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.
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.
The primary outcome measures were feasibility and acceptability of IVIG treatment, and any serious adverse events.
Inclusion criteria:
Detailed inclusion and exclusion criteria can be found in the study protocol.
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).
Digestive Diseases
Interventional
2013-10
2016-07
Bile Duct Disorder, Biliary Atresia
Clinical Trial, Hepatic Portoenterostomy (HPE), Billary Atresia, Intravenous Immunoglobulin (IVIG)
DDN
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-up | csv (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 status | csv (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 events | csv (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 status | csv (1.79 KB); sas7bdat (408 KB) | ||
Research Labs - Plasma Specimens Dataset | Contains plasma lab results | csv (13.57 KB); sas7bdat (1.7 MB) | ||
Diet Dataset | Captures dietary data, including food and nutrient intake and feeding method | csv (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 data | csv (141.95 KB); sas7bdat (1.11 MB) | ||
Research Labs - Whole Blood Dataset | Contains whole blood lab results | sas7bdat (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 status | csv (5.52 KB); sas7bdat (512 KB) | ||
Laboratory Dataset | Captures laboratory results data | csv (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-up | csv (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 data | csv (75.22 KB); sas7bdat (8.38 MB) | ||
Protocol Deviation Log Dataset | Captures information related to any protocol deviations | csv (1.79 KB); sas7bdat (408 KB) | ||
Derived Dataset | Contains derived data used in primary publication analysis | csv (12.95 KB); sas7bdat (240 KB) | ||
Physical Exam Dataset | Captures data from physical exam, such as vital signs and symptoms | csv (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 data | sas7bdat (200 KB); csv (664 B) | ||
IV Dataset | Captures information related to IV access, including event status and access required | csv (4 KB); sas7bdat (612 KB) | ||
DD Adverse Events Dataset | Captures information related to adverse events | csv (22.86 KB); sas7bdat (1 MB) | ||
Dosing Derived Dataset | Captures derived data related to dosing, such as IVIG dose, dosing weight, and compliance | csv (15.1 KB); sas7bdat (1008 KB) |