NCT04254419

Phase 1 Study of Locoregional Injections of Ex Vivo Expanded Natural Killer Cells

Phase 1 Study of Locoregional Injections of Ex Vivo Expanded Natural Killer Cells in Children and Young Adults With Recurrent, Progressive, or Refractory Brain Tumors

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: Nationwide Children's Hospital · Academic / Other
• Sex: All
• Age: 12 Months – 39 Years
• Healthy volunteers: Not accepted

Summary

Each patient will receive up to 12 cycles of TGFβi NK cell infusions. Each cycle will be of 4 weeks duration. During the first 3 weeks, TGFβi NK cells will be infused once weekly. The 4th week will be a rest week. TGFβi NK cell infusions should be delivered at least 3 days apart (e.g., Friday of Week 1 and Monday of Week 2). Dose will be escalated in an inter-patient stepwise fashion consisting of 3 dose levels.

Detailed description

A cycle is 28 days (4 weeks) consisting of weekly infusions of UD TGFβi NK cells via Ommaya or a programable ventriculoperitoneal (VP) shunt for three weeks followed by one week of rest. The dose-limiting toxicity (DLT) period is the first 28 days (4 weeks). A DLT is defined as any event that is at least possibly attributable to the TGFβi NK cell product and that occurs from the time of initial NK cell infusion through the end of the first cycle (28 days). Patients with stable or improved disease will receive up to 12 cycles of TGFβi NK cell infusions.

Conditions

• High Grade Glioma

Interventions

Type	Name	Description
BIOLOGICAL	NK cells	The universal donor TGFβi NK cells will be cryopreserved until they are delivered bedside for infusion. The trained staff will thaw the product by the bedside. The administration of the cells will be done via an Ommaya intra-cavitary/a programable ventriculoperitoneal (VP) shunt. Once the infusion is ready for administration patients will be admitted to the infusion unit for monitoring. NK cells will be administered through the Ommaya/VP shunt in approximately 3 milliliters over approximately 2-5 minutes; followed by 1.5-2 milliliter preservative-free normal saline flush over approximately 1 minute.

Timeline

• Start date: 2025-03-04
• Primary completion: 2030-12-31
• Completion: 2031-10-01
• First posted: 2020-02-05
• Last updated: 2025-05-13

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study