Congenital Cytomegaloviral (CMV) DiseasePhase 2NCT05564598
CMV CTLs in Neonates With CMV Infection
A Phase 2 study testing CMV Cytotoxic T-Lymphocytes and Anti-viral Therapy in people with Congenital Cytomegaloviral (CMV) Disease, run by New York Medical College at 4 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 to 21 days
- Conditions the study is for
- Congenital Cytomegaloviral (CMV) Disease
- What is being tested?
- CMV Cytotoxic T-Lymphocytes and Anti-viral Therapy
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 4 sitesUnited States
- Study dates
- 1 Jul 2023to 31 Oct 2028
- Phase
- Phase 2
- 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 0 to 21 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age: ≤ 21 days of life
- Birth Weight: ≥ 2500 gms. . For babies with intrauterine growth retardation (IUGR) (small for gestational age [SGA]) > 1500 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
Show all 25Show fewer
- 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.
Who cannot join
- 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.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Edo Schaefer, MD contact
United States4 sites, 4 recruiting
- Recruiting
Nationwide Children's Hosptial
Columbus, Ohio, 43205
Masako Shimamura, MD, masako.shimamura@nationwidechildrens.org
- Recruiting
Children's Hospital Los Angeles
Los Angeles, California, 90027
Ashley Gray, MD, agray@chla.usc.edu
Phillippe Friedlich, MD, pfriedlich@chla.usc.edu
- Recruiting
Washington University
St Louis, Missouri, 63130
Carol Kao, MD, kaoc@wustl.edu
- Recruiting
New York Medical College
Vallhala, New York, 10595
Mitchell Cairo, MD, 914-594-3650, mitchell_cairo@nymc.edu
Lauren Harrison, MSN, 617-285-7844, lauren_harrison@nymc.edu
About this study
Official title: A Phase II Open-Label Randomized Study of Anti-Viral Antibiotic Therapy With and Without Familial (Maternal) Cytomegalovirus (CMV) Cytotoxic T Lymphocytes (CTLs) in Neonates With Moderate/Severe Maternal Acquired CMV Infection
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. There is a cross-over design for patient assigned to SOC only if they have persistent disease.
Funding source: FDA OOPD
Read the detailed description
Given the vulnerability and poor outcomes of preterm neonates and neonates in general to viral infection, including the need for prolonged antiviral therapy for 6 or more months to achieve just modest improvements in sensorineural functions, CMV CTL therapy offers a promising alternative. CMV CTL treatment will build on the hosts innate immune capacity to create a more effective and permanent defense against collateral injury arising from CMV infections.
Patients who meet all inclusion/exclusion criteria with a maternal donor who meet all donor criteria will be enrolled onto study.
Cohort 1 is a safety run-in; the first 3 patients enrolled will be treated with anti-viral and CMV CTLs. The external DSMB will review the data from the first patient, and if there are no adverse events or dose-limiting toxicities observed, approve patient 2, and then 3, 28 days after the prior patients last CTL infusion. Assuming there are no adverse events in any of the first 3 patients, the study will proceed to Cohort 2.
Cohort 2 will be randomized 1:1 to either anti-viral treatment alone or anti-viral treatment plus CMV CTLs.
Patients who are randomized to receive CMV CTLs will get their first infusion on Day 0. If the patient fails to achieve a CR, they may receive one infusion every 2 weeks up to 5 maximum CMV CTL infusions as long as there are no DLTs or AEs observed
Patients who are assigned the SOC are only may be able to cross-over to to receive CMV CTLs if they have persistent disease at day 28.
- Conditions
- Congenital Cytomegaloviral (CMV) Disease
- Being tested
- CMV Cytotoxic T-Lymphocytes, Anti-viral Therapy
- Medical subject headings
- Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CMV Cytotoxic T-LymphocytesBiological
Also called CTLs
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 TherapyDrug
Also called valganciclovir, ganciclovir
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
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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 Receives: Biological: CMV Cytotoxic T-Lymphocytes; Drug: Anti-viral Therapy |
| Cohort 2 Antiviral medication only | Active comparator | Patients will only receive anti-viral therapy Receives: Drug: Anti-viral Therapy |
| Cross-Over | Experimental | Patients assigned to the SOC may be eligible to cross-over to the CVM CTL treatment if they are not responding to SOC treatment with continued elevated CMV levels. Receives: Biological: CMV Cytotoxic T-Lymphocytes; Drug: Anti-viral Therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2022
- Study start1 Jul 2023
- Primary completion31 Oct 2027 (estimated)
- Study completion31 Oct 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Cytomegaloviral (CMV) DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 23 participants (estimated)
Sponsor and investigators
- Lead sponsor
- New York Medical College
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mitchell Cairo, New York Medical College
- Investigators
- Mitchell Cairo, MD, Principal investigator, New York Medical College
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05564598
- Sponsor's protocol ID
- NYMC 597
- Other IDs
- 1R01FD007837-01
- Status last verified
- Oct 2026
- First submitted
- 26 Sep 2022
- Last updated
- 28 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Open-Label Randomized Study of Anti-Viral Antibiotic Therapy With and Without Familial (Maternal) Cytomegalovirus (CMV) Cytotoxic T Lymphocytes (CTLs) in Neonates With Moderate/Severe Maternal Acquired CMV Infection
- ClinicalTrials.gov ID
- NCT05564598
- Acronym / sponsor ID
- NYMC 597
- Conditions
- Congenital Cytomegaloviral (CMV) Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 23 participants (estimated)
- Lead sponsor
- New York Medical CollegeUniversity, hospital or other
- Study start
- 1 Jul 2023 (actual)
- Primary completion
- 31 Oct 2027 (estimated)
- Study completion
- 31 Oct 2028 (estimated)
- First posted
- 3 Oct 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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. There is a cross-over design for patient assigned to SOC only if they have persistent disease.
Funding source: FDA OOPD
Detailed description
Given the vulnerability and poor outcomes of preterm neonates and neonates in general to viral infection, including the need for prolonged antiviral therapy for 6 or more months to achieve just modest improvements in sensorineural functions, CMV CTL therapy offers a promising alternative. CMV CTL treatment will build on the hosts innate immune capacity to create a more effective and permanent defense against collateral injury arising from CMV infections.
Patients who meet all inclusion/exclusion criteria with a maternal donor who meet all donor criteria will be enrolled onto study.
Cohort 1 is a safety run-in; the first 3 patients enrolled will be treated with anti-viral and CMV CTLs. The external DSMB will review the data from the first patient, and if there are no adverse events or dose-limiting toxicities observed, approve patient 2, and then 3, 28 days after the prior patients last CTL infusion. Assuming there are no adverse events in any of the first 3 patients, the study will proceed to Cohort 2.
Cohort 2 will be randomized 1:1 to either anti-viral treatment alone or anti-viral treatment plus CMV CTLs.
Patients who are randomized to receive CMV CTLs will get their first infusion on Day 0. If the patient fails to achieve a CR, they may receive one infusion every 2 weeks up to 5 maximum CMV CTL infusions as long as there are no DLTs or AEs observed
Patients who are assigned the SOC are only may be able to cross-over to to receive CMV CTLs if they have persistent disease at day 28.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 Safety Run-in | Experimental | Biological: CMV Cytotoxic T-Lymphocytes, Drug: Anti-viral TherapyThe 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. |
| Cohort 2 Antiviral medication + CMV CTLs | Experimental | Biological: CMV Cytotoxic T-Lymphocytes, Drug: Anti-viral TherapyPatients will receive both anti-viral medication and CMV CTLs |
| Cohort 2 Antiviral medication only | Active comparator | Drug: Anti-viral TherapyPatients will only receive anti-viral therapy |
| Cross-Over | Experimental | Biological: CMV Cytotoxic T-Lymphocytes, Drug: Anti-viral TherapyPatients assigned to the SOC may be eligible to cross-over to the CVM CTL treatment if they are not responding to SOC treatment with continued elevated CMV levels. |
Who may qualify
- Ages
- 0 to 21 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (25 listed)
- Age: ≤ 21 days of life
- Birth Weight: ≥ 2500 gms. . For babies with intrauterine growth retardation (IUGR) (small for gestational age [SGA]) > 1500 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
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CMV Cytotoxic T-LymphocytesAlso: CTLsPatients 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. | Biological | Cohort 1 Safety Run-in, Cohort 2 Antiviral medication + CMV CTLs, Cross-Over |
| Anti-viral TherapyAlso: valganciclovir, ganciclovirAll 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 | Drug | Cohort 1 Safety Run-in, Cohort 2 Antiviral medication + CMV CTLs, Cohort 2 Antiviral medication only, Cross-Over |
Primary outcome
To determine the safety of giving CMV CTLs combined with anti-viral therapy in neonates with CMV
Time frame: 12 weeks
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
To determine response rates to treatment with CMV CTLS and anti-viral medication
Time frame: 12 weeks
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2026.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Edo Schaefer, MD contact
Mitchell Cairo, MD principal investigator
New York Medical College
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CMV Cytotoxic T-Lymphocytes and Anti-viral Therapy in people with Congenital Cytomegaloviral (CMV) Disease, run by New York Medical College at 4 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 25 inclusion and 5 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 28 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 4 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Contact Mitchell Cairo, MD (914-594-2150, mitchell_cairo@nymc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To determine the safety of giving CMV CTLs combined with anti-viral therapy in neonates with CMV (time frame: 12 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 28 Oct 2026.