Mycophenolate Mofetil Versus Azathioprine in Treatment Naive Autoimmune Hepatitis
Status:
Unknown status
Trial end date:
2019-12-01
Target enrollment:
Participant gender:
Summary
Rationale: Current standard therapy of autoimmune hepatitis consists of a combination of
prednisolone and azathioprine. However, a significant proportion of patients does not respond
to, or is intolerant for, azathioprine. Mycophenolate mofetil (MMF) has surpassed
azathioprine as therapy to prevent organ transplant rejection and is sometimes used as an
alternative option for autoimmune hepatitis. Several case series and one prospective study
have documented the efficacy and safety of mycophenolate mofetil as induction therapy for
autoimmune hepatitis. Robust evidence from a formal randomized clinical trial is lacking.
Objective: To assess the efficacy and safety of mycophenolate mofetil as induction therapy in
patients with treatment naive autoimmune hepatitis.
Study design: Multicenter, randomised, open-label intervention study Study population:
Patients with newly diagnosed autoimmune hepatitis who are in need of induction therapy
according to current guidelines.
Intervention: The intervention group will receive oral mycophenolate mofetil for 24 weeks.
The control group will be treated with azathioprine for 24 weeks. Both groups will be treated
with steroid induction which will closely follow the schedule from the recent Clinical
Practice Guidelines by the European Association for Study of the Liver (EASL).
Main study parameters/endpoints: The primary outcome is the proportion of patients in
remission, defined as normalization of serum alanine transaminase (ALT) and immunoglobulin G
(IgG) levels after 24 weeks of treatment, per treatment group. Secondary endpoints include
safety and tolerability of mycophenolate mofetil, time to remission, changes in Model For
End-Stage Liver Disease (MELD) -score (and its components bilirubin, INR, creatinine),
albumin, pseudocholinesterase and N-terminal procollagen-III-peptide, ELF (Enhanced Liver
Fibrosis) -score and aspects of quality of life. In a sub-study, drug levels will be
measured.