Mycophenolate Mofetil w/ Prednisone for Immune Related Hepatitis From Checkpoint Inhibitor Therapy

NCT07663422 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 25-1048GU-228
• Study Type: interventional
• Target: 31
• Locations: 0 countries
• Sites: N/A

Brief Summary

This phase II study evaluates whether early treatment with mycophenolate mofetil (MMF) plus prednisone improves liver inflammation caused by immune checkpoint inhibitors. The study includes patients who develop moderate to severe immune-related hepatitis after receiving PD-(L)1 or CTLA-4-based cancer therapy. The main goal is to determine how many patients experience improvement in liver function within 30 days while successfully tapering steroids. Safety and treatment-related side effects will also be monitored.

Conditions

Immune-mediated Hepatitis; Immune-related Adverse Event

Keywords

irAE; Immune-mediated hepatitis; Immune-related Adverse Event

Outcome Measures

Primary Outcomes (1)

• Response rate of immunotherapy (IO)-related hepatitis to MMF and prednisone

  The response rate of IO-related hepatitis to MMF and prednisone defined as achievement of corticosteroid (CS) dose of ≤ 20 mg prednisone with improvement to G1 or better as protocol defined at day 30 following treatment initiation

  30 days

Secondary Outcomes (1)

• Frequency of bacterial, viral, or fungal infection requiring systemic treatment

  90 days

Study Arms (1)

Mycophenolate mofetil (MMF) and prednisone

EXPERIMENTAL

Patients who present with G2-3 irAE hepatitis by lab value and confirmed diagnosis by liver biopsy will be treated with combination therapy of MMF plus prednisone for 30 days

Drug: Mycophenolate Mofetil plus prednisone

Interventions

Mycophenolate Mofetil plus prednisone DRUG

MMF dosing is 1000 mg orally twice daily, and dosing for individual patients will be managed based on toxicity monitoring rules. The addition of alternative agents for treatment of irAEs will be at the treating Oncologist/Hematologist's discretion. Patients for whom prednisone is held may resume those agents at treating Oncologist/Hematologist's discretion, while rules for MMF re-challenge are stipulated. At the end of the 30-day primary study period, patients may continue one or both agents as deemed appropriate by the treating physician.

Mycophenolate mofetil (MMF) and prednisone

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must be ≥ 18 years at the time of irAE diagnosis and must have been diagnosed with a solid tumor or hematologic malignancy being treated with non-curative intent.
• Patients must be diagnosed or presumed to have by a treating Medical Oncology or Hematology-Oncology clinician with immune-related hepatitis, G2-G3 (as defined in section 11.0), with plan for at least temporary interruption of IO therapy and initiation of corticosteroid treatment.
• Patients must have been previously treated for any malignancy with at least one dose of an IO agent targeting the Programmed cell death protein 1 (PD-1)/programmed death-ligand 1 (PDL-1) or CTLA-4 axis. IO use may have occurred at any time prior to diagnosis of irAE.
• Serum aspartate aminotransferase (AST), alanine aminotransferase (ALT), and total bilirubin must have been measured within screening window outlined in Section 8.0 (Study Calendar).
• Patient must be able to take study medications by mouth.
• Baseline hemoglobin (HgB) at time of enrollment ≥8.0 g/dL, absolute neutrophil count (ANC) ≥ 1500/mm3, and platelet count ≥ 100,000/mm3 without red blood cell or platelet transfusion in the two weeks preceding measurement of lab values. Lab values are to be assessed within screening window outlined in Section 8.0 (Study Calendar).
• Baseline estimated Glomerular Filtration Rate (eGFR) \< 30 mL/min/1.73m2
• Patients must be able to understand and willing to sign a written informed consent and HIPAA consent document.

You may not qualify if:

• Patients previously treated for irAE hepatitis.
• Patients diagnosed or presumed to have G4 immune-related hepatitis.
• Patients receiving renal replacement therapy with hemodialysis or peritoneal dialysis at time of enrollment.
• Patients with a documented history of Child-Turcotte-Pugh class B and C liver dysfunction.
• Patients with significant liver dysfunction at time of presentation, as defined by an otherwise unexplained change in mental status or International Normalized Ratio (INR) ≥ 1.5 attributed to synthetic liver dysfunction.
• Patients with uncontrolled human immunodeficiency virus (HIV), Epstein-Barr virus (EBV), cytomegalovirus (CMV), herpes simplex virus (HSV), or Varicella-zoster virus (VZV). Confirmatory testing for these infections is not required.
• Patients with a history of gastrointestinal bleeding, ulceration, or perforations within one year of trial enrollment. Use of proton-pump inhibitor therapy is allowed.
• Patients with any history of solid organ or allogeneic stem cell transplantation.
• Patients with type I diabetes mellitus (DM).
• Patients with type II DM with most recent glycated hemoglobin measurement greater than or equal to 9.0%.
• Patients with a documented history of phosphoribosyl-transferase deficiency.
• History of allergic reactions to MMF, prednisone, methylprednisolone, or dexamethasone.
• Patients with decompensated congestive heart failure at time of enrollment, at the discretion of treating clinician, regardless of ejection fraction.
• Patients with history of suicidal ideation or past suicide attempt.
• Uncontrolled or intercurrent severe medical illness at the time of enrollment, as defined below:

Sponsors & Collaborators

• Fox Chase Cancer Center: lead

MeSH Terms

Interventions

Mycophenolic Acid; Prednisone

Intervention Hierarchy (Ancestors)

Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids; Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Matthew Zibelman, MD

  Fox Chase Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ryan Romasko, MBA

CONTACT

2678388380; ProtocolDev@fccc.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 17, 2026
• First Posted: June 23, 2026
• Study Start: June 30, 2026
• Primary Completion (Estimated): March 31, 2028
• Study Completion (Estimated): September 30, 2028
• Last Updated: June 23, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share