hepatic cirrhosis in Cystic Fibrosis.
Eligibility: Children ages 3 through 12 years of age with Cystic Fibrosis and pancreatic insufficiency who are enrolled in the CFF or Toronto CF registry studies. This study is closed to enrollment.
ClinicalTrials.gov Study NCT01144507
 

MRE: Study to assess the feasibility of using MRI based liver stiffness determination in children with CF.
Eligibility: Children with Cystic Fibrosis and pancreatic insufficiency who are enrolled in the PUSH Study at one of the participating clinical centers.
ClinicalTrials.gov Study NCT02979340
Current Enrollment: 114
 

ELASTIC: A study to determine if liver stiffness as measured by transient elastography (TE), when combined with ultrasound (US) pattern characterization can improve the identification of children with cystic fibrosis and liver disease.
Eligibility: Children with Cystic Fibrosis and pancreatic insufficiency who are enrolled in the PUSH Study at one of the participating clinical centers.
ClinicalTrials.gov Study NCT03001388
Current Enrollment: 141
 

Clinical Therapy Trials

START: A clinical trial to test the efficacy and safety of corticosteroids in the treatment of biliary atresia following hepatic portoenterostomy at a ChiLDREN study site.
Eligibility: Infants up to 6 months of age that have been diagnosed with biliary atresia and have undergone hepatic portoenterostomy within 72 hours at a ChiLDREN study site. This study is closed to enrollment.
ClinicalTrials.gov Study NCT00294684
 

ITCH: A clinical trial to test the efficacy and safety of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the treatment of Pruritus in Alagille Syndrome Patients.
Eligibility: Children and adults between the ages of 12 months and 18 years of age that have been diagnosed with Alagille Syndrome and Pruritus. This study is closed to enrollment.
ClinicalTrials.gov Study NCT02057692
 

PRIME: This is a multi-center open label phase I/IIa clinical trial of high dose IVIG in infants with biliary atresia to determine if the administration of intravenous immunoglobulin (IVIG) in these infants is feasible, well tolerated and safe, to determine if there is a trend towards improved clinical outcomes, and to examine mechanisms that might explain the effects of IVIG in this disease.
Eligibility: Infants up to 4 months of age that have been diagnosed with biliary atresia and have undergone hepatic portoenterostomy within 72 hours at a participating ChiLDREN study site. This study is closed to enrollment.
ClinicalTrials.gov Study NCT01854827
 

IMAGINE II: An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (IMAGINE II).
Eligibility: Children and adults between the ages of 12 months and 18 years of age that have been diagnosed with Alagille Syndrome and Pruritus and completed the ITCH treatment protocol of LUM001. This study is closed to enrollment.
ClinicalTrials.gov Study NCT02117713