A Randomised Clinical Trial Assessing the Efficacy and Safety of Mycophenolate Mofetil Versus Azathioprine for Induction of Remission in Treatment Primary Biliary Cholangitis-Autoimmune Hepatitis Overlap Syndrome

Xiaoli Fan

Status and phase: Unknown; Phase 4

Conditions: Autoimmune Hepatitis; Primary Biliary Cirrhosis

Treatments: Drug: Methylprednisolone and azathioprine; Drug: Methylprednisolone and Mycophenolate mofetil

Study type: Interventional

Funder types: Other

Identifiers

NCT04933292

OS-2

Details and patient eligibility

About

Current standard therapy of primary biliary cholangitis-autoimmune hepatitis overlap syndrome(PBC-AIH overlap) consists of a combination of prednisolone and azathioprine. However, a significant proportion of patients may do not respond to, or is intolerant for azathioprine. Several studies have documented the efficacy and safety of mycophenolate mofetil(MMF) as second-line therapy for PBC-AIH overlap. However, robust evidence from a formal randomized clinical trial for the first-line immunosuppressor is in need.

Enrollment: 78 estimated patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Patients aged 18-70 years;
2. Diagnosed with PBC-AIH overlap syndrome according to Paris criteria;
3. Agreed to participate in the trial, and assigned informed consent;
4. The WBC count ≥2.5x10^9/L and platelet count ≥50x10^9/L.

Exclusion criteria

1. The presence of hepatitis A, B, C, D, or E virus infection;
2. Patients with presence of serious decompensated cirrhosis;
3. Patients have a history of glucocorticoid or immunosuppressant medication before enrollment;
4. Liver damage caused by other reasons: such as primary sclerosing cholangitis, non-alcoholic steatohepatitis, drug induced liver disease or Wilson disease.
5. Pregnant and breeding women;
6. Severe disorders of other vital organs, such as severe heart failure, cancer;
7. Parenteral administration of blood or blood products within 6 months before screening;
8. Recent treatment with drugs having known liver toxicity;
9. Taken part in other clinic trials within 6 months before enrollment.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

78 participants in 2 patient groups

Methylprednisolone and Mycophenolate mofetil

Experimental group

Treatment:

Drug: Methylprednisolone and Mycophenolate mofetil

Methylprednisolone and Azathioprine

Active Comparator group

Treatment:

Drug: Methylprednisolone and azathioprine