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Hematopoietic and Lymphoid Cell NeoplasmPhase 3NCT05711667

A Study of the Drug Letermovir as Prevention of Cytomegalovirus Infection After Stem Cell Transplant in Pediatric Patients

A Phase 3 study testing Biospecimen Collection and Letermovir in people with Hematopoietic and Lymphoid Cell Neoplasm and Malignant Solid Neoplasm, run by Children's Oncology Group at 33 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This phase III single arm trial determines whether taking prophylactic letermovir will reduce the likelihood of infection with cytomegalovirus (CMV) in children and adolescents after stem cell transplant compared to estimated rate of infection without prophylaxis. Read more about this study

Who is it for?
Ages 2 to 18 years
What is being tested?
Biospecimen Collection and Letermovir
Could I receive a placebo?
No, everyone receives an active treatment
How many people
105 participants
Where
33 sitesUnited States
Study dates
11 Jul 2024to 30 Jun 2029
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • >= 2 years and < 18 years at the time of enrollment
  • Weight must be >= 6 kg at the time of enrollment
  • Planned allogeneic HCT (bone marrow, peripheral blood stem cell, or cord blood transplant)
  • Patient must be CMV sero-positive (i.e., recipient CMV immunoglobulin G positive)
  • Note: If a patient has hypogammaglobulinemia but has previously been documented as CMV sero-positive, that is acceptable for study inclusion. For all patients already confirmed to be CMV IgG seropositive, repeat testing is not required within 7 days prior to enrollment. However, the laboratory data determining eligibility must be available in the patient's medical/research record for verification
  • Patient is eligible for entry only if it is feasible for plasma CMV PCR testing to be sent and resulted within the protocol mandated time period
Show all 12Show fewer
  • Reminder: To limit the likelihood of positive plasma CMV PCR post-enrollment and prior to start of study treatment period, it is recommended that patient enrollment proceed after patients start their transplant preparative regimen
  • Patient must have a performance status corresponding to Lansky/Karnofsky scores > 50
  • Note: Use Lansky for patients =< 16 years of age and Karnofsky for patients > 16 years of age. For further reference, see performance status scales scoring under the standard sections for protocols among protocol reference materials provided on the Children's Oncology Group (COG) member website: https://members.childrensoncologygroup.org/prot/reference_materials.asp
  • Estimated glomerular filtration rate > 10 mL/min/1.73 m^2 and not receiving dialysis
  • Direct bilirubin =< 2 mg/dL and serum glutamate-pyruvate transaminase (SPGT) (alanine transaminase [ALT]) =<10 x upper limit of normal (ULN) for age
  • Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L

Who cannot join

  • Expected inability to tolerate oral formulation of letermovir
  • Hypersensitivity to letermovir or any component of the formulation
  • History of CMV end organ disease within 6 months (180 days) prior to enrollment
  • Note: CMV end organ disease based on proposed definitions by Ljungman et al. and inclusive of proven, probable or possible disease
  • Receipt of prior allogeneic HCT within one year of study enrollment
  • Planned prophylactic administration of other anti-CMV medications or cellular products during the study, including:
Show all 26Show fewer
  • High dose acyclovir (defined as doses >= 1500 mg/m^2 IV or >= 3200 mg oral (patients >= 40 kg) or >= 2400 mg/m^2 (patients < 40 kg) per day)
  • High dose valacyclovir (defined as doses >= 3000 mg/day in patients > 20 kg)
  • Foscarnet
  • Ganciclovir
  • Valganciclovir
  • CMV-directed cytotoxic T lymphocytes
  • Planned receipt of the following contraindicated medications during the study treatment period; contraindicated medications must be discontinued at least 14 days prior to Day +1
  • Contraindicated medications for all patients:
  • Pimozide
  • Ergot alkaloids
  • Contraindicated medications for patients planned to receive cyclosporine:
  • Bosentan
  • Pitavastatin
  • Simvastatin
  • Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted in certain animal reproduction studies with letermovir. A pregnancy test is required for female patients of childbearing potential
  • Lactating females who plan to breastfeed their infants
  • Sexually active female patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their letermovir treatment and through at least 4 weeks after the last dose of letermovir.
  • Note: No contraception measures are needed specifically during letermovir treatment for male trial participants who have pregnant or non-pregnant female partner(s) of reproductive potential. Contraception measures may be required for other aspects of the HCT procedure.
  • All patients and/or their parents or legal guardians must sign a written informed consent
  • All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

Trial locations and contacts

33 sites in 1 country. Contact the study team or a site near you to ask about taking part.

33 sites in 1 country

United States33 sites, 32 recruiting

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

    Site Public Contact, 303-764-5056, josh.b.gordon@nsmtp.kp.org

    Sanam Shahid

    Recruiting

  • Johns Hopkins University/Sidney Kimmel Cancer Center

    Baltimore, Maryland, 21287

    Site Public Contact, 410-955-8804, jhcccro@jhmi.edu

    Heather J. Symons

    Recruiting

  • Children's Hospital of Alabama

    Birmingham, Alabama, 35233

    Site Public Contact, 205-638-9285, oncologyresearch@peds.uab.edu

    Joseph H. Chewning

    Recruiting

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

    Site Public Contact, 614-722-6039, Melinda.Triplet@nationwidechildrens.org

    Mark A. Ranalli

    Recruiting

  • Medical City Dallas Hospital

    Dallas, Texas, 75230

    Site Public Contact, 972-566-5588

    Maurizio L. Ghisoli

    Recruiting

  • UT Southwestern/Simmons Cancer Center-Dallas

    Dallas, Texas, 75390

    Site Public Contact, 214-648-7097, canceranswerline@UTSouthwestern.edu

    Victor M. Aquino

    Recruiting

  • Children's Hospital of Michigan

    Detroit, Michigan, 48201

    Site Public Contact, helpdesk@childrensoncologygroup.org

    Erin Goode

    Recruiting

  • Cook Children's Medical Center

    Fort Worth, Texas, 76104

    Site Public Contact, 682-885-2103, CookChildrensResearch@cookchildrens.org

    Richard P. Howrey

    Recruiting

  • Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital

    Grand Rapids, Michigan, 49503

    Site Public Contact, 616-391-1230, crcwm-regulatory@crcwm.org

    Kathleen Y. Butler

    Recruiting

  • Penn State Children's Hospital

    Hershey, Pennsylvania, 17033

    Site Public Contact, 717-531-6012

    Lisa M. McGregor

    Recruiting

  • Kapiolani Medical Center for Women and Children

    Honolulu, Hawaii, 96826

    Site Public Contact, 808-983-6090

    Wade T. Kyono

    Recruiting

  • Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

    Houston, Texas, 77030

    Site Public Contact, 713-798-1354, burton@bcm.edu

    Anil George

    Recruiting

  • Riley Hospital for Children

    Indianapolis, Indiana, 46202

    Site Public Contact, 800-248-1199

    April Rahrig

    Recruiting

  • University of Iowa/Holden Comprehensive Cancer Center

    Iowa City, Iowa, 52242

    Site Public Contact, 800-237-1225

    Andrew P. Groves

    Recruiting

  • Nemours Children's Clinic-Jacksonville

    Jacksonville, Florida, 32207

    Site Public Contact, 302-651-5572, Allison.bruce@nemours.org

    Sridhi Patel

    Recruiting

  • Children's Mercy Hospitals and Clinics

    Kansas City, Missouri, 64108

    Site Public Contact, 816-302-6808, COGResearchGroup@cmh.edu

    Lauren C. Wilson

    Recruiting

  • Norton Children's Hospital

    Louisville, Kentucky, 40202

    Site Public Contact, 502-629-5500, CancerResource@nortonhealthcare.org

    Michael J. Ferguson

    Recruiting

  • University of Wisconsin Carbone Cancer Center - University Hospital

    Madison, Wisconsin, 53792

    Site Public Contact, 800-622-8922, clinicaltrials@cancer.wisc.edu

    Christian M. Capitini

    Recruiting

  • Saint Jude Children's Research Hospital

    Memphis, Tennessee, 38105

    Active, not recruiting

  • Nicklaus Children's Hospital

    Miami, Florida, 33155

    Site Public Contact, 888-624-2778

    Ossama M. Maher

    Recruiting

  • The Children's Hospital at TriStar Centennial

    Nashville, Tennessee, 37203

    Site Public Contact, 615-342-1919

    Clinton M. Carroll

    Recruiting

  • Vanderbilt University/Ingram Cancer Center

    Nashville, Tennessee, 37232

    Site Public Contact, 800-811-8480

    Carrie L. Kitko

    Recruiting

  • Children's Hospital New Orleans

    New Orleans, Louisiana, 70118

    Site Public Contact, 504-894-5377

    Maria C. Velez-Yanguas

    Recruiting

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609

    Site Public Contact, 510-428-3264, PedOncRschOAK@ucsf.edu

    Jennifer G. Michlitsch

    Recruiting

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, 73104

    Site Public Contact, 405-271-8777, ou-clinical-trials@ouhsc.edu

    Rene Y. McNall-Knapp

    Recruiting

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

    Site Public Contact, 267-425-5544, CancerTrials@email.chop.edu

    Caitlin W. Elgarten

    Recruiting

  • VCU Massey Comprehensive Cancer Center

    Richmond, Virginia, 23298

    Site Public Contact, 804-628-6430, CTOclinops@vcu.edu

    Elizabeth Krieger

    Recruiting

  • Primary Children's Hospital

    Salt Lake City, Utah, 84113

    Site Public Contact, 801-585-5270

    Soohee Cho

    Recruiting

  • Methodist Children's Hospital of South Texas

    San Antonio, Texas, 78229

    Site Public Contact, 210-575-6240, Vinod.GidvaniDiaz@hcahealthcare.com

    Jose M. Esquilin

    Recruiting

  • UCSF Medical Center-Mission Bay

    San Francisco, California, 94158

    Site Public Contact, 877-827-3222, cancertrials@ucsf.edu

    Lena E. Winestone

    Recruiting

  • Washington University School of Medicine

    St Louis, Missouri, 63110

    Site Public Contact, 800-600-3606, info@siteman.wustl.edu

    Shalini Shenoy

    Recruiting

  • New York Medical College

    Valhalla, New York, 10595

    Site Public Contact, 914-594-3794

    Andrew J. Bellantoni

    Recruiting

  • Alfred I duPont Hospital for Children

    Wilmington, Delaware, 19803

    Site Public Contact, 302-651-5572, Allison.bruce@nemours.org

    Sridhi Patel

    Recruiting

Other trials in United States

About this study

Official title: Letermovir Prophylaxis for Cytomegalovirus in Pediatric Hematopoietic Cell Transplantation

This phase III single arm trial determines whether taking prophylactic letermovir will reduce the likelihood of infection with cytomegalovirus (CMV) in children and adolescents after stem cell transplant compared to estimated rate of infection without prophylaxis. The treatments used to prepare for hematopoietic cell transplant (HCT) reduce the body's natural infection-fighting ability and increase the likelihood of an infection with a virus called cytomegalovirus. "Prophylaxis" means to take a drug to prevent a disease or side effect. Letermovir is an antiviral drug that stops cytomegalovirus from multiplying and may prevent cytomegalovirus infection and make the disease less severe.

Read the detailed description

PRIMARY OBJECTIVE:

  1. To evaluate the efficacy of letermovir prophylaxis in the prevention of clinically significant CMV infection through Week 14 (~100 days) post-transplant in children and adolescents receiving allogeneic hematopoietic cell transplant (allo-HCT).

SECONDARY OBJECTIVE:

  1. To evaluate the efficacy of letermovir prophylaxis as assessed by CMV-free survival through 24 weeks (~6 months) post-transplant in pediatric patients.

EXPLORATORY OBJECTIVES:

  1. To evaluate the incidence of clinically significant CMV infection through 24 and 52 weeks post-transplant in patients who receive letermovir prophylaxis.

II. To evaluate overall survival post-transplant in patients who receive letermovir prophylaxis.

III. To evaluate time to engraftment and describe the cumulative incidence of non-engraftment among patients who receive letermovir.

IV. To examine the following clinically significant adverse events among patients exposed to letermovir: the total duration of neutropenia through week 14 (~100 days) post-transplant, the cumulative incidence of acute kidney injury and chronic kidney disease by 52 weeks post-transplant, and total inpatient hospital days by 14 weeks (~100 days) and 52 weeks post-transplant.

  1. Describe patterns of anti-viral resistance at the onset of CMV DNAemia after allo-HCT among patients who receive letermovir prophylaxis.

VI. To describe immune reconstitution and CMV-specific immunity among patients who receive letermovir prophylaxis.

OUTLINE: Enrolled patients will be added to the single arm of the study and receive letermovir prophylaxis.

ARM A: Patients receive letermovir orally (PO) or intravenously (IV) over 60 minutes once daily (QD) starting on day +1 post-transplant for 14 weeks. Patients undergo collection of blood samples for CMV polymerase chain reaction (PCR) analysis weekly for 14 weeks, every 2 weeks until week 24, week 32, week 40 and week 52.

ARM B (CLOSED TO ACCRUAL 09/29/2025): Patients undergo collection of blood samples for CMV PCR analysis weekly for 14 weeks, every 2 weeks until week 24, week 32, week 40 and week 52.

Being tested
Biospecimen Collection, Letermovir
Medical subject headings
Hematologic Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection

    Undergo collection of blood samples

  • LetermovirDrug

    Also called 2-((4S)-8-Fluoro-2-(4-(3-methoxyphenyl)piperazin-1-yl)-3-(2-methoxy-5-(trifluoromethyl)phenyl)-4H-quinazolin-4-yl)acetic Acid, AIC246, MK-8228, Prevymis

    Given PO or IV

Study groups in NCT05711667
Study groupRoleWhat they receive
ARM A (Letermovir prophylaxis)ExperimentalPatients receive letermovir PO or IV over 60 minutes QD starting on day +1 post-transplant for 14 weeks. Patients undergo collection of blood samples for CMV PCR analysis weekly for 14 weeks, every 2 weeks until week 24, week 32, week 40 and week 52.
Receives: Procedure: Biospecimen Collection; Drug: Letermovir
Arm B (No prophylaxis)Active comparatorPatients undergo collection of blood samples for CMV PCR analysis weekly for 14 weeks, every 2 weeks until week 24, week 32, week 40 and week 52. (CLOSED TO ACCURAL 09/29/2025)
Receives: Procedure: Biospecimen Collection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Clinically significant cytomegalovirus (CMV) infection

    Clinically significant CMV is defined as the first of (1) initiation of anti-CMV preemptive therapy for documented CMV DNAemia or (2) onset of CMV end-organ disease. Will estimate the 14 week cause-specific cumulative incidence of clinically significant CMV under the non-parametric, Aalen-Johansen estimator of the cumulative incidence function. Will estimate the 95% upper 1-sided confidence limit of the cause-specific cumulative incidence at 100-days post-hematopoietic cell transplantation using a bias-corrected and accelerated bootstrap-based confidence interval.

    Measured: Up to week 14 post-transplant

Secondary outcomes (2)

  1. Detection of CMV DNAemia

    Will estimate the cumulative incidence of CMV DNAemia at 24-weeks post-transplant by study arm and will report the corresponding 95% confidence intervals.

    Measured: Up to week 24 post-transplant

  2. CMV-free survival

    Will estimate the cumulative incidence of the occurrence of CMV DNAemia or death by week 24 post-transplant by study arm and will report the corresponding 95% confidence intervals.

    Measured: Up to 24 weeks post-transplant

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Feb 2023
  2. Study start11 Jul 2024
  3. Primary completion30 Jun 2029 (estimated)
  4. Study completion30 Jun 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Prevention
Allocation
Randomised
Intervention model
Parallel groups
Masking
Single blind
Who is masked
Outcomes assessor
Enrolment
105 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05711667
Sponsor's protocol ID
ACCL1932
Other IDs
NCI-2022-10769, ACCL1932, COG-ACCL1932, ACCL1932, U24CA196173, UG1CA189955
Status last verified
Jul 2026
First submitted
23 Jan 2023
Last updated
6 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Biospecimen Collection and Letermovir in people with Hematopoietic and Lymphoid Cell Neoplasm and Malignant Solid Neoplasm, run by Children's Oncology Group at 33 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 6 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 33 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Clinically significant cytomegalovirus (CMV) infection (time frame: Up to week 14 post-transplant). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05711667.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Oct 2026.

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