Clinical trials: Neonatal Cholestasis
Alagille Syndrome
An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol.
- Trial name
- IMAGINE-II (NCT02117713)
- Testing
- LUM001 (Maralixibat)
- Where
- 11 sites in United States, Canada
- Volunteers
- 34 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Alagille Syndrome, Cholestasis
Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease
OBJECTIVES: I. Determine the role of magnesium deficiency in the pathogenesis of decreased serum vitamin D and reduced bone density in children with chronic cholestatic liver disease.
- Conditions
- Alagille Syndrome, Cholestasis, Biliary Atresia
- Trial name
- NCT00007033
- Testing
- magnesium gluconate, magnesium sulfate
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Center for Research Resources (NCRR)
Cholestasis
Low Dose Fat for the Prevention of Liver Disease in Babies With Gastrointestinal Disorders
Neonates with congenital/acquired gastrointestinal disorders are at high risk for Parenteral Nutrition Associated Cholestasis (PNAC).
- Trial name
- NCT01373918
- Testing
- Intralipid
- Where
- 2 sites in United States
- Volunteers
- 41 people
- Sponsor
- University of California, Los Angeles
Intrahepatic, Cholestasis
Reduction of Neonatal Parenteral Nutrition Associated Cholestasis Through Lipid Emulsions
Parenteral nutrition associated cholestasis (PNAC) is a common complication of prolonged and exclusive parenteral nutrition (PN).
- Trial name
- NCT02633384
- Testing
- SMOFlipid, Lipofundin
- Volunteers
- 52 people
- Sponsor
- Centro Hospitalar de Lisboa Central
Cholestasis of Parenteral Nutrition
Comparison of Smoflipid to Soy-based Lipid Reduction for Cholestasis Prevention in Surgical Neonates
Intestinal failure associated liver disease is a cholestatic liver disease associated with prolonged need for parenteral nutrition that can lead to such significant complications as liver failure.
- Trial name
- NCT03387579
- Testing
- Smoflipid 20% Lipid Emulsion for Injection, Intralipid, 20% Intravenous Emulsion
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Indiana University
Cholestasis in Newborn Infant, Biliary Atresia
Dry Blood Spot Screening Test for Neonatal Cholestasis Patients
Cholestatic jaundice with multitude of causes affects approximately 1 in every 2,500 infants. Of the many conditions that cause neonatal cholestasis, the most commonly identifiable are biliary atresia (BA) (25%-35%).
- Trial name
- DBS-SCReNC (NCT03667534)
- Testing
- Dry blood spot screening
- Where
- Taiwan
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Biliary Atresia
A Prospective Database of Infants With Cholestasis
Biliary atresia, idiopathic neonatal hepatitis, and specific genetic cholestatic conditions are the most common causes of jaundice and hyperbilirubinemia that continue beyond the newborn period.
- Trial name
- PROBE (NCT00061828)
- Where
- 16 sites in United States, Canada
- Volunteers
- 1,000 people
- Sponsor
- Arbor Research Collaborative for Health
Intrahepatic Cholestasis of Pregnancy, Respiratory Distress Syndrome, Newborn
Impact of Intrahepatic Cholestasis of Pregnancy on Neonatal Respiratory Outcomes
Intrahepatic cholestasis of pregnancy is a liver condition in late pregnancy, causing itching and high bile acid levels that return to normal after birth.
- Trial name
- CHOLE-RESP (NCT06679972)
- Where
- Romania
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Carol Davila University of Medicine and Pharmacy
Dental Diseases
Dental Dyschromia and Quality of Life in Early Prolonged Hyperbilirubinemia
The main objective of this study is to assess the prevalence of dental discoloration (dental dyschromia) in children who experienced high levels of bilirubin in their blood (hyperbilirubinemia) during their early years.
- Trial name
- SMILE (NCT06506734)
- Testing
- Questionnaires
- Where
- 9 sites in France
- Volunteers
- 120 people
- Sponsor
- University Hospital, Toulouse
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Hepatitis Autoimmune Disease, Primary Biliary Cholangitis (PBC)
Spanish Registry of Autoimmune and Cholestatic Liver Diseases (ColHai)
The purpose of the registry is to know the status of primary biliary colgantis, autoimmune hepatitis, primary sclerosing cholagitis and genetic cholestatic diseases in Spain.
- Conditions
- Hepatitis Autoimmune Disease, Primary Biliary Cholangitis (PBC), Primary Sclerosing Cholangitis (PSC), Cholestatic Genetic Diseases
- Trial name
- ColHai (NCT06767605)
- Where
- Spain
- Volunteers
- 5,000 people
- Sponsor
- Asociación Española para el Estudio del Hígado
Alagille Syndrome
Positional Cloning of the Gene(s) Responsible for Alagille Syndrome
The goal of the project is to identify and clone the gene(s) responsible for the Alagille Syndrome (AGS) by a positional cloning approach.
- Trial name
- NCT00001642
- Where
- United States
- Volunteers
- 225 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Short Bowel Syndrome
Elemental Formula in Neonates Post Small Bowel Resection: Improved Weaning From Total Parenteral Nutrition?
In neonates with recent small bowel resection or congenital bowel anomalies (gastroschisis or omphalocele), does an elemental formula as compared to a partially hydrolyzed formula allowed the infant to wean off Total Parenteral…
- Trial name
- NCT01891279
- Testing
- elemental formula Elecare®, partially hydrolyzed formula
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- The University of Texas Health Science Center, Houston
Biliary Atresia
Outcome of Kasai Portoenterostomy in Biliary Atresia in Upper Egypt
Kasai portoenterostomy is the key standard operation for biliary atresia. The aim of the study is to evaluate the short-term outcome of Kasai portoenterostomy for biliary atresia infants in Upper Egypt.
- Trial name
- NCT05925543
- Testing
- Kasai portoenterostomy operation.
- Where
- Egypt
- Volunteers
- 45 people
- Sponsor
- Aswan University Hospital
Biliary Atresia, Neonatal Cholestasis
Development and Validation of a Screening Score for the Biliary Atresia in Infantile Cholestasis: A Prospective Study
Better survival and prognosis of biliary atresia (BA) depend on early diagnosis and timely Kasai portoenterostomy. Identifying BA from other causes of infantile cholestasis at early stage of the disease still remains a major…
- Conditions
- Biliary Atresia, Neonatal Cholestasis, Infantile Hepatitis Syndrome
- Trial name
- NCT03842150
- Testing
- biliary atresia screen score
- Where
- China
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Essential Fatty Acid Deficiency (EFAD)
Pediatric Study to Evaluate Risk of Developing Essential Fatty Acid Deficiency When Receiving Clinolipid or Standard-of-Care Lipid Emulsion (Part A)
This will be a descriptive study designed to evaluate the propensity for hospitalized pediatric patients treated adequately with Clinolipid or standard of care (Intralipid) from 7 up to 90 days to develop Essential Fatty Acid…
- Trial name
- NCT04555044
- Testing
- Clinolipid, Intralipid
- Where
- 9 sites in United States
- Volunteers
- 101 people
- Sponsor
- Baxter Healthcare Corporation
Essential Fatty Acid Deficiency (EFAD)
Pediatric Study to Evaluate Risk of Developing Essential Fatty Acid Deficiency When Receiving Clinolipid or Standard-of-Care Lipid Emulsion
This will be a descriptive study designed to evaluate the propensity for hospitalized pediatric patients treated adequately with Clinolipid or standard of care for up to 90 days to develop Essential Fatty Acid Deficiency (EFAD).
- Trial name
- NCT02476994
- Testing
- Clinolipid, Intralipid
- Where
- 4 sites in United States
- Volunteers
- 1 people
- Sponsor
- Baxter Healthcare Corporation
Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease
The purpose of the study is to validate the ItchRO instrument (a clinical outcome assessment measure of itching) prior to the analysis of longitudinal treatment effect data being generated in ongoing clinical trials.
- Trial name
- NCT02131623
- Where
- 5 sites in United States, Canada
- Volunteers
- 23 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Primary Biliary Cholangitis, Cardiovascular Abnormalities
Early Identification of Myocardial Impairment in PBC
Primary biliary cholangitis (PBC) is a chronic inflammatory liver disease leading to cirrhosis. Researches reported patients with PBC may involve abnormalities on skeleton, thyroid and exocrine glands.
- Trial name
- EARLY-MYO-PBC (NCT03545672)
- Testing
- CMR examination
- Where
- China
- Volunteers
- 119 people, healthy volunteers welcome
- Sponsor
- RenJi Hospital