CMV CTLs in Neonates With CMV Infection

NCT05564598 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: NYMC 5971R01FD007837-01
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 4

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

Outcome Measures

Primary Outcomes (2)

• To determine the safety of giving CMV CTLs combined with anti-viral therapy in neonates with CMV

  the incidence and severity of Grade I-IV acute GVHD within 8 weeks that is probably or directly related to CMV-CTL infusion after last CMV CTL infusion will be evaluated to determine the safety profile of CMV CTLs in neonates

  12 weeks

• To determine response rates to treatment with CMV CTLS and anti-viral medication

  response rates will be measured by monitoring CMV PCR levels. A complete response to CMV-CTLs will be those with undetectable viral load by qRT-PCR

  12 weeks

Study Arms (3)

Cohort 1 Safety Run-in

EXPERIMENTAL

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.

Biological: CMV Cytotoxic T-Lymphocytes; Drug: Anti-viral Therapy

Cohort 2 Antiviral medication + CMV CTLs

EXPERIMENTAL

Patients will receive both anti-viral medication and CMV CTLs

Biological: CMV Cytotoxic T-Lymphocytes; Drug: Anti-viral Therapy

Cohort 2 Antiviral medication only

ACTIVE COMPARATOR

Patients will only receive anti-viral therapy

Drug: Anti-viral Therapy

Interventions

CMV Cytotoxic T-Lymphocytes BIOLOGICAL

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.

Also known as: CTLs

Cohort 1 Safety Run-in; Cohort 2 Antiviral medication + CMV CTLs

Anti-viral Therapy DRUG

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

Also known as: valganciclovir, ganciclovir

Cohort 1 Safety Run-in; Cohort 2 Antiviral medication + CMV CTLs; Cohort 2 Antiviral medication only

Eligibility Criteria

• Age: 0 Days - 21 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• New York Medical College: lead

Study Sites (4)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

Washington University

St Louis, Missouri, 63130, United States

RECRUITING

New York Medical College

Valhalla, New York, 10595, United States

RECRUITING

Nationwide Children's Hosptial

Columbus, Ohio, 43205, United States

RECRUITING

MeSH Terms

Conditions

Disease

Interventions

Valganciclovir; Ganciclovir

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Acyclovir; Guanine; Hypoxanthines; Purinones; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Mitchell Cairo, MD

  New York Medical College

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mitchell Cairo, MD

CONTACT

914-594-2150; mitchell_cairo@nymc.edu

Edo Schaefer, MD

CONTACT

eshaefe@nymc.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 26, 2022
• First Posted: October 3, 2022
• Study Start: July 1, 2023
• Primary Completion (Estimated): October 31, 2027
• Study Completion (Estimated): October 31, 2028
• Last Updated: March 30, 2026

Record last verified: 2026-03

Locations

US (4)