Short versus long maribavir treatment after allogeneic stem cell transplant

Comparing the Efficacy of Different Durations of Maribavir Treatment Regimens in Patients With Refractory Cytomegalovirus Infection Following Allo-HSCT: a Prospective, Multicenter, Randomized, Controlled Clinical Trial

This trial tests whether a shorter or longer course of maribavir helps adults with refractory CMV infection after their first allogeneic stem cell transplant.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 218 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Ruijin Hospital (Academic / other)
• Locations: 1 site (Shanghai)
• Trial ID: NCT07511127 on ClinicalTrials.gov

What this trial studies

This Phase 3, multicenter randomized controlled trial compares different durations of maribavir therapy in adults who develop refractory or drug‑resistant CMV following a first allogeneic hematopoietic stem cell transplant. Participants will be randomized to prespecified maribavir treatment lengths and followed for virologic clearance, clinical response, and safety outcomes. The protocol is informed by retrospective data suggesting treatment duration may affect outcomes and uses standardized CMV DNA monitoring and clinical assessments. The lead site is Ruijin Hospital, Shanghai JiaoTong University School of Medicine.

Who should consider this trial

Good fit: Adults aged 18 or older who had their first allogeneic HSCT and have confirmed refractory, drug‑resistant, or treatment‑intolerant CMV infection and can give informed consent.

Not a fit: Patients without refractory or drug‑resistant CMV, those under 18, those with prior allogeneic transplants, or those unable to tolerate maribavir are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, the trial could identify an optimal maribavir treatment length that improves CMV clearance while reducing toxicity and relapse.

How similar studies have performed: Previous trials and regulatory decisions have shown maribavir is effective for resistant or refractory CMV, but randomized evidence on optimal treatment duration is limited.

Eligibility criteria

Inclusion Criteria:

1. First allogeneic hematopoietic stem cell transplantation;
2. Age ≥ 18 years;
3. Confirmed refractory CMV infection;

   Refractory CMV infection is defined as fulfillment of any one of the following criteria:
   * Persistent or increasing CMV viremia despite ≥2 weeks of appropriate antiviral therapy-specifically, CMV DNA levels remain unchanged (i.e., change ≤ log₁₀) or increase (i.e., change \> log₁₀)-concomitant with lack of clinical improvement or ongoing disease progression;
   * Drug-resistant CMV infection-defined as detection of specific CMV gene mutations associated with reduced susceptibility to one or more anti-CMV agents, in patients who otherwise meet the criteria for refractory CMV infection;
   * Intolerance to anti-CMV therapy-defined as inability to continue antiviral treatment due to severe adverse effects, such as clinically significant bone marrow suppression or renal impairment;
4. Provision of written informed consent and willingness to participate in this clinical study.

Exclusion Criteria:

1. Known allergic constitution, particularly hypersensitivity to any component of maribavir;
2. Active hepatitis B infection, defined as HBV DNA level ≥ 1 × 10³ IU/mL;
3. Confirmed HIV infection;
4. Severe impairment of major organ function, including but not limited to respiratory failure, cardiac failure, decompensated hepatic insufficiency, or renal insufficiency;
5. Central nervous system CMV infection;
6. History of substance use disorder or chronic alcoholism that may compromise the validity or interpretation of trial outcomes;
7. Presence of a psychiatric disorder or cognitive impairment precluding the provision of informed consent;
8. Any other condition deemed by the investigator to render the participant unsuitable for enrollment in this clinical trial.

Where this trial is running

Shanghai

• Ruijin Hospital, Shanghai JiaoTong University School of Medicine — Shanghai, China (Recruiting)

Study contacts

• Study coordinator: Xiaoxia Hu

Conditions Hematologic Malignancy