Comparing the Efficacy of Different Durations of Maribavir Treatment Regimens in Allo-HSCT

Official title

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

Overview

To comparing the efficacy of different durations of Maribavir treatment regimens in patients suffering from refractory CMV infection after allo-HSCT.

Detailed description

Hematopoietic stem cell transplantation (HSCT) represents the only potentially curative modality for hematologic malignancies. Nevertheless, post-transplant infections substantially elevate the risk of transplant-related mortality, with cytomegalovirus (CMV) infection being among the most prevalent complications. Although advances in prophylactic strategies and preemptive antiviral therapy have contributed to a measurable reduction in both the incidence of CMV infection and CMV disease, refractory or drug-resistant (R/R) CMV infection following HSCT remains a significant global therapeutic challenge. Recent epidemiologic data indicate that the incidence of drug-resistant CMV infection in HSCT recipients ranges from 1.7% to 14.5%, while that of refractory CMV infection falls between 29% and 39%. Notably, in China, the incidence of refractory CMV infection after HSCT is slightly higher than the global average-approximately 47% . Therefore, the investigator conduct a multicenter, randomized, controlled study based on retrospective research to further explore the efficacy of different durations of Maribavir treatment regimens in Allo-HSCT.

Interventions

• Drug Maribavir
  During the treatment period, maribavir is administered orally at a dosage of 400 mg twice daily. Participants will be stratified by clinical trial and received oral medication for varying durations.

Primary outcome measures

• The incidence of recurrent CMV infection [Time frame: The primary endpoint is assessed 8 weeks after maribavir discontinuation following allo-HSCT]

Secondary outcome measures (3)

• The incidence of recurrent CMV infection [Time frame: The secondary endpoint is assessed 16 weeks after maribavir discontinuation following allo-HSCT]
• The incidence of recurrent CMV disease [Time frame: Follow-up is conducted for 8 weeks following maribavir discontinuation.]
• CMV resistance mutations [Time frame: Through study completion. It is expected within one year post-transplant.]

Eligibility criteria

Inclusion criteria

• First allogeneic hematopoietic stem cell transplantation;
• Age ≥ 18 years;
• 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;
• Provision of written informed consent and willingness to participate in this clinical study.

Exclusion criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Open label

Primary purpose

Treatment

Study locations

China · 1 center

• Ruijin Hospital, Shanghai JiaoTong University School of Medicine — Shanghai

Identifiers

NCT: NCT07511127 · RJBMT-2026CMV

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗