Recruiting
Phase 2

Valganciclovir with CMV CTLs

Sponsor:

New York Medical College

Code:

NCT05564598

Conditions

Congenital Cytomegaloviral (CMV) Disease

Eligibility Criteria

Sex: All

Age: 0

Healthy Volunteers: Not accepted

Interventions

CMV Cytotoxic T-Lymphocytes

Anti-viral Therapy

Study Details

Brief summary:

Patients with moderate or severe CMV disease less than 21 days old who have a maternal donor who has a CMV response to the peptivators will be screened.

All patients will receive treatment with valganciclovir or ganciclovir. There is a safety run in with treatment with CMV CTLs in cohort 1 and if found to be safe, will proceed to cohort 2 for randomization to receive antiviral therapy with or without CMV CTLs.

Funding source: FDA OOPD

Conditions

Congenital Cytomegaloviral (CMV) Disease

Study ID

NCT05564598

Start date

Jul 1, 2023

Status verified date

Mar, 2026

Completion date

Oct 31, 2028

Anticipated

Primary completion date

Oct 31, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Age: ≤ 21 days of life
  • Birth Weight: ≥ 2500 gms
  • Gestational age: ≥ 34 weeks of age
  • Diagnosis of CMV viremia, viruria, and/or infection:Either one or more:

Elevated CMV by RT-PCR in urine, saliva, CSF, or plasma; and/or Positive urine culture for CMV

\- Moderate or Severe CMV Disease

Any one or more of the following attributable to congenital CMV infection:

  • Thrombocytopenia (≤ 50,000 mm3)
  • Multiple petechiae
  • Hepatomegaly
  • Splenomegaly
  • Intrauterine growth retardation
  • Increased transaminases
  • Increased bilirubin
  • Microcephaly
  • Ventriculomegaly
  • Intracerebral calcifications
  • Periventricular echogenicity
  • Cortical or cerebral malformation
  • Chorioretinitis
  • Severe neonatal hearing loss
  • CMV DNA by PCR in CNS
  • Increased WBC for age in CNS

  • Minimal Organ Criteria Hematological: ANC ≥ 750/mm3, HgB ≥ 8gm/dl, Platelets ≥ 20,000/kmm3 Renal: Serum creatinine ≤ 1.0 mg/dl Hepatic: ALT/SGOT ≤3x upper normal limits
  • Donor Availability: Maternal donor available with a T-cell response CMV MACS® PepTivators. the donor is considered suitable if the percentage of IFN-gamma+ T cells is > 0.01% after stimulation with PepTivators.

Exclusion Criteria -

  • Patient receiving steroids (> 0.5 mg/kg prednisone equivalent) on the same day of CMV CTL infusion. Antenatal steroids for lung maturation will have been cleared prior to CMV diagnosis.
  • Concomitant enrollment in another experimental clinical trial investigating the treatment of neonatal CMV viremia and/or infection.
  • Any medical condition that could compromise participation in the study according to the investigator's assessment.
  • Known history of HIV infection in the mother.
  • Patient's legally authorized representative unwilling or unable to comply with the protocol or unable to give informed consent.

Study Design

Enrollment

23 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1 Safety Run-in

The first 3 patients enrolled will receive both anti-viral medication and CMV CTLs, and treatment will be staggered every 28 days from the last dose of CMV CTLs from the prior patient.

experimental: Cohort 2 Antiviral medication + CMV CTLs

Patients will receive both anti-viral medication and CMV CTLs

active comparator: Cohort 2 Antiviral medication only

Patients will only receive anti-viral therapy

Interventions

CMV Cytotoxic T-Lymphocytes

Patients will receive maternal CMV CTLs on day 0. Additional doses of CMV CTLs may be re-infused at a minimum of every two weeks for a maximum of five total infusions (maximum 2.5 x 104 CD3/kg) only in patients not achieving a CR and no prior dose limiting toxicity of any prior dose.

Anti-viral Therapy

All patients will receive anti-viral therapy with one of the following:

4.2.2 Valganciclovir Dosing: 16 mg/kg/dose PO q12h OR 4.2.3 Ganciclovir Dosing: 6 mg/kg/dose IV q12h

Dose adjustments:

  • Reduce dose by 50% for ANC less than 500 cells/mm3
  • Hold the dose if ≤ 200 cells/mm3 until recovery ≥ 500 cells/mm3
  • Treatment will continue for 6 months

Primary outcome measure

  • To determine the safety of giving CMV CTLs combined with anti-viral therapy in neonates with CMV [ Time Frame: 12 weeks ]
  • To determine response rates to treatment with CMV CTLS and anti-viral medication [ Time Frame: 12 weeks ]

Central Contacts and Locations

Central contacts

Mitchell Cairo, MD

914-594-2150mitchell_cairo@nymc.edu

Edo Schaefer, MD

eshaefe@nymc.edu

Locations

Children's Hospital Los Angeles

Recruiting

Los Angeles, California, United States, 90027

Contacts

Neena Kapoor, MD

nkapoor@chla.usc.edu

Phillippe Friedlich, MD

pfriedlich@chla.usc.edu

Washington University

Recruiting

St Louis, Missouri, United States, 63130

Contacts

Carol Kao, MD

kaoc@wustl.edu

New York Medical College

Recruiting

Valhalla, New York, United States, 10595

Contacts

Mitchell Cairo, MD

914-594-3650mitchell_cairo@nymc.edu

Lauren Harrison, MSN

617-285-7844lauren_harrison@nymc.edu

Nationwide Children's Hosptial

Recruiting

Columbus, Ohio, United States, 43205

Contacts

Masako Shimamura, MD

masako.shimamura@nationwidechildrens.org

More Information

Sponsor

New York Medical College

Last update posted

Mar 30, 2026

Last verified

Mar, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by New York Medical College on 2026-03-30.