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

16. září 2026 aktualizováno: Fox Chase Cancer Center

GU-228: Mycophenolate Mofetil in Combination With Prednisone as First-line Treatment of Immune Related Hepatitis Resulting From Checkpoint Inhibitor Therapy

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.

Přehled studie

Detailní popis

This is a phase II, interventional clinical trial evaluating the efficacy and safety of combination immunosuppression with mycophenolate mofetil (MMF) plus prednisone for the treatment of grade 2-3 immune-related adverse event (irAE) hepatitis associated with immune checkpoint inhibitor therapy (PD-(L)1 and/or Cytotoxic T-lymphocyte associated protein 4 (CTLA-4) inhibitors).

Eligible patients are those with laboratory-defined grade 2-3 hepatitis and a clinical diagnosis consistent with irAE hepatitis. When feasible, liver biopsy is obtained within 120 hours of diagnosis to confirm the diagnosis and evaluate alternative causes of liver injury; however, biopsy is not required in patients where the procedure is deemed unsafe (e.g., due to coagulopathy or ascites). Treatment initiation must occur within 72 hours of diagnosis. In select patients where clinically appropriate, immunosuppressive therapy may be delayed up to 48 hours to allow for diagnostic evaluation, including biopsy and viral testing.

Patients in whom an alternative cause of liver injury is considered more likely (e.g., tumor involvement) or whose liver function improves to grade 1 or lower without intervention will not receive protocol-directed treatment and will instead enter an observational follow-up cohort. Patients with grade 4 hepatitis at presentation are excluded due to safety concerns with the study's treatment approach.

All treated patients receive combination therapy consisting of fixed-dose MMF and physician-directed prednisone. Prednisone tapering is guided by suggested protocols but ultimately determined by the treating physician. Dose modification guidelines are provided for MMF.

The primary endpoint is treatment response at 30 days from irAE hepatitis diagnosis, defined as improvement to grade 0-1 hepatitis with successful steroid taper to ≤20 mg prednisone equivalent daily. The primary analysis will be conducted on an intention-to-treat basis.

The study uses a Simon two-stage design to evaluate efficacy. The null hypothesis assumes a response rate of ≤60%, while the alternative hypothesis assumes a response rate of ≥80%. Stage I includes 11 evaluable patients, and Stage II includes 20 patients, with interim futility analyses conducted at each stage. Early safety monitoring includes toxicity assessment in the first 8 patients. A steering committee will regularly review safety data to monitor for patient harm.

Treatment failure is defined as lack of response at 30 days or death attributable to irAE, severe infection, or treatment-related toxicity. Death due to underlying cancer progression is not considered treatment failure.

This study aims to determine whether early combination immunosuppression can improve outcomes in patients with immune-mediated hepatitis while maintaining an acceptable safety profile.

Typ studie

Intervenční

Zápis (Odhadovaný)

31

Fáze

  • Fáze 2

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní kontakt

Studijní místa

    • Pennsylvania
      • Philadelphia, Pennsylvania, Spojené státy, 19111
        • Nábor
        • Fox Chase Cancer Center
        • Kontakt:

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

  • Dospělý
  • Starší dospělý

Přijímá zdravé dobrovolníky

Ne

Popis

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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 active hepatitis B (HBV), hepatitis C (HCV), or tuberculosis. Confirmatory testing for these infections may be performed if no recent result is available in the patient's records and, in the opinion of the treating physician, such testing is clinically warranted. Past HBV is exclusionary. Patients with treated HCV infection and documented eradication are eligible to enroll.
  • 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:

    1. Patients being treated with vasopressors
    2. Patient requiring mechanical ventilation or extracorporeal membrane oxygenation
    3. Patients being treated with parenteral antibiotic, antifungal, or antiviral medications
  • Patients with chronic autoimmune diseases requiring long-term immunosuppressive treatment at the time of enrollment, or for whom the mechanism of index inflammatory changes is too uncertain to initiate irAE-directed treatment at the discretion of treating provider.
  • Patients with congenital (e.g. combine variable immunodeficiency) or acquired (e.g. chronic leukemia) immunodeficiency requiring long-term prophylactic antimicrobial treatment at time of enrollment (e.g. intravenous immunoglobulin (IVIg), prophylactic antiviral treatment, etc.)
  • Women of child-bearing potential (WOCBP) who are pregnant or breast-feeding.
  • Patients unable to commit to use of reliable contraception throughout the study period.

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

  • Primární účel: Léčba
  • Přidělení: N/A
  • Intervenční model: Přiřazení jedné skupiny
  • Maskování: Žádné (otevřený štítek)

Zbraně a zásahy

Skupina účastníků / Arm
Intervence / Léčba
Experimentální: Mycophenolate mofetil (MMF) and prednisone
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
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.

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Response rate of immunotherapy (IO)-related hepatitis to MMF and prednisone
Časové okno: 30 days
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

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Frequency of bacterial, viral, or fungal infection requiring systemic treatment
Časové okno: 90 days
The frequency of bacterial, viral, or fungal infection requiring systemic treatment within 90 days of initiation of MMF plus prednisone
90 days

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Vyšetřovatelé

  • Vrchní vyšetřovatel: Matthew Zibelman, MD, Fox Chase Cancer Center

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia (Aktuální)

23. července 2026

Primární dokončení (Odhadovaný)

31. března 2028

Dokončení studie (Odhadovaný)

30. září 2028

Termíny zápisu do studia

První předloženo

17. června 2026

První předloženo, které splnilo kritéria kontroly kvality

17. června 2026

První zveřejněno (Aktuální)

23. června 2026

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Aktuální)

17. září 2026

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

16. září 2026

Naposledy ověřeno

1. září 2026

Více informací

Termíny související s touto studií

Plán pro data jednotlivých účastníků (IPD)

Plánujete sdílet data jednotlivých účastníků (IPD)?

NE

Informace o lécích a zařízeních, studijní dokumenty

Studuje lékový produkt regulovaný americkým FDA

Ano

Studuje produkt zařízení regulovaný americkým úřadem FDA

Ne

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