A phase 2, multicentric study to assess the efficacy and safety of maribavir treatment in hematopoietic stem cell transplant (HSCT) recipients with cytomegalovirus (CMV) infection with a medical condition that contraindicate the administration of ganciclovir, valganciclovir or foscarnet and in those who discontinued first line preemptive therapy with ganciclovir, valganciclovir or foscarnet due to toxicity or intolerance.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Gruppo Italiano Per Il Trapianto Di Midollo Osseo Cellule Staminali Emopoietiche E Terapia Cellulare

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15], Diseases [C] - Virus Diseases [C02]

Trial duration

15 May 2026 → ongoing

Decision date (initial)

2025-11-17

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Takeda Italia S.p.A.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy, Safety

- To evaluate the efficacy of maribavir, at the end of Study Week 8, in HSCT recipients with CMV infection treated with the antiviral drug as first line therapy in patients with 1) a medical condition that contraindicate the administration of ganciclovir, valganciclovir or foscarnet and 2) as second line therapy in patients who discontinued first line preemptive therapy with ganciclovir, valganciclovir or foscarnet due to toxicity or intolerance.

- To assess the side effects requiring treatment discontinuation of maribavir in HSCT recipients with CMV infection administered as first line therapy in patients with 1) a medical condition that contraindicate the administration of ganciclovir, valganciclovir or foscarnet and 2) as second line therapy in those who discontinued first line preemptive antiviral therapy with ganciclovir, valganciclovir or foscarnet due to toxicity or intolerance.

Secondary objectives 3

1. To evaluate the recurrence of CMV DNAemia when patients are on treatment and off treatment up to 8 weeks from the discontinuation of maribavir therapy.
2. Evaluation of late response in patients with partial response at 8 weeks who continued treatment up to a maximum of 12 weeks.
3. To evaluate the overall safety and tolerability of maribavir during the period from the start of treatment through day 7 after the last dose.

Conditions and MedDRA coding

Allogeneic peripheral hematopoietic stem cell transplant

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 14

1. Allogeneic hematopoietic stem cell transplant performed within previous twelve months.
2. Diagnosis of CMV infection.
3. Patients with a medical condition that contraindicate the administration of ganciclovir, valganciclovir of foscarnet (patients with kidney failure, kidney disease, kidney dysfunction; patients with delayed state or degree of bone marrow engraftment; patients with previous CMV infections who have experienced SoC toxicity), or patients who discontinued first line antiviral therapy with ganciclovir, valganciclovir or foscarnet due to toxicity or intolerance.
4. Age > 18 years.
5. Weigh ≥40 kg.
6. Able to swallow tablets.
7. Performance status: ECOG <3.
8. Adequate hepatic function (bilirubin ≤2 UNL; ALT/AST ≤2,5 UNL).
9. Adequate renal function (creatinine clearance ≥50 ml/min).
10. Absence of diarrhea or other intestinal symptoms or active intestinal pathology.
11. Life expectancy not severely limited by concomitant illness.
12. Women of childbearing potential must use highly effective contraception for at least 1 month after the last dose of maribavir
13. Willing and able to comply with all of the requirements and visits in the protocol.
14. ten and signed informed consent.

Exclusion criteria 7

1. Severe vomiting, diarrhea, or other severe gastrointestinal illness within 24 hours prior to the first dose of study drug that would preclude administration of oral/enteral medication.
2. Any active, uncontrolled infection.
3. End-organ CMV disease.
4. Patients with other life-threatening concurrent disease.
5. Subjects with known hypersensitivity to any of the component medication.
6. Non-cooperative behaviour or non-compliance.
7. Participation in another clinical trial within 1 month before the start of this trial.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. The primary efficacy end point is the percentage of patients with a response to treatment, defined as plasma CMV DNA level below the lower limit of quantification (confirmed at least in two consecutive tests), at 8 weeks after the start of treatment.
2. The primary safety end point is the incidence of side effects that occurred or worsened during the treatment period and required maribavir therapy discontinuation.

Secondary endpoints 3

1. The secondary efficacy endpoint is to describe the incidence and frequency of CMV DNAemia detection occurring when patients are on treatment and off treatment up to 8 weeks from the discontinuation of maribavir therapy. The efficacy will be evaluated also according to the CMV infection risk.
2. The secondary endpoint of late response is the proportion of patients with a partial response at 8 weeks of treatment who continued on therapy per investigator’s judgment, with a subsequent response within 12 weeks, defined as CMV DNAaemia below the level of quantification (confirmed in at least two consecutive tests).
3. The secondary safety endpoint is to describe the incidence of grade > 2 side effects considered related to maribavir therapy during the treatment period.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Gruppo Italiano Per Il Trapianto Di Midollo Osseo Cellule Staminali Emopoietiche E Terapia Cellulare

Sponsor organisation

Gruppo Italiano Per Il Trapianto Di Midollo Osseo Cellule Staminali Emopoietiche E Terapia Cellulare

Address

Via De' Poeti 1/7

City

Bologna

Postcode

40124

Country

Italy

Scientific contact point

Organisation

Gruppo Italiano Per Il Trapianto Di Midollo Osseo Cellule Staminali Emopoietiche E Terapia Cellulare

Contact name

GITMO Clinical Trials Office

Public contact point

Organisation

Gruppo Italiano Per Il Trapianto Di Midollo Osseo Cellule Staminali Emopoietiche E Terapia Cellulare

Contact name

GITMO Secretary

Third parties 2

Locations

1 EU/EEA country · 20 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 9 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications