NCT03811886

Natalizumab in Recurrent, Refractory or Progressive Pulmonary Metastatic Osteosarcoma

A Phase I/II Study of Natalizumab as a Single Agent in Children, Adolescents and Young Adults With Recurrent, Refractory or Progressive Pulmonary Metastatic Osteosarcoma

• Status: Withdrawn
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Case Comprehensive Cancer Center · Academic / Other
• Sex: All
• Age: 5 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and tolerability of Natalizumab in children, adolescent and young adult patients with pulmonary metastatic osteosarcoma (pOS) and to assess clinical response associated with this treatment as well as overall survival.

Detailed description

This study is a single-arm, open-label, proof of concept clinical trial in children, adolescent and young adult patients with unresectable pOS that have progressed, relapsed or are refractory to standard systemic therapy. All participants will receive the study therapy and there will be dose escalation in a traditional 3 + 3 design during the Phase I study of this trial. In the Phase II study of the trial, treatment will continue if the subject has Complete Response (CR), Partial Response (PR) or Stable Disease (SD) of pOS after every 3 cycles after the first 6 cycles but not beyond 24 cycles, unless it is judged to be in his/her best interest. Approximately 3-9 subjects will be enrolled in the phase I part and 10-12 in the phase II part of this trial. Participants will be followed for toxicity for 30 days after treatment has been discontinued or until one of the protocol-defined reasons This study seeks to evaluate if Natalizumab can be used safely and effectively as immunotherapy in children, adolescent and young adult patients with pOS. Natalizumab is currently Food and Drug Administration (FDA) approved for the treatment of T-cell mediated autoimmune disorders The study team will evaluate the safety and tolerability of Natalizumab as well as the clinical response associated with Natalizumab treatment and evaluate overall survival.

Conditions

• Pulmonary Metastatic Osteosarcoma (pOS)

Interventions

Type	Name	Description
DRUG	Natalizumab	Natalizumab is an FDA approved monotherapy for treatment of relapsing forms of MS. It is also indicated for inducing and maintaining clinical response and remission in adult patients with moderately to severely active CD who have had an inadequate response to, or intolerance of, conventional therapies and TNF-α inhibitors. Traditional 3+3 escalation of Natalizumab at a weight-based dosing 2mg/kg not to exceed 300mg. If no subjects experience a dose limiting toxicity (DLT), 3 more subjects are enrolled at the next dose of 3mg/kg, not to exceed 300mg. If no subjects experience a DLT, 3 more subjects will be enrolled at the next and final dose of 4mg/kg, not to exceed 300mg. Phase II will continue if the subject has Complete Response (CR), Partial Response (PR) or Stable Disease (SD) of pOS, defined by RECIST 1.1 criteria after every 3 cycles after the first 6 cycles but not beyond 24 cycles. If subject has progressive disease after cycle 6, they will be removed from the study.

Timeline

• Start date: 2024-12-01
• Primary completion: 2025-12-01
• Completion: 2026-10-01
• First posted: 2019-01-22
• Last updated: 2025-01-15

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study