Pediatric Brain TumorPhase 1PNOC028NCT05887882
Intra-Tumoral Injections of Natural Killer Cells for Recurrent Malignant Pediatric Brain Tumors
A Phase 1 study testing Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) Cells, Implantation and Magnetic Resonance Imaging (MRI) in people with Pediatric Brain Tumor and Recurrent Pediatric Brain Tumor, run by Sabine Mueller, MD, PhD at 10 sites in United States.
Trial summary
This phase I trial tests the safety, side effects, and best dose of ex vivo expanded natural killer cells in treating patients with cancerous (malignant) tumors affecting the upper part of the brain (supratentorial) that have come back (recurrent) or that are growing, spreading, or getting worse (progressive). Read more about this study
- Who is it for?
- Ages 1 year to 38 years
- Conditions the study is for
- Pediatric Brain Tumor, Recurrent Pediatric Brain Tumor, Pediatric Neoplasm
- What is being tested?
- Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) Cells, Implantation and Magnetic Resonance Imaging (MRI)
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 10 sitesUnited States
- Study dates
- 31 May 2024to 31 Dec 2027
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 1 year to 38 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participants must have a histologically-confirmed recurrent or progressive malignant brain tumor including, but not limited to, infant-type hemispheric glioma, gliosarcoma, intracranial sarcoma and WHO Grade II ependymoma.
- Participants should be candidates for resection of the recurrent tumor and be deemed candidate for placement of an Ommaya reservoir placed intra-cavitary/intra-tumoral; measurable residual tumor after surgery is not required for study entry. Pre-operative imaging needs to estimate that the resection cavity will be at least 2 cm x 2 cm in two dimensions for participants to be eligible.
- Given the lack of a standard of care treatment for children with recurrent or progressive malignant brain tumors, participants must have completed first-line treatment with radiation and/or chemotherapy prior to participating in this trial if applicable.
- All participants must be ≥ 1 year of age and ≤ 39 years of age at the time of entry into the study. The first 3 participants must be ≥ 8 years of age and ≤ 39 years of age at the time of entry into the study.
- Performance Score: Karnofsky ≥ 50 for participants > 16 years of age and Lansky ≥ 50 for participants ≤ 16 years of age. Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
- Must have recovered from the acute toxic effects of prior therapy (i.e., NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5, grade 1 or less)
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- An interval of at least 12 weeks must have elapsed since the completion of radiation therapy.
- Chemotherapy/biologic therapy: All cytotoxic chemotherapy/biologic therapy must be discontinued ≥ 7 days prior to enrollment (except 3 weeks for temozolomide and 6 weeks from last dose of nitrosoureas)
- Immunotherapy: The last dose of anti-tumor antibody therapy must be at least 3 half-lives or 30 days, whichever is shorter, from the time of enrollment
- For targeted agents only, participants should have recovered from any toxicity of the agent and have a minimum of 2 weeks since the last dose.
- For participants who have received prior bevacizumab, at least 4 weeks is required.
- Organ Function Requirements:
- Adequate Bone Marrow Function Defined as:
- Peripheral absolute neutrophil count (ANC) >750/mm^3.
- Platelet count >75,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to registration).
- Adequate Renal Function Defined as:
- A serum creatinine ≤ 1.5 x upper limit normal (ULN) based on age/gender.
- Adequate Liver Function Defined as:
- Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age; in presence of Gilbert's syndrome, total bilirubin ≤ 3 x ULN or direct bilirubin ≤ 1.5 x ULN.
- Alanine aminotransferase (ALT) ≤ 3 x ULN.
- Aspartate aminotransferase (AST) ≤ 3 x ULN.
- Adequate Neurologic Function Defined as:
- Participants with seizure disorder may be enrolled if seizures are well-controlled. Participants on non-enzyme inducing anticonvulsants may be excluded pending interaction(s) with study drug.
- Signs and symptoms of neurologic deficit must be stable for ≥ 1 week prior to registration.
- The effects of Transforming growth factor beta resistant (TGFβi) natural killer (NK) cells on the developing human fetus are unknown. For this reason and because TGFβi NK cells as well as other therapeutic agents used in this trial are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation and 6 months after completion of TGFβi NK cells administration. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
- Participants must enroll on PNOC COMP if PNOC COMP is open to accrual at the enrolling institution
- A legal parent/guardian or patient must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate.
- Corticosteroids: Participants who are receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to registration. The patient steroid dose should be no more than a steroid-equivalent of dexamethasone 0.1 mg/kg/day (or maximum 4 mg/day; whichever is the lower dose) at time of enrollment.
Who cannot join
- Tumor involvement that would require ventricular or brainstem injection or access through a ventricle or significant risk of ventricular penetration in order to deliver the TGFβi NK cells.
- Participants undergoing needle or open biopsy.
- Participants who are receiving any other investigational agents.
- Women of childbearing potential must not be pregnant or breast-feeding.
- Evidence of active uncontrolled infection or unstable or severe intercurrent medical conditions.
- Any medical condition that precludes surgery.
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- Prothrombin time/international normalized ratio (PT/INR) or partial thromboplastin time (PTT) > 1.5 x ULN.
- Participants with a known disorder that affects their immune system, such as human immunodeficiency virus (HIV), or an auto- immune disorder requiring systemic cytotoxic or immunosuppressive therapy are not eligible.
- Evidence of bleeding diathesis or use of anticoagulant medication or any medication which may increase the risk of bleeding. If the medication can be discontinued >1 week prior to NK cell infusion then the subject may be eligible following consultation with the Study Chairs.
- Participants with significant systemic or major illnesses including but not limited to: congestive heart failure, ischemic heart disease, kidney disease or renal failure, organ transplantation, or significant psychiatric disorder.
- History or current diagnosis of any medical or psychological condition that in the Investigator's opinion, might interfere with the participants ability to participate or inability to obtain informed consent because of psychiatric or complicating medical problems.
- Important note: The eligibility criteria listed above are interpreted literally and cannot be waived.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Johns Hopkins
Baltimore, Maryland, 21287
University of Alabama Birmingham
Birmingham, Alabama, 35294
Nationwide Children's Hospital
Columbus, Ohio, 43205
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Indiana University
Indianapolis, Indiana, 46202
Children's Hospital Los Angeles
Los Angeles, California, 90027
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of California, San Francisco
San Francisco, California, 94143
Seattle Children's
Seattle, Washington, 98105
Washington University in Saint Louis
St Louis, Missouri, 63130
About this study
Official title: A Phase I Study of Intra-Tumoral Injections of Ex Vivo Expanded Natural Killer Cells in Children and Young Adults With Recurrent or Progressive Malignant Brain Tumors
This phase I trial tests the safety, side effects, and best dose of ex vivo expanded natural killer cells in treating patients with cancerous (malignant) tumors affecting the upper part of the brain (supratentorial) that have come back (recurrent) or that are growing, spreading, or getting worse (progressive). Natural killer (NK) cells are immune cells that recognize and get rid of abnormal cells in the body, including tumor cells and cells infected by viruses. NK cells have been shown to kill different types of cancer, including brain tumors in laboratory settings. Giving NK cells from unrelated donors who are screened for optimal cell qualities and determined to be safe and healthy may be effective in treating supratentorial malignant brain tumors in children and young adults.
Read the detailed description
PRIMARY OBJECTIVES:
- To determine the safety and tolerability of natural killer (NK) cells that have been propagated ex vivo with genetically-modified feeder cells and administered intra-tumoral via an Ommaya reservoir in participants with recurrent or progressive malignant brain tumors.
II. To determine the recommended phase 2 dose (RP2D) for natural killer (NK) cells that have been propagated ex vivo with genetically-modified feeder cells and administered intra-tumoral via an Ommaya reservoir in participants with recurrent or progressive malignant brain tumors.
EXPLORATORY OBJECTIVES:
- To determine the 6 months overall survival (OS), defined as the percentage of participants in the study who are alive at 6 months following start of treatment.
II. To determine the persistence, immuno-phenotype and function of adoptively-transferred expanded NK cells and correlate the findings with the overall response.
III. To determine the immune signature-based profile of each patient's tumor. IV. To determine changes in the T-cell receptor (TCR) repertoire diversity before and after Transforming growth factor beta imprinted (TGFβi) NK cell treatment.
- To evaluate the effect of systemic steroids on the persistence and efficacy of TGFβi NK cells.
VI. To assess Quality of Life (QOL) and cognitive measures in children and young adults with recurrent or progressive malignant brain tumors.
VII. To assess patient and/or proxy satisfaction with study participation via patient-reported outcome (PRO) measures in the context of race ethnicity and other health related social risks.
VIII. To assess on therapy toxicity in the context of race, ethnicity and other health related social risks.
OUTLINE: This is a dose-escalation study.
Participants undergo placement of an Ommaya reservoir and receive universal donor expanded TGF-beta-imprinted NK cells (TGFBi NK cells) intratumorally over 5 minutes via Ommaya reservoir on day 1 of each cycle. Cycles repeat every 28 days for 3 cycles in the absence of disease progression or unacceptable toxicity. Participants also undergo magnetic resonance imaging (MRI) of the brain during screening and on study and collection of tumor-associated fluid via Ommaya reservoir on study. Participants may also undergo MRI of the spine and lumbar puncture as clinically indicated.
- Being tested
- Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) Cells, Implantation, Magnetic Resonance Imaging (MRI), Quality-of-Life Assessment
- Keywords
- Transforming growth factor beta imprinted, Natural Killer Cells
- Medical subject headings
- Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) CellsBiological
Also called UD TGFβi NK cells, UD TGFβi NK cell product
The TGFβi NK cell product will be manufactured in the Cell Therapy Laboratory at Nationwide Children's Hospital and given via infusion.
ImplantationProcedure or surgery
Undergo placement of Ommaya reservoir
Magnetic Resonance Imaging (MRI)Procedure or surgery
Also called MRI
Imaging procedure
Quality-of-Life AssessmentOther
Also called QOL Assessment
Ancillary studies
| Study group | Role | What they receive |
|---|---|---|
| Dose Level 2 (3x10^6) - Starting Dose | Experimental | Enrolled participants must proceed to surgery for tumor resection and Ommaya placement into the resection cavity within 14 days of enrollment. Starting dose of 3x10^6 of TGFβi NK cells (first dose) may be administered at least 14 days after the Ommaya reservoir placement and may not start until all acute surgical complications have resolved (maximum of 6 weeks after enrollment). TGFβi NK cell infusions through the Ommaya reservoir will occur once weekly for three weeks followed by one rest week. If participants have stable or improved disease, participants may continue to receive therapy for a total of 3 cycles. Receives: Biological: Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) Cells; Procedure: Implantation; Procedure: Magnetic Resonance Imaging (MRI); Other: Quality-of-Life Assessment |
| Dose Level 3 (3x10^7) | Experimental | If no safety or toxicity events are demonstrated by the starting dose cohort, enrolled participants in the next dosing cohort must proceed to surgery for tumor resection and Ommaya placement into the resection cavity within 14 days of enrollment. The dose of 3x10^7 of TGFβi NK cells (first dose) may be administered at least 14 days after the Ommaya reservoir placement and may not start until all acute surgical complications have resolved (maximum of 6 weeks after enrollment). TGFβi NK cell infusions through the Ommaya reservoir will occur once weekly for three weeks followed by one rest week. If participants have stable or improved disease, participants may continue to receive therapy for a total of 3 cycles. Receives: Biological: Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) Cells; Procedure: Implantation; Procedure: Magnetic Resonance Imaging (MRI); Other: Quality-of-Life Assessment |
| Dose Level 4 (3x10^8) | Experimental | If no safety or toxicity events are demonstrated by the previous dose cohort, enrolled participants in the next dosing cohort must proceed to surgery for tumor resection and Ommaya placement into the resection cavity within 14 days of enrollment. The dose of 3x10^8 of TGFβi NK cells (first dose) may be administered at least 14 days after the Ommaya reservoir placement and may not start until all acute surgical complications have resolved (maximum of 6 weeks after enrollment). TGFβi NK cell infusions through the Ommaya reservoir will occur once weekly for three weeks followed by one rest week. If participants have stable or improved disease, participants may continue to receive therapy for a total of 3 cycles. Receives: Biological: Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) Cells; Procedure: Implantation; Procedure: Magnetic Resonance Imaging (MRI); Other: Quality-of-Life Assessment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants with treatment-emergent adverse events
Adverse events and clinically significant laboratory abnormalities (meeting grade 3, 4, or 5 criteria according to NCI CTCAE) will be summarized by maximum intensity and relationship to study drug(s). Grade 1 and 2 adverse events will be summarized if related to study therapy. Descriptive statistics will be utilized to display the data on toxicity seen. Toxicities will be summarized by tabulation in terms of type, grade and attribution for each dose level of each group of patients studied at the end of the trial.
Measured: From initiation of study treatment until 30 days from the end of therapy, approximately 1 year
Recommended Phase II dose (RP2D)
RP2D is defined as the dose level at which fewer than one-third of participants experience a dose limiting toxicity (DLT)
Measured: From initiation of study treatment until 30 days from the end of therapy, approximately 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2023
- Study start31 May 2024
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Why it stopped: Enrollment on hold while reviewing a SAE.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric Brain TumorStudies 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sabine Mueller, MD, PhD
- Sponsor type
- University, hospital or other
- Collaborators
- Rally Foundation, Washington University School of Medicine, Nationwide Children's Hospital, CureSearch, Tommy Strong Foundation
- Responsible party
- Sponsor-investigator, Sabine Mueller, MD, PhD, University of California, San Francisco
- Investigators
- Sabine Mueller, MD, PhD, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05887882
- Sponsor's protocol ID
- 210831
- Other IDs
- NCI-2023-03216
- Acronym
- PNOC028
- Status last verified
- Apr 2026
- First submitted
- 24 May 2023
- Last updated
- 24 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified participant data may be shared with collaborating researchers.
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of Intra-Tumoral Injections of Ex Vivo Expanded Natural Killer Cells in Children and Young Adults With Recurrent or Progressive Malignant Brain Tumors
- ClinicalTrials.gov ID
- NCT05887882
- Acronym / sponsor ID
- PNOC028 / 210831
- Keywords
- Transforming growth factor beta imprinted, Natural Killer Cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: Enrollment on hold while reviewing a SAE.
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Sabine Mueller, MD, PhDUniversity, hospital or other
- Collaborators
- Rally Foundation, Washington University School of Medicine, Nationwide Children's Hospital, CureSearch, Tommy Strong Foundation
- Study start
- 31 May 2024 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 5 Jun 2023 (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.
This phase I trial tests the safety, side effects, and best dose of ex vivo expanded natural killer cells in treating patients with cancerous (malignant) tumors affecting the upper part of the brain (supratentorial) that have come back (recurrent) or that are growing, spreading, or getting worse (progressive). Natural killer (NK) cells are immune cells that recognize and get rid of abnormal cells in the body, including tumor cells and cells infected by viruses. NK cells have been shown to kill different types of cancer, including brain tumors in laboratory settings. Giving NK cells from unrelated donors who are screened for optimal cell qualities and determined to be safe and healthy may be effective in treating supratentorial malignant brain tumors in children and young adults.
Detailed description
PRIMARY OBJECTIVES:
- To determine the safety and tolerability of natural killer (NK) cells that have been propagated ex vivo with genetically-modified feeder cells and administered intra-tumoral via an Ommaya reservoir in participants with recurrent or progressive malignant brain tumors.
II. To determine the recommended phase 2 dose (RP2D) for natural killer (NK) cells that have been propagated ex vivo with genetically-modified feeder cells and administered intra-tumoral via an Ommaya reservoir in participants with recurrent or progressive malignant brain tumors.
EXPLORATORY OBJECTIVES:
- To determine the 6 months overall survival (OS), defined as the percentage of participants in the study who are alive at 6 months following start of treatment.
II. To determine the persistence, immuno-phenotype and function of adoptively-transferred expanded NK cells and correlate the findings with the overall response.
III. To determine the immune signature-based profile of each patient's tumor. IV. To determine changes in the T-cell receptor (TCR) repertoire diversity before and after Transforming growth factor beta imprinted (TGFβi) NK cell treatment.
- To evaluate the effect of systemic steroids on the persistence and efficacy of TGFβi NK cells.
VI. To assess Quality of Life (QOL) and cognitive measures in children and young adults with recurrent or progressive malignant brain tumors.
VII. To assess patient and/or proxy satisfaction with study participation via patient-reported outcome (PRO) measures in the context of race ethnicity and other health related social risks.
VIII. To assess on therapy toxicity in the context of race, ethnicity and other health related social risks.
OUTLINE: This is a dose-escalation study.
Participants undergo placement of an Ommaya reservoir and receive universal donor expanded TGF-beta-imprinted NK cells (TGFBi NK cells) intratumorally over 5 minutes via Ommaya reservoir on day 1 of each cycle. Cycles repeat every 28 days for 3 cycles in the absence of disease progression or unacceptable toxicity. Participants also undergo magnetic resonance imaging (MRI) of the brain during screening and on study and collection of tumor-associated fluid via Ommaya reservoir on study. Participants may also undergo MRI of the spine and lumbar puncture as clinically indicated.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dose Level 2 (3x10^6) - Starting Dose | Experimental | Biological: Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) Cells, Procedure: Implantation, Procedure: Magnetic Resonance Imaging (MRI), Other: Quality-of-Life AssessmentEnrolled participants must proceed to surgery for tumor resection and Ommaya placement into the resection cavity within 14 days of enrollment. Starting dose of 3x10^6 of TGFβi NK cells (first dose) may be administered at least 14 days after the Ommaya reservoir placement and may not start until all acute surgical complications have resolved (maximum of 6 weeks after enrollment). TGFβi NK cell infusions through the Ommaya reservoir will occur once weekly for three weeks followed by one rest week. If participants have stable or improved disease, participants may continue to receive therapy for a total of 3 cycles. |
| Dose Level 3 (3x10^7) | Experimental | Biological: Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) Cells, Procedure: Implantation, Procedure: Magnetic Resonance Imaging (MRI), Other: Quality-of-Life AssessmentIf no safety or toxicity events are demonstrated by the starting dose cohort, enrolled participants in the next dosing cohort must proceed to surgery for tumor resection and Ommaya placement into the resection cavity within 14 days of enrollment. The dose of 3x10^7 of TGFβi NK cells (first dose) may be administered at least 14 days after the Ommaya reservoir placement and may not start until all acute surgical complications have resolved (maximum of 6 weeks after enrollment). TGFβi NK cell infusions through the Ommaya reservoir will occur once weekly for three weeks followed by one rest week. If participants have stable or improved disease, participants may continue to receive therapy for a total of 3 cycles. |
| Dose Level 4 (3x10^8) | Experimental | Biological: Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) Cells, Procedure: Implantation, Procedure: Magnetic Resonance Imaging (MRI), Other: Quality-of-Life AssessmentIf no safety or toxicity events are demonstrated by the previous dose cohort, enrolled participants in the next dosing cohort must proceed to surgery for tumor resection and Ommaya placement into the resection cavity within 14 days of enrollment. The dose of 3x10^8 of TGFβi NK cells (first dose) may be administered at least 14 days after the Ommaya reservoir placement and may not start until all acute surgical complications have resolved (maximum of 6 weeks after enrollment). TGFβi NK cell infusions through the Ommaya reservoir will occur once weekly for three weeks followed by one rest week. If participants have stable or improved disease, participants may continue to receive therapy for a total of 3 cycles. |
Who may qualify
- Ages
- 1 year to 38 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (28 listed)
- Participants must have a histologically-confirmed recurrent or progressive malignant brain tumor including, but not limited to, infant-type hemispheric glioma, gliosarcoma, intracranial sarcoma and WHO Grade II ependymoma.
- Participants should be candidates for resection of the recurrent tumor and be deemed candidate for placement of an Ommaya reservoir placed intra-cavitary/intra-tumoral; measurable residual tumor after surgery is not required for study entry. Pre-operative imaging needs to estimate that the resection cavity will be at least 2 cm x 2 cm in two dimensions for participants to be eligible.
- Given the lack of a standard of care treatment for children with recurrent or progressive malignant brain tumors, participants must have completed first-line treatment with radiation and/or chemotherapy prior to participating in this trial if applicable.
- All participants must be ≥ 1 year of age and ≤ 39 years of age at the time of entry into the study. The first 3 participants must be ≥ 8 years of age and ≤ 39 years of age at the time of entry into the study.
- Performance Score: Karnofsky ≥ 50 for participants > 16 years of age and Lansky ≥ 50 for participants ≤ 16 years of age. Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
Key exclusion criteria (12 listed)
- Tumor involvement that would require ventricular or brainstem injection or access through a ventricle or significant risk of ventricular penetration in order to deliver the TGFβi NK cells.
- Participants undergoing needle or open biopsy.
- Participants who are receiving any other investigational agents.
- Women of childbearing potential must not be pregnant or breast-feeding.
- Evidence of active uncontrolled infection or unstable or severe intercurrent medical conditions.
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 |
|---|---|---|
| Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) CellsAlso: UD TGFβi NK cells, UD TGFβi NK cell productThe TGFβi NK cell product will be manufactured in the Cell Therapy Laboratory at Nationwide Children's Hospital and given via infusion. | Biological | Dose Level 2 (3x10^6) - Starting Dose, Dose Level 3 (3x10^7), Dose Level 4 (3x10^8) |
| ImplantationUndergo placement of Ommaya reservoir | Procedure or surgery | Dose Level 2 (3x10^6) - Starting Dose, Dose Level 3 (3x10^7), Dose Level 4 (3x10^8) |
| Magnetic Resonance Imaging (MRI)Also: MRIImaging procedure | Procedure or surgery | Dose Level 2 (3x10^6) - Starting Dose, Dose Level 3 (3x10^7), Dose Level 4 (3x10^8) |
| Quality-of-Life AssessmentAlso: QOL AssessmentAncillary studies | Other | Dose Level 2 (3x10^6) - Starting Dose, Dose Level 3 (3x10^7), Dose Level 4 (3x10^8) |
Primary outcome
Proportion of participants with treatment-emergent adverse events
Time frame: From initiation of study treatment until 30 days from the end of therapy, approximately 1 year
Adverse events and clinically significant laboratory abnormalities (meeting grade 3, 4, or 5 criteria according to NCI CTCAE) will be summarized by maximum intensity and relationship to study drug(s). Grade 1 and 2 adverse events will be summarized if related to study therapy. Descriptive statistics will be utilized to display the data on toxicity seen. Toxicities will be summarized by tabulation in terms of type, grade and attribution for each dose level of each group of patients studied at the end of the trial.
Recommended Phase II dose (RP2D)
Time frame: From initiation of study treatment until 30 days from the end of therapy, approximately 1 year
RP2D is defined as the dose level at which fewer than one-third of participants experience a dose limiting toxicity (DLT)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2026.
- Individual participant dataSharing planned: Yes. De-identified participant data may be shared with collaborating researchers.
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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. This study is not recruiting at the moment.
Sabine Mueller, MD, PhD principal investigator
University of California, San Francisco
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Universal Donor (UD) Transforming growth factor beta imprinting (TGFβi) Natural Killer (NK) Cells, Implantation and Magnetic Resonance Imaging (MRI) in people with Pediatric Brain Tumor and Recurrent Pediatric Brain Tumor, run by Sabine Mueller, MD, PhD at 10 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 28 inclusion and 12 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: paused, as of the sponsor's last update on 24 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of participants with treatment-emergent adverse events (time frame: From initiation of study treatment until 30 days from the end of therapy, approximately 1 year). 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 24 Apr 2026.