A Phase 2b, Double-Blind, Three Arm, Randomized, Placebo Controlled Trial Testing High Dose Methylprednisolone or Equine Anti-Thymocyte Globulin for Acute Liver Failure in Children

What this Trial Is About

Researchers are studying treatments for children with Pediatric Acute Liver Failure (PALF), a rare and serious condition that affects liver function and can lead to death or the need for a liver transplant. This trial focuses on whether suppressing the immune system with either corticosteroids or equine anti-thymocyte globulin can improve survival in children aged 1 to under 18 years suffering from PALF with unknown causes. The study is a phase 2b, double-blind, randomized controlled trial aiming to evaluate the effectiveness and safety of these treatments compared to supportive care alone. Participants will be randomly assigned to one of three groups: one receiving high-dose methylprednisolone intravenously for four days, another receiving intravenous equine anti-thymocyte globulin for four days, or a third group receiving supportive care with placebo infusions. Following the initial treatment, those in the two active treatment arms will taper prednisolone doses orally from day 5 through day 42, while the supportive care group will receive a matching placebo taper. The trial also includes an observational cohort of up to 50 patients who meet eligibility but do not undergo randomized treatment, instead providing long-term observational data. During the study, participants will have follow-up visits at 1, 2, and 3 weeks after starting treatment, with some assessments done in hospital or outpatient settings. Additional visits at 6 weeks, 3 months, 6 months, and 12 months will be scheduled for ongoing monitoring and sample collection. Researchers will primarily measure survival with the native liver at 21 days and also evaluate survival at 180 days. Safety and treatment effects will be closely monitored throughout, with total participation lasting up to one year.

TReatment for ImmUne Mediated PathopHysiology