Studies

Pancreatitis:

• INSPPIRE2 – Pediatric Longitudinal Cohort Study of Chronic Pancreatitis
• WebMAP-CP – A Randomized Trial of a Web-based Non-Pharmacological Pain Intervention for Pediatric Chronic Pancreatitis
• Understanding the Trauma of Pediatric Chronic Illness: A Descriptive Analysis of Pediatric Medical Traumatic Stress in Patients with Cystic Fibrosis and Chronic Pancreatitis

Liver Transplant:

• iMALT – Improving Medication Adherence in Adolescents who had a Liver Transplant
• SPLIT – Studies in Pediatric Liver Transplantation (SPLIT) Clinical Research Network Prospective Observational Study to Assess Outcomes in Pediatric Liver Transplant Recipients
• SOCIAL-Tx – Social & Contextual Impact on children undergoing Liver Transplantation

Inflammatory Bowel Disease:

• DEVELOP: A Multicenter, Prospective, Long-term, Observational Registry of Pediatric Patients with Inflammatory Bowel Disease (IBD)
• Quality Improvement Initiative called Improve Care Now (ICN) in Inflammatory Bowel Disease
• PRODUCE: Personalized Research on Diet in Ulcerative Colitis and Crohn’s Disease; a N-of-1 study of the Specific Carbohydrate Diet in pediatric patients
• SaFR Kids: Relation of Social Factors with Racial Disparities in Pediatric Inflammatory Bowel Disease
• Very Early Onset Inflammatory Bowel Disease (VEOIBD) Biospecimen Repository Project
• Detection of Serum Protein Biomarkers to Predict and Monitor Response to Infliximab using SOMAscan
• MLN0002-2003: A Phase 2, Randomized, Double-Blind, Dose-Ranging Study to Determine the Pharmacokinetics, Safety and Tolerability of Vedolizumab IV in Pediatric (<30kg) Subjects With Ulcerative Colitis or Crohn’s Disease
• Vedolizumab-2005: A Phase 2b, Extension Study to Determine the Long-term Safety of Vedolizumab IV in Pediatric Subjects With Ulcerative Colitis or Crohn's Disease; Long-term Safety With Vedolizumab IV in Pediatric Subjects With Ulcerative Colitis or Crohn's Disease

Crohn’s Disease:

• CAPE: A Long-Term Non-Interventional Registry to Assess Safety and Effectiveness of HUMIRA® (Adalimumab) in Pediatric Patients with Moderately to Severely Active Crohn’s Disease (CD)
• RISK: Risk Stratification and Identification of Immunogenetic and Microbial Markers of Rapid Disease Progression in Children with Crohn’s Disease
• GA29144: A Phase III, Randomized, Double Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Etrolizumab as an Induction and Maintenance Treatment for Adult Patients with Moderately to Severely Active Crohn’s Disease (CD)
• GA29145: An Open-Label Extension And Safety Monitoring Study of Patients With Moderately to Severely Active Crohn's Disease Previously Enrolled in the Etrolizumab Phase III Protocol GA29144
• AMAM: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo- and Active-Controlled, Treat-Through Study to Evaluate the Efficacy and Safety of Mirikizumab in Patients with Moderately to Severely Active Crohn's Disease
• M14-431: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Induction Study of the Efficacy and Safety of Upadacitinib (ABT-494) in Subjects with Moderately to Severely Active Crohn's Disease Who Have Inadequately Responded to or are Intolerant to Biologic Therapy
• M14-433: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Induction Study of the Efficacy and Safety of Upadacitinib (ABT-494) in Subjects with Moderately to Severely Active Crohn's Disease Who Have Inadequately Responded to or are Intolerant to Conventional and/or Biologic Therapies
• M14-430: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Maintenance and Long-Term Extension Study of the Efficacy and Safety of Upadacitinib (ABT-494) in Subjects with Crohn's Disease Who Completed the Studies M14-431 or M14-433
• M16-006: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Induction Study of the Efficacy and Safety of Risankizumab in Subjects with Moderately to Severely Active Crohn's Disease
• M16-000: A Multicenter, Randomized, Double-Blind, Placebo-Controlled 52-Week Maintenance and an Open-Label Extension Study of the Efficacy and Safety of Risankizumab in Subjects with Crohn's Disease Who Responded to Induction Treatment in M16-006 or M15-991
• RCP01-3202: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of Oral Ozanimod as Induction Therapy for Moderately to Severely Active Crohn's Disease
• RCP01-3203: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of Oral Ozanimod as Maintenance Therapy for Moderately to Severely Active Crohn's Disease
• RCP01-3204: A Phase 3, Multicenter, Open-Label Extension Study of Oral Ozanimod for Moderately to Severely Active Crohn's Disease

Ulcerative Colitis:

• GA29102: A Phase 3, Double-Blind, Placebo Controlled, Multicenter Study of the Efficacy and Safety of Etrolizumab During Induction and Maintenance in Adult Patients with Moderate to Severe Active Ulcerative Colitis who are Naïve to TNF Inhibitors sponsored by Genentech
• GA28951: An Open-Label Extension and Safety Monitoring Study of Moderate to Severe Ulcerative Colitis Patients Previously Enrolled in Etrolizumab Phase III Studies
• CNTO148UCO3003: A Phase 3 Randomized, Open-Label Study to Assess the Efficacy, Safety, and Pharmacokinetics of Golimumab Treatment, a Human anti-TNFa Monoclonal Antibody, Administered Subcutaneously in Pediatric Participants with Moderately to Severely Active Ulcerative Colitis
• AMAN: A Phase 3, Multicenter, Randomized, Double-Blind, Parallel, Placebo-Controlled Induction Study of Mirikizumab in Conventional-Failed and Biologic-Failed Adult (18+) Patients with Moderately to Severely Active Ulcerative Colitis
• AMBG: A Phase 3, Multicenter, Randomized, Double-Blind, Parallel-Arm, Placebo-Controlled Maintenance Study of Mirikizumab in Patients with Moderately to Severely Active Ulcerative Colitis
• AMAP: A Phase 3, Multicenter, Open-Label Extension Study to Evaluate the Long-Term Efficacy and Safety of Mirikizumab in Patients with Moderately to Severely Active Ulcerative Colitis
• AMBU: A Multicenter, Open-Label PK Study of Mirikizumab in Pediatric Patients with Moderately to Severely Active Ulcerative Colitis
• A3921210: Open-Label Induction and Double-Blind Randomized Maintenance Study of Oral CP-690,550 (Tofacitinib) in Children with Moderate to Severe Ulcerative Colitis

Eosinophilic Esophagitis:

• R668-EE-1877: A Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Dupilumab in Pediatric Patients with Active Eosinophilic Esophagitis

GERD, Constipation, Pseudo-obstruction:

• CIS-USA-156: Limited Access Protocol for the Use of Oral Cisapride in the Treatment of Pediatric Patients with Refractory Gastroesophageal Reflux Disease and Other Gastrointestinal Motility Disorders

C. difficile Infection

• A Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of a Single Infusion of Bezlotoxumab (MK-6072, Human Monoclonal Antibody to C. difficile Toxin B) in Children Aged 1 to <18 Years Receiving Antibacterial Drug Treatment for C. difficile Infection (MODIFY III)

Cholestatic Liver Disease:

• PROBE: A Prospective Database of Infants with Cholestasis
• LOGIC: Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis
• FORCE: FibroScan in Pediatric Cholestatic Liver Disease
• PALF: Pedatric Acute Liver Failure Network

Hepatitis B:

• HBRN: Hepatitis B Research Network
• A Randomized, Double-Blind Evaluation of the Pharmacokinetics, Safety, and Antiviral Efficacy of Tenofovir Alafenamide (TAF) in Children and Adolescent Subjects with Chronic Hepatitis B Virus Infection

Hepatitis C:

• An Open-Label, Multicenter Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of Ombitasvir (OBV), Paritaprevir (PTV), Ritonavir (RTV) With or Without Dasabuvir (DSV) and With or Without Ribavirin (RBV) in Pediatric Subjects With Genotype 1 or 4 Chronic Hepatitis C Virus (HCV)
• An Open-Label, Multicenter Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of Glecaprevir/Pibrentasvir in Pediatric Subjects with Genotypes 1 – 6 Chronic Hepatitis C Virus (HCV) Infection
• GS-US-334-1113: A Phase 4, a Long Term Follow-up Registry for Adolescent and Pediatric Subjects Who Received a Gilead Hepatitis C Virus Diret Acting Antiviral (DAA) in Gilead- Sponsored Chronic Hepatitis C Infection Trial

PFIC:

• An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2), CLINICAL STUDY PROTOCOL A4250-008
• MRX-502 Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestatis (PFIC) - MARCH-PFIC
• MRX-503 An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC)

Biliary Atresia:

• BASIC: Biliary Atresia Study in Infants and Children
• A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy

Rare Liver Disease:

• A Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated with Cholbam®/Kolbam® (Cholic Acid)
• IMAGINE II: A Multicenter extension study to evaluate the long-term safety and durability of the Therapeutic effect of LUM001, an Apical Sodicum-Dependent Bile Acid Transporter Inhibitor (ABSTI), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects with Alagille Syndrome
• MITOHEP: Longitudinal Study of Mitochondrial Hepatopathies
• AROAAT2001 –A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD)

New Studies

Childhood Obesity and Sugar Sweetened Beverage Consumption in Huslia, Alaska and Old Crow, Canada: A Pilot, Fieldwork Study (supported by the Pacific Rim Research Program). This study compared consumption patterns among children in locations with the highest amounts of sugar sweetened beverages, and assessed attitudes toward fast food consumption as a secondary outcome.

Risk Factors for Infant Overweight in a Latino Cohort (supported by grants from NIH/NIDDK and the Allen Foundation). This study examines the relationship between maternal depression and risk for obesity in Latino children while also evaluating levels of insulin-like growth factor (IGF-1), leptin, ghrelin and insulin at birth and two years of age.

Ongoing Studies

Maternal Depression and Child Weight Gain in a Latino Cohort (supported by grants from HIH/NIDDK, the Hellman Family Foundation and CDHNF/Nestle). This study evaluates prenatal and postnatal depression exposures and other early life environmental exposures in relation to risk for development of childhood overweight and obesity.

The Use of the Resperate Device in Obese Children (supported by a grant from UCSF's Center for Health and Community). This study evaluates the qualitative experience of the use of the Resperate device, a clinically proven device to lower blood pressure in adults, in obese children.

An Evaluation of a Fruit Distribution Program at Balboa High School, San Francisco (In conjunction with the SFUSD Committee on Nutrition and Physical Activity).

Maternal and Adolescent Nutritional Knowledge, Dietary Behaviors and Participation in the WIC Supplemental Food Program (Supported by the UC Davis/ERS RIDGE Small Grant Program in Nutrition).

For more information, please contact
Janet Wojcicki at (415) 476-2380 or via email

New Studies

A Randomized, Double-Blind Evaluation of the Antiviral Efficacy, Safety and Tolerability of Tenofovir Disoproxil Fumarate Vs. Placebo in Pediatric Patients with Chronic HBV Infection – Sponsored by Gilead

A Phase 2, Open-Label, Multicenter, Multi-cohort Study to Investigate the Safety and Efficacy of Ledipasvir/Sofosbuvir Fixed Dose Combination in Adolescents and Children with Chronic HCV Infection (GS1116) - Sponsored by Gilead Sciences, Inc.

A Phase 2, Open-Label, Multicenter, Multi-cohort, Single-Arm Study to Investigate the Safety and Efficacy of Sofosbuvir + Ribavirin in Adolescents and Children with Genotype 2 or 3 Chronic HCV Infection (GS1112) – Sponsored by Gilead Sciences, Inc.

An Open-Label Multicenter Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of Ombitasvir (OBV), Paritaprevir (PTV), Ritonavir (RTV) With or Without Dasabuvir (DSV) and With or Without Ribavirin (RBV) in Pediatric Subjects with Genotype 1 or 4 Chronic Hepatitis C Virus Infection – Sponsored by Abbvie

Ongoing Studies

STOPSC- Studies of Primary Sclerosing Cholangitis – a multi-center database to further the understanding of AIH and PSC in children.

Non-Alcoholic Steatohepatitis Study(NASH) sponsored by the National Institutes of Health (NIH)

Liver Transplantation in HIV+ Children sponsored by the National Institutes of Health (NIH) National Institute Allergy and Infectious Disease

Immunosuppression Withdrawal for Pediatric Living-donor Liver Transplant Recipients with the Department of Health and Human Service

Closed Studies

Interferon +/- Ribavirin for Children with HCV (PEDS-C) sponsored by the National Institutes of Health (NIH) / National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) – CLOSED to enrollment

SPLIT--Studies of Pediatric Liver Transplantation, (is a multi-center, prospective study to collect scientific data in pediatric liver transplantation) sponsored by the NIH- CLOSED to enrollment

Pegylated Interferon +/- Ribavirin for Children with HCV (PEDS-C) sponsored by the National Institutes of Health (NIH) / National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)- CLOSED to enrollment

SPLIT--Studies of Pediatric Liver Transplantation, (is a multi-center, prospective study to collect scientific data in pediatric liver transplantation) sponsored by the NIH- CLOSED to enrollment

FOG 1- Functional Outcomes of Liver Transplant (HRQOL) – ancillary study to SPLIT, sponsored by the NIH- CLOSED to enrollment

FOG 2- Functional Outcomes of Liver Transplant (Cognitive Development & School Performance) ancillary study to SPLIT, sponsored by the NIH- CLOSED to enrollment

TONIC-Treatment of Non-alcoholic liver disease in children - sponsored by the NIH- CLOSED to enrollment

CLIC – Longitudinal Study of Genetic Causes or Intrahepatic Cholestasis, a multi-center study investigating PFIC, BRIC, A1AT, Alagille Syndrome, and BAD, Sponsored by the NIH. -CLOSED to enrollment

CLIC-Mito – Longitudinal Study of mitochondrial Hepatopathies, sponsored by the NIH- CLOSED to enrollment

Evaluation of the Pharmacokinetic, Safety, Tolerability and Efficacy of Entecavir (ETV) in Pediatric Subjects with Chronic Hepatitis B Virus (HBV) Infection who are HBeAG-Positive; sponsored by Bristol-Myers Squibb- CLOSED to enrollment

Pediatric Acute Liver Failure Study (ALF) sponsored by the National Institutes of Health (NIH) CLOSED to enrollment

PROBE - A Prospective Database of Infants with Cholestasis conducted by the Biliary Atresia Research Consortium, sponsored by the National Institute of Diabetes, Digestive, and Kidney Diseases of the National Institutes of Health (NIH)- CLOSED to enrollment

START – A Randomized double-blinded, placebo-controlled trial of corticosteroid therapy following portoenterostomy in infants with biliary atresia (NIH)- CLOSED to enrollment

BASIC – Biliary Atresia Study in Infants and Children (NIH)- CLOSED to enrollment

For more information, please contact:

• Camille Langlois at (415) 476-1756 or via email
• Natasha Feier at (415) 476-1539 or via email

• Optimizing liver and cardiovascular health after pediatric liver transplant. This biobanking project, sponsored by UCSF Transplant Surgery, aims to identify liver biopsy findings and other biomarkers associated with long-term liver and cardiovascular health in people that had liver transplants as children (Enrolling)
• Causes and consequences of post-transplant metabolic syndrome in pediatric liver transplant recipients. This is a prospective study to investigate the contribution of obesity, corticosteroid exposure, and immunosuppression medications to the risk of developing post-transplant metabolic syndrome (PTMS) (Sponsored by NIDDK, enrolling)
• A study of post-traumatic stress symptoms (PTSS) in adolescent liver transplant recipients. This study is being done to better understand if post-traumatic stress symptoms play a role in the way adolescent transplant recipients take their prescribed medicine (Enrolling soon)
• Studies in Pediatric Liver Transplantation (SPLIT). A Multi-center observational study focused on improving a wide variety of outcomes among pediatric patients who have undergone liver transplant (Enrolling)

Please contact Dr. Emily Perito for more information ([email protected])