Maribavir for CMV Infection

Recruiting at 74 trial locations

Overseen By Takeda Contact

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Takeda

Must not be taking: Valganciclovir, Ganciclovir, others

Disqualifiers: CNS CMV, Severe liver disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

80 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and effectiveness of maribavir for treating CMV (Cytomegalovirus) infections in children and teens who have undergone a transplant. The study will determine the best dose of maribavir, administered as tablets or in liquid form, over an 8-week period. Children and teens under 18 who have had a solid organ or stem cell transplant and are dealing with CMV infections unresponsive to other treatments may qualify for this trial. Participants will visit their doctor for check-ups during a 12-week follow-up period after the treatment concludes. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications like valganciclovir, ganciclovir, cidofovir, foscarnet, leflunomide, letermovir, or artesunate before starting the study treatment. If you are on any of these, you will need to stop them before participating.

Is there any evidence suggesting that maribavir is likely to be safe for humans?

Research has shown that maribavir is generally safe. In studies, maribavir caused fewer issues with low white blood cell counts compared to treatments like valganciclovir and ganciclovir. It also led to fewer kidney problems than foscarnet. This suggests maribavir might be gentler on the blood and kidneys than these other drugs.

The FDA has approved maribavir for treating certain difficult-to-treat CMV infections, indicating its general safety for use.

Overall, maribavir appears to be well-tolerated by patients. However, as with any treatment, discussing possible side effects with a doctor is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for CMV infection?

Most treatments for CMV infection, like ganciclovir and valganciclovir, work by inhibiting viral DNA synthesis. But maribavir stands out because it targets a unique part of the virus called the UL97 protein kinase, disrupting the virus's ability to replicate in a different way. This novel mechanism of action offers hope for patients, especially those who might not respond well to existing treatments. Researchers are excited because maribavir could provide a more effective option with potentially fewer side effects and a different resistance profile compared to current therapies.

What evidence suggests that maribavir might be an effective treatment for CMV infection?

Research has shown that maribavir effectively treats CMV (cytomegalovirus) infections in organ transplant patients. In some studies, up to 69% of patients experienced clearance of their CMV infections while taking maribavir. One study found that maribavir helped treat CMV infections unresponsive to other treatments, particularly in kidney and lung transplant patients. Notably, about 43.8% of patients with difficult-to-treat CMV infections achieved viral clearance within eight weeks. This trial will evaluate maribavir in different dosages based on age and weight, with participants receiving treatment for up to eight weeks. Overall, maribavir has shown promising results in treating challenging CMV cases. ^{2 6 7 8 9}

Who Is on the Research Team?

Study Director

Principal Investigator

Takeda

Are You a Good Fit for This Trial?

This trial is for children and teenagers under 18 who've had a solid organ or stem cell transplant, have CMV infection with specific blood test results, and whose transplants are working. They need to be healthy enough in terms of blood cells and kidney function, agree to birth control if applicable, and expect to live at least 8 more weeks.

Inclusion Criteria

I have a confirmed CMV infection with specific test results.

Have life expectancy of >= 8 weeks.

I am under 18 years old. If in Cohort 3, I was born full-term and weigh at least 5 kg.

Exclusion Criteria

I don't have any health issues that could affect the study or make it unsafe for me.

I do not have HIV, confirmed by a test within the last 3 months.

My body weight is low, affecting how much blood can be taken for the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive maribavir for the treatment of CMV infection

8 weeks

Regular visits for dose administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

Participants need to visit their doctor during this period

What Are the Treatments Tested in This Trial?

Interventions

• Maribavir

Trial Overview The study tests the safety, tolerability, and proper dosing of Maribavir (an antiviral medication) in treating CMV infections in young transplant recipients. Participants will take Maribavir for 8 weeks with follow-up doctor visits over a subsequent 12-week period.

How Is the Trial Designed?

Group I: Cohort 3: Maribavir 400, 200, 100 or 50 mg Experimental Treatment 1 Intervention

Participants with 0 to \< 6 years of age will receive maribavir 400 mg (2\*200 mg tablets or powder for oral suspension) BID based on body weight \>= 25 kg; or 200 mg tablet or powder for oral suspension BID based on body weight 14 to \< 25 kg; or 100 mg powder for oral suspension BID based on body weight 10 to \< 14 kg; or 50 mg powder for oral suspension BID based on body weight 7 to \< 10 kg; or 50 mg powder for oral suspension once daily (QD) based on body weight 5 to \<7 kg for up to 8 weeks treatment period (Day 1/Week 0 to Day 56/Week 8).

Group II: Cohort 2: Maribavir 400, 200 or 100 mg Experimental Treatment 1 Intervention

Participants with \>= 6 to \< 12 years of age will receive maribavir 400 mg (2\*200 mg tablets or powder for oral suspension) BID based on body weight \>= 25 kg; or 200 mg tablet or powder for oral suspension BID based on body weight 14 to \< 25 kg; or 100 mg powder for oral suspension BID based on body weight 10 to \< 14 kg orally for up to 8 weeks treatment period (Day 1/Week 0 to Day 56/Week 8).

Group III: Cohort 1: Maribavir 400, 200 or 100 mg Experimental Treatment 1 Intervention

Participants with greater than or equal to (\>=) 12 to less than (\<) 18 years of age will receive maribavir 400 milligrams (mg) (2\*200 mg tablets or powder for oral suspension) twice daily (BID) based on body weight \>= 25 kilogram (kg); or 200 mg tablet or powder for oral suspension BID based on body weight 14 to \< 25 kg; or 100 mg powder for oral suspension BID based on body weight 10 to \< 14 kg for up to 8 weeks treatment period (Day 1/Week 0 to Day 56/Week 8).

Who Is Running the Clinical Trial?

Takeda

Lead Sponsor

Trials

1,255

Recruited

4,219,000+

Dr. Naoyoshi Hirota

Takeda

Chief Medical Officer since 2020

MD from University of Tokyo

Christophe Weber

Takeda

Chief Executive Officer since 2015

PhD in Molecular Biology from Université de Montpellier

Takeda Development Center Americas, Inc.

Industry Sponsor

Trials

58

Recruited

10,800+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39826146/

Real-World Experience With Maribavir for Treatment of ... While on maribavir, nine (69%) patients had a resolution of CMV infection. Treatment-emergent maribavir resistance was documented in one patient ...

2. jhltonline.org jhltonline.org/article/S1053-2498(24)01971-5/fulltext

Maribavir for refractory cytomegalovirus infection (with or ... The results demonstrated an efficacy and safety benefit of maribavir in treating refractory CMV after common SOTs, such as kidney and lung ...

3. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1600613525030692

Determinants of Virological Outcomes in Kidney Transplant ... Maribavir has demonstrated promising efficacy in treating refractory cytomegalovirus (CMV) infections among solid organ transplant ...

4. ectrx.org ectrx.org/detail/current/2025/23/4/0/306/0

Two Cases of Maribavir Treatment of Refractory/Resistant ... Among patients with refractory (nonresistant) CMV infection, only 43.8% reached viremia clearance within 8 weeks. In contrast, 62.8% of patients ...

5. academic.oup.com academic.oup.com/jid/article/231/3/e470/7762822

Comparative Emergence of Maribavir and Ganciclovir ... Viral clearance was confirmed in 17 (74%) of 23 patients with emergent maribavir resistance after retreatment with an alternative CMV antiviral ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9464078/

Maribavir for Refractory Cytomegalovirus Infections With or ... Maribavir demonstrated an improved safety profile versus valganciclovir/ganciclovir with respect to neutropenia and versus foscarnet with respect to AKI, which ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8926008/

Evaluating the Safety of Maribavir for the Treatment of ... Maribavir is a novel agent with CMV activity approved by Federal Food and Drug Administration (FDA) in December 2021 for resistant/refractory disease.

8. clinicaltrials.gov clinicaltrials.gov/study/NCT02931539

NCT02931539 | Efficacy and Safety Study of Maribavir ... Percentage of participants who achieved confirmed CMV viremia clearance at end of Week 8 after starting maribavir rescue treatment were reported. From start of ...

9. sciencedirect.com sciencedirect.com/science/article/pii/S1053249824019715

Maribavir for refractory cytomegalovirus infection (with or ... In this subgroup analysis, a higher proportion of patients treated with maribavir achieved CMV viremia clearance compared with that in the IAT arm, across all ...