NCT06607991

Blinatumomab for CNI-Resistant/Intolerant SRNS in Children

Study of Blinatumomab for the Treatment of Calcineurin Inhibitor-Resistant or Intolerant Steriod-Resistant Nephrotic Syndrome in Pediatric Patients

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: The Children's Hospital of Zhejiang University School of Medicine · Academic / Other
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

This exploratory clinical trial aims to evaluate the efficacy and safety of Blinatumomab in treating children with calcineurin inhibitor (CNI)-resistant or multidrug-resistant steroid-resistant nephrotic syndrome (SRNS). Eligible participants include pediatric patients aged 2 to 17 years who have either failed to respond to adequate CNI therapy or are resistant to at least two classes of immunosuppressants, including CNIs and biologics. A short course of low-dose Blinatumomab will be administered in an open-label, single-arm, self-controlled trial design. The study seeks to determine whether Blinatumomab can reduce proteinuria and induce clinical remission in this difficult-to-treat population, offering a potential new therapeutic option for children with limited response to conventional therapies.

Detailed description

Nephrotic syndrome (NS) in children is characterized by excessive proteinuria, hypoalbuminemia, hyperlipidemia, and edema. Approximately 15-20% of pediatric NS cases are classified as steroid-resistant nephrotic syndrome (SRNS), a condition associated with poor prognosis and limited response to standard therapies. Calcineurin inhibitors (CNIs) are frequently used as first-line immunosuppressants in SRNS; however, a subset of patients demonstrate resistance to or intolerance of CNIs. Moreover, a proportion of patients may be refractory to multiple classes of immunosuppressive agents, including biologics, posing a significant therapeutic challenge. This exploratory, single-center, open-label clinical trial is designed to evaluate the safety and efficacy of Blinatumomab-a bispecific T-cell engager targeting CD19-positive B cells-in pediatric patients with CNI-resistant or multidrug-resistant SRNS. Eligible participants will include children aged 2 to 17 years who have either not responded to adequate CNI therapy or have failed to achieve remission despite treatment with at least two classes of immunosuppressive agents (including CNIs and biologics). A total of 6 patients will be enrolled and administered two short courses of low-dose Blinatumomab intravenously, each lasting 5 days. The primary efficacy outcome will be the rate of complete or partial remission of proteinuria. Secondary outcomes will include safety and tolerability assessments, changes in immunologic markers, and renal function monitoring. By selectively depleting CD19-positive B cells, Blinatumomab may modulate aberrant immune activation that underlies treatment-resistant SRNS. This study seeks to generate preliminary data on the potential therapeutic role of Blinatumomab in this difficult-to-treat pediatric population, with the ultimate goal of identifying a novel immunomodulatory approach for SRNS patients with limited treatment options.

Conditions

• CNI-resistant Steriod Resistant Nephrotic Syndrome
• CNI-intolerent
• Steriod Resistant Nephrotic Syndrome
• Multidrug Resistant Nephrotic Syndrome

Interventions

Type	Name	Description
DRUG	Blinatumomab Treatment	Blinatumomab treatment for CNI-resistant/Intolerant pediatric steriod-resistant nephrotic syndrome Patients will receive two 5-day cycles of Blinatumomab (5 µg/m²/day, maximum dose 9 µg/day), administered intravenously. The second cycle will begin on the first day of the third week following the first cycle.

Timeline

• Start date: 2024-09-19
• Primary completion: 2026-09-19
• Completion: 2027-09-18
• First posted: 2024-09-23
• Last updated: 2025-08-06

Locations

1 site across 1 country: China

Regulatory

• FDA-regulated drug study