Allogeneic Stem Cell TransplantPhase 1/2VSTsNCT02048332
Donor-Derived Viral Specific T-cells (VSTs)
A Phase 1/2 study testing Viral specific VST Infusion in people with Allogeneic Stem Cell Transplant and Viral Infection, run by Children's Hospital Medical Center, Cincinnati at 3 sites in United States.
Trial summary
In this research study, the investigators want to learn more about the use of donor-derived viral specific T-cells (VSTs) to treat viral infections that occur after allogeneic stem cell transplant. Read more about this study
- Who is it for?
- Ages 4 weeks and older
- Conditions the study is for
- Allogeneic Stem Cell Transplant, Viral Infection, Viral Reactivation
- What is being tested?
- Viral specific VST Infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1,000 participants
- Where
- 3 sitesUnited States
- Study dates
- 5 Feb 2014to Jan 2027
- Phase
- Phase 1/2
- 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 4 weeks and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Recipient must be at least 21 days after stem cell infusion
- Clinical status must allow tapering of steroids to 0.5mg/kg prednisone or other steroid equivalent
- Recipient must have achieved engraftment with ANC ≥ 500
Who cannot join
- Active acute GVHD grades II-IV
- Uncontrolled bacterial or fungal infection
- Uncontrolled relapse of malignancy requiring treatment with chemotherapy
- Infusion of ATG or alemtuzumab within 2 weeks of VST infusion
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 2 recruiting
- Recruiting
Akron Children's Hospital
Akron, Ohio, 44308
Courtney Culbertson, CNP, 330-543-3338, cculbertson@akronchildrens.org
Megan Sampson, MD
- Completed
University of Cincinnati Medical Center
Cincinnati, Ohio, 45219
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Jamie Wilhelm, 513-803-1102, Jamie.Wilhelm@cchmc.org
Michael Grimley, MD
About this study
Official title: Donor-Derived Viral Specific T-cells (VSTs) for Treatment of Viral Infections After Allogeneic Stem Cell Transplant
In this research study, the investigators want to learn more about the use of donor-derived viral specific T-cells (VSTs) to treat viral infections that occur after allogeneic stem cell transplant. A viral specific T cell is a T lymphocyte (a type of white blood cell) that kills cells that are infected (particularly with viruses). Allogeneic means the stem cells come from another person. These VSTs are cells specially designed to fight the virus infections that can happen after a bone marrow transplant.
The investigators are asking people who have undergone or will undergo an allogeneic stem cell transplant to enroll in this research study, because viral infections are a common problem after allogeneic stem cell transplant and can cause significant complications including death.
Stem cell transplant reduces a person's ability to fight infections. There is an increased risk of getting new viral infections or reactivation of viral infections that the patient has had in the past, such as cytomegalovirus (CMV), Epstein-Barr virus (EBV), adenovirus (ADV), BK virus (BKV), and JC virus. There are anti-viral medicines available to treat these infections, though not all patients will respond to the standard treatments. Moreover, treatment of viral infections is expensive and time consuming, with families often administering prolonged treatments with intravenous anti-viral medications, or patients requiring prolonged admissions to the hospital. The medicines can also have side effects like damage to the kidneys or reduction in the blood counts, so in this study the investigators are trying to find an easier way to treat these infections.
Read the detailed description
The stem cell matched donor will be asked to provide a blood donation for the VSTs generation. In the laboratory, the investigators will treat this blood sample to select out the cells that will help fight viruses. The cells will be grown with peptides (protein fragments that represent parts of the virus that will encourage the donor immune cells to grow). The cells will be grown in the laboratory so that there is a stock of virus fighting cells for the patient to use in the future. The investigators will freeze the cells and store them in a freezer in the laboratory.
If the patient has signs of virus in their blood after the transplant, they will be given the cells to help fight the infection. If there are signs that the cells are helping fight the infection, more cells may be given. The patient may get the cells up to 5 times, with 21 days between each treatment (this timeframe may be shortened to 14 days for patients with no evidence of viral response). If the patient does not show signs of a virus, the cells will stay in the freezer.
Following VST infusion, (s)he will be monitored with physical exams daily while inpatient and weekly while outpatient as well as blood tests weekly until 30 days after the last infusion of cells. The patient will have 3 teaspoons (15 mL) of blood drawn and urine collected before each cell infusion and then once a week after each infusion for 4 weeks and then once a month if possible for 1 year after the last infusion, all to monitor for the viral response.
- Being tested
- Viral specific VST Infusion
- Keywords
- Epstein-Barr Virus (EBV), Adenovirus (ADV), t-cells, donor, transplant, children, cytomegalovirus (CMV), BK virus (BKV)
- Medical subject headings
- Virus Diseases, Epstein-Barr Virus Infections, Adenoviridae Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Viral specific VST InfusionBiological
VSTs will be infused into stem cell transplant recipients who have evidence of viral infection or reactivation defined as any of the below:
- Blood adenovirus PCR ≥ 1,000
- Blood CMV PCR ≥ 500
- Blood EBV PCR ≥ 9,000
- Plasma BKV PCR >1,000
- Plasma JC Virus PCR >1,000
- Evidence of invasive adenovirus infection or disease, defined as the presence of adenoviral positivity in one or more sites.
- Evidence of invasive CMV infection, eg pneumonitis, retinitis, colitis
- Evidence of invasive EBV disease/infection, EBV-associated lymphoproliferation (EBV-LPD) defined as proven EBV-LPD by biopsy or probable EBV-LPD defined as an elevated EBV DNA level in the blood associated with clinical symptoms (adenopathy or fever or masses on imaging) but without biopsy confirmation, or EBV-associated malignancies.
- Evidence of symptomatic BK virus infection, which may include symptomatic hemorrhagic cystitis, or BK nephropathy.
- Evidence of PML or other CNS infection due to JC virus.
| Study group | Role | What they receive |
|---|---|---|
| Viral Specific VST Infusion | Experimental | Viral reactivation or infection. VST Reinfusion required. Receives: Biological: Viral specific VST Infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Successful production of viral specific T-cells
Of the patients who had a VST culture initiated, successful production of VST cells is defined as meeting the protocol-defined release criteria.
Measured: Within 30 days post culture initiation
Percentage of patients who do not have infusional toxicity
Patients will be monitored for infusional toxicity
Measured: Through 30 minutes post infusion
Incidence of GVHD associated with VST infusion
Patients will be monitored for the development of VST associated GVHD
Measured: Through 30 days after infusion
Secondary outcomes (2)
Presence of viral-specific T-cells
Presence of viral-specific T-cells in the participant's blood will be assessed by Elispot assay
Measured: At 30 days after infusion
Viral burden
The viral burden will be assessed using the protocol-defined efficacy assessment
Measured: At 30 days after infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2014 (estimated)
- Study start5 Feb 2014
- Primary completionJan 2027 (estimated)
- Study completionJan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Allogeneic Stem Cell TransplantStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Hoxworth Blood Center
- Responsible party
- Sponsor
- Investigators
- Michael Grimley, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02048332
- Sponsor's protocol ID
- 2013-2777
- Acronym
- VSTs
- Status last verified
- Sep 2026
- First submitted
- 27 Jan 2014
- Last updated
- 4 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Galletta TJ, Lane A, Lutzko C, Leemhuis T, Cancelas JA, Khoury R, Wang YM, Hanley PJ, Keller MD, Bollard CM, Davies SM, Grimley MS, Rubinstein JD. Third-Party and Patient-Specific Donor-Derived Virus-Specific T Cells Demonstrate Similar Efficacy and Safety for Management of Viral Infections after Hematopoietic Stem Cell Transplantation in Children and Young Adults. Transplant Cell Ther. 2023 May;29(5):305-310. doi: 10.1016/j.jtct.2023.01.027. Epub 2023 Feb 3.
PubMed 36736781Rubinstein JD, Zhu X, Leemhuis T, Pham G, Ray L, Emberesh S, Jodele S, Thomas S, Cancelas JA, Bollard CM, Hanley PJ, Keller MD, Grimley O, Clark D, Clark T, Lindestam Arlehamn CS, Sette A, Davies SM, Nelson AS, Grimley MS, Lutzko C. Virus-specific T cells for adenovirus infection after stem cell transplantation are highly effective and class II HLA restricted. Blood Adv. 2021 Sep 14;5(17):3309-3321. doi: 10.1182/bloodadvances.2021004456.
PubMed 34473237
Study at a glance
From the registry record, last updated by the sponsor on 4 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Donor-Derived Viral Specific T-cells (VSTs) for Treatment of Viral Infections After Allogeneic Stem Cell Transplant
- ClinicalTrials.gov ID
- NCT02048332
- Acronym / sponsor ID
- VSTs / 2013-2777
- Keywords
- Epstein-Barr Virus (EBV), Adenovirus (ADV), t-cells, donor, transplant, children, cytomegalovirus (CMV), BK virus (BKV)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- Hoxworth Blood Center
- Study start
- 5 Feb 2014 (actual)
- Primary completion
- Jan 2027 (estimated)
- Study completion
- Jan 2027 (estimated)
- First posted
- 29 Jan 2014 (estimated)
- 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.
In this research study, the investigators want to learn more about the use of donor-derived viral specific T-cells (VSTs) to treat viral infections that occur after allogeneic stem cell transplant. A viral specific T cell is a T lymphocyte (a type of white blood cell) that kills cells that are infected (particularly with viruses). Allogeneic means the stem cells come from another person. These VSTs are cells specially designed to fight the virus infections that can happen after a bone marrow transplant.
The investigators are asking people who have undergone or will undergo an allogeneic stem cell transplant to enroll in this research study, because viral infections are a common problem after allogeneic stem cell transplant and can cause significant complications including death.
Stem cell transplant reduces a person's ability to fight infections. There is an increased risk of getting new viral infections or reactivation of viral infections that the patient has had in the past, such as cytomegalovirus (CMV), Epstein-Barr virus (EBV), adenovirus (ADV), BK virus (BKV), and JC virus. There are anti-viral medicines available to treat these infections, though not all patients will respond to the standard treatments. Moreover, treatment of viral infections is expensive and time consuming, with families often administering prolonged treatments with intravenous anti-viral medications, or patients requiring prolonged admissions to the hospital. The medicines can also have side effects like damage to the kidneys or reduction in the blood counts, so in this study the investigators are trying to find an easier way to treat these infections.
Detailed description
The stem cell matched donor will be asked to provide a blood donation for the VSTs generation. In the laboratory, the investigators will treat this blood sample to select out the cells that will help fight viruses. The cells will be grown with peptides (protein fragments that represent parts of the virus that will encourage the donor immune cells to grow). The cells will be grown in the laboratory so that there is a stock of virus fighting cells for the patient to use in the future. The investigators will freeze the cells and store them in a freezer in the laboratory.
If the patient has signs of virus in their blood after the transplant, they will be given the cells to help fight the infection. If there are signs that the cells are helping fight the infection, more cells may be given. The patient may get the cells up to 5 times, with 21 days between each treatment (this timeframe may be shortened to 14 days for patients with no evidence of viral response). If the patient does not show signs of a virus, the cells will stay in the freezer.
Following VST infusion, (s)he will be monitored with physical exams daily while inpatient and weekly while outpatient as well as blood tests weekly until 30 days after the last infusion of cells. The patient will have 3 teaspoons (15 mL) of blood drawn and urine collected before each cell infusion and then once a week after each infusion for 4 weeks and then once a month if possible for 1 year after the last infusion, all to monitor for the viral response.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Viral Specific VST Infusion | Experimental | Biological: Viral specific VST InfusionViral reactivation or infection. VST Reinfusion required. |
Who may qualify
- Ages
- 4 weeks and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Recipient must be at least 21 days after stem cell infusion
- Clinical status must allow tapering of steroids to 0.5mg/kg prednisone or other steroid equivalent
- Recipient must have achieved engraftment with ANC ≥ 500
Key exclusion criteria (4 listed)
- Active acute GVHD grades II-IV
- Uncontrolled bacterial or fungal infection
- Uncontrolled relapse of malignancy requiring treatment with chemotherapy
- Infusion of ATG or alemtuzumab within 2 weeks of VST infusion
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 |
|---|---|---|
| Viral specific VST InfusionVSTs will be infused into stem cell transplant recipients who have evidence of viral infection or reactivation defined as any of the below: * Blood adenovirus PCR ≥ 1,000 * Blood CMV PCR ≥ 500 * Blood EBV PCR ≥ 9,000 * Plasma BKV PCR >1,000 * Plasma JC Virus PCR >1,000 * Evidence of invasive adenovirus infection or disease, defined as the presence of adenoviral positivity in one or more sites. * Evidence of invasive CMV infection, eg pneumonitis, retinitis, colitis * Evidence of invasive EBV disease/infection, EBV-associated lymphoproliferation (EBV-LPD) defined as proven EBV-LPD by biopsy or probable EBV-LPD defined as an elevated EBV DNA level in the blood associated with clinical symptoms (adenopathy or fever or masses on imaging) but without biopsy confirmation, or EBV-associated malignancies. * Evidence of symptomatic BK virus infection, which may include symptomatic hemorrhagic cystitis, or BK nephropathy. * Evidence of PML or other CNS infection due to JC virus. | Biological | Viral Specific VST Infusion |
Primary outcome
Successful production of viral specific T-cells
Time frame: Within 30 days post culture initiation
Of the patients who had a VST culture initiated, successful production of VST cells is defined as meeting the protocol-defined release criteria.
Percentage of patients who do not have infusional toxicity
Time frame: Through 30 minutes post infusion
Patients will be monitored for infusional toxicity
Incidence of GVHD associated with VST infusion
Time frame: Through 30 days after infusion
Patients will be monitored for the development of VST associated GVHD
2 secondary outcomes
Presence of viral-specific T-cells
Time frame: At 30 days after infusion
Viral burden
Time frame: At 30 days after infusion
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 4 Sep 2026.
- Derived from this study: PubMed 36736781Galletta TJ, Lane A, Lutzko C, Leemhuis T, Cancelas JA, Khoury R, Wang YM, Hanley PJ, Keller MD, Bollard CM, Davies SM, Grimley MS, Rubinstein JD. Third-Party and Patient-Specific Donor-Derived Virus-Specific T Cells Demonstrate Similar Efficacy and Safety for Management of Viral Infections after Hematopoietic Stem Cell Transplantation in Children and Young Adults. Transplant Cell Ther. 2023 May;29(5):305-310. doi: 10.1016/j.jtct.2023.01.027. Epub 2023 Feb 3.
- Derived from this study: PubMed 34473237Rubinstein JD, Zhu X, Leemhuis T, Pham G, Ray L, Emberesh S, Jodele S, Thomas S, Cancelas JA, Bollard CM, Hanley PJ, Keller MD, Grimley O, Clark D, Clark T, Lindestam Arlehamn CS, Sette A, Davies SM, Nelson AS, Grimley MS, Lutzko C. Virus-specific T cells for adenovirus infection after stem cell transplantation are highly effective and class II HLA restricted. Blood Adv. 2021 Sep 14;5(17):3309-3321. doi: 10.1182/bloodadvances.2021004456.
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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.
Michael Grimley, MD principal investigator
Children's Hospital Medical Center, Cincinnati
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Viral specific VST Infusion in people with Allogeneic Stem Cell Transplant and Viral Infection, run by Children's Hospital Medical Center, Cincinnati at 3 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 3 inclusion and 4 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 4 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 Jamie Wilhelm, BS ((513) 803-1102, Jamie.Wilhelm@cchmc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Successful production of viral specific T-cells (time frame: Within 30 days post culture initiation). The study lists 3 primary outcomes in all. 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 NCT02048332, which also lists 2 linked publications.
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 4 Sep 2026.