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

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

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

Study Overview

• Status: Recruiting
• Conditions: Hematologic Malignancy
• Intervention / Treatment: Drug: Maribavir

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.

• Study Type: Interventional
• Enrollment (Estimated): 218
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Xiaoxia Hu; Phone Number: +86-021-64370045; Email: hxx12276@rjh.com.cn
• Study Contact Backup: Name: Luxiang Wang; Phone Number: +86-021-64370045; Email: wlx12369@rjh.com.cn

Study Locations

• China
  • Shanghai, China
    • Recruiting
    • Ruijin Hospital, Shanghai Jiaotong University School of Medicine
    • Contact: Luxiang Wang; Phone Number: +86 021 64370045; Email: wlx12369@rjh.com.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Fixed-duration group; The fixed-duration group that discontinued maribavir treatment after 8 weeks.	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.
Experimental: PCR-guided group; The experimental group discontinued maribavir after achieving two consecutive negative CMV-DNA PCR test, with a one-week interval, following drug administration.	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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The incidence of recurrent CMV infection	The incidence of recurrent CMV infection within 8 weeks after maribavir discontinuation	The primary endpoint is assessed 8 weeks after maribavir discontinuation following allo-HSCT

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Ruijin Hospital

Study record dates

Study Major Dates

• Study Start (Actual): February 28, 2026
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2028

Study Registration Dates

• First Submitted: March 13, 2026
• First Submitted That Met QC Criteria: March 31, 2026
• First Posted (Actual): April 6, 2026

Study Record Updates

• Last Update Posted (Actual): April 6, 2026
• Last Update Submitted That Met QC Criteria: March 31, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Hematologic Neoplasms; maribavir
• Other Study ID Numbers: RJBMT-2026CMV

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No