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Citation
Magee, John (2024). A Phase I/IIa Trial of Intravenous Immunoglobulin (IVIG) Therapy Following Portoenterostomy in Infants with Biliary Atresia (PRIME) (Version 1) [Dataset] NIDDK Central Repository. https://doi.org/10.58020/g2nf-m919
Data Availability Statement
Data from the A Phase I/IIa Trial of Intravenous Immunoglobulin (IVIG) Therapy Following Portoenterostomy in Infants with Biliary Atresia (PRIME) [(Version 1) https://doi.org/10.58020/g2nf-m919] reported here are available for request at the NIDDK Central Repository (NIDDK-CR) website, Resources for Research (R4R), https://repository.niddk.nih.gov/.
Acknowledgment Statement
The PRIME study was conducted by the study investigators and supported by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). The resources from the PRIME study reported here were supplied by NIDDK Central Repository (NIDDK-CR) and are available for request at https://repository.niddk.nih.gov. This manuscript was not prepared under the auspices of the PRIME study and does not necessarily reflect the opinions or views of the PRIME study, NIDDK-CR, or NIDDK.
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General Description

The PRIME study was a multicenter prospective phase I/IIA open label trial, aimed at assessing the feasibility, tolerability, and safety of intravenous immunoglobulin (IVIG) therapy following hepatic portoenterostomy (HPE) in infants with biliary atresia (BA). After enrollment, participants received three intravenous doses of IVIG at designated intervals over the first 60 days following HPE and were followed for 360 days. All infants in this trial were also treated with standardized doses of other routine standard-of-care treatments for BA and this routine clinical care was not modified by participation in this study.

Objectives

The primary objectives the PRIME study were to determine the feasibility, acceptability, tolerability, and safety profile of IVIG treatment administered to infants after hepatic portoenterostomy (HPE) for biliary atresia, and investigate preliminary evidence of activity and explore mechanisms of action.

Outcome Measure

The primary outcome measures were feasibility and acceptability of IVIG treatment, and any serious adverse events.

Eligibility Criteria

Inclusion criteria:

  • Infant under 120 days old with established diagnosis of BA
  • Start treatment with 3-5 days of the standard HPE operation performed for BA
  • Post-conception age ≥ 36 weeks at time of enrollment
  • Weight at enrolment ≥ 2000 grams
  • Written informed consent to participate in the study obtained within 3 days of completion of HPE
Exclusion criteria:
  • Laparoscopic HPE or "gall bladder Kasai" (cholecysto-portostomy) surgery was performed
  • Biliary atresia splenic malformation syndrome (presence of asplenia, polysplenia or double spleen)
  • History of a hypercoagulable disorder
  • Renal disease defined as serum creatinine > 1.0 mg/dL prior to enrollment or presence of complex renal anomalies found on imaging
  • Evidence of congestive heart failure or fluid overload
  • Presence of significant systemic hypertension for age (defined as persistent systolic blood pressure ≥ 112 mmHg measured on at least 3 occasions following HPE)
  • Infants whose mother is known to have human immunodeficiency virus infection, serum HBsAg, or hepatitis C virus antibody positive
  • Previous treatment with intravenous immunoglobulin therapy, corticosteroid therapy, or any other investigational agent
  • History of allergic reaction to any human blood product infusion
  • Infants with other severe concurrent illnesses, such as neurological, cardiovascular, pulmonary, metabolic, endocrine, and renal disorders, that would interfere with the conduct and results of the study
  • Any other clinical condition that is a contraindication to the use of IVIG

Detailed inclusion and exclusion criteria can be found in the study protocol.

Outcome

Administration of IVIg infusions was feasible and acceptable in 79%. None of the serious adverse events (SAEs) were directly related to IVIg infusions; however, 90% of participants had a SAE. Compared with a historical placebo arm group, there was no significant increase in the proportion of IVIg participants with a serum total bilirubin < 1.5 mg/dL at 90, 180, or 360 days post-HPE. Survival with the native liver in the IVIg participants showed no significant benefit over the historical placebo arm, with a difference at 360 days of -11.9% (IVIg: 58.6%, placebo: 70.5%; 90% UCB: 2.1%; p > 0.05).

Research Area

Digestive Diseases

Study Type

Interventional

Study Start Date

2013-10

Study End Date

2016-07

Condition

Bile Duct Disorder, Biliary Atresia

Keywords

Clinical Trial, Hepatic Portoenterostomy (HPE), Billary Atresia, Intravenous Immunoglobulin (IVIG)

NIDDK Division

DDN

30
Participants

Target Population
Children
Age statistics is not available for this study
Location statistics is not available for this study

Public Documents Table
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Description
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File Format
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Datasets (27)
Datasets Table
Dataset Name
Description
# of Records
# of Variables
File Format(s)
Start Final DD Dataset
Contains data including age, BMI, laboratory values, procedures, etc., from baseline to the end of follow-upcsv (105.41 KB); sas7bdat (480 KB)
Adverse Event Log 2 Dataset
Captures detailed adverse event data, including event start date and status, severity, outcome, treatment, whether the event was during IVIG infusion, relationship to treatment, etc.csv (91.92 KB); sas7bdat (3.5 MB)
Final Status Dataset
Captures information on final statuscsv (3.26 KB); sas7bdat (744 KB)
Infusion 2 Dataset
Captures infusion data, including adverse events, dose administered, time and date of dosage start/stop, caretaker request, reasons why full dose was not administered, etc.csv (7.38 KB); sas7bdat (756 KB)
Laboratory Adverse Event Dataset
Captures laboratory test results including adverse eventscsv (59.1 KB); sas7bdat (4.75 MB)
Transplant Dataset
Captures transplant date, including growth failure, complications, cadaveric type, donor type, age, gender, weight, and blood type, etc.csv (2.98 KB); sas7bdat (512 KB)
IV Access Log Dataset
Captures data from IV Access Log, including access location, attempts, and success, venous access type, date removed, reason unsuccessful, etc.csv (14.93 KB); sas7bdat (1 MB)
Adverse Event Log 1 Dataset
Captures adverse event data, including event start date and statuscsv (1.79 KB); sas7bdat (408 KB)
Research Labs - Plasma Specimens Dataset
Contains plasma lab resultscsv (13.57 KB); sas7bdat (1.7 MB)
Diet Dataset
Captures dietary data, including food and nutrient intake and feeding methodcsv (28.13 KB); sas7bdat (1.5 MB)
Additional Variables Dataset
Additional variables including subgroups according to the patterns of hepatic radicles at the porta hepatis, subtypes according to the patterns of distal ducts, main types, exam date, length/height (cm), date height/length taken, randid, date of surgery, date weight taken, weight (kg) csv (32.55 KB); sas7bdat (384 KB)
Patient Dataset
Captures patient information including demographic datacsv (141.95 KB); sas7bdat (1.11 MB)
Research Labs - Whole Blood Dataset
Contains whole blood lab resultssas7bdat (1 MB); csv (9.09 KB)
Infusion Dataset
Captures infusion data, including adverse events, dose administered, time and date of dosage start/stop, caretaker request, reasons why full dose was not administered, etc.csv (10.3 KB); sas7bdat (756 KB)
Eligibility Dataset
Captures information on study inclusion and exclusion criteria and subject eligibility statuscsv (5.52 KB); sas7bdat (512 KB)
Laboratory Dataset
Captures laboratory results datacsv (153.38 KB); sas7bdat (832 KB)
Start Final DD 2 Dataset
Contains data including age, BMI, laboratory values, procedures, etc., from baseline to the end of follow-upcsv (92.79 KB); sas7bdat (384 KB)
DD P007 Dataset
Captures information on kasai date (days from consent date), month end date (days from icdt), and quarter date sas7bdat (128 KB); csv (598 B)
008 Laboratory Dataset
Contains laboratory results datacsv (75.22 KB); sas7bdat (8.38 MB)
Protocol Deviation Log Dataset
Captures information related to any protocol deviationscsv (1.79 KB); sas7bdat (408 KB)
Derived Dataset
Contains derived data used in primary publication analysiscsv (12.95 KB); sas7bdat (240 KB)
Physical Exam Dataset
Captures data from physical exam, such as vital signs and symptomscsv (52.51 KB); sas7bdat (1.97 MB)
PROBE Link Dataset
Captures linking data to the PROBE study, including case report form status and version, date of birth, event and trasnfer status data, etc.csv (1.95 KB); sas7bdat (512 KB)
Listing 2 Dataset
Contains ID datasas7bdat (200 KB); csv (664 B)
IV Dataset
Captures information related to IV access, including event status and access requiredcsv (4 KB); sas7bdat (612 KB)
DD Adverse Events Dataset
Captures information related to adverse eventscsv (22.86 KB); sas7bdat (1 MB)
Dosing Derived Dataset
Captures derived data related to dosing, such as IVIG dose, dosing weight, and compliancecsv (15.1 KB); sas7bdat (1008 KB)
Specimens (0)
There are currently no specimens available