NCT04334863

WP1066 in Children With Refractory and Progressive or Recurrent Malignant Brain Tumors

A Phase I Study of WP1066 in Children With Refractory and Progressive or Recurrent Malignant Brain Tumors (AflacST1901)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Emory University · Academic / Other
• Sex: All
• Age: 3 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

In this Phase I clinical study, the investigators plan to offer investigational treatment with the novel JAK2/STAT3 inhibitor WP1066 (Moleculin Biotech, Inc.) to pediatric patients with any progressive or recurrent malignant brain tumor that is refractory to standard treatment and is without known cure.

Detailed description

The goal of this clinical research study is to find the highest tolerable dose of WP1066 that can be given to pediatric patients with recurrent (has returned after treatment) cancerous brain tumors or melanoma that has gotten worse and spread to the brain. The safety of this drug will also be studied. WP1066 is designed to target the STAT3 pathway in cancer cells, which makes these cells divide, increases new blood vessels to the tumor, causes the cancer cells to move throughout the body and brain, and avoids them being detected by the immune system. Targeting this pathway may cause the immune system to kill the cancer cells. The investigators will administer five escalating doses of WP1066, starting at lowest dose currently found to be safe and tolerable in adults. WP1066 is not FDA approved or commercially available. It is currently being used for research purposes only. Up to 36 participants will be enrolled in this study. All will take part at Children's Healthcare of Atlanta (CHOA).

Conditions

• Brain Tumor
• Medulloblastoma
• Brain Metastases

Interventions

Type	Name	Description
DRUG	WP1066	WP1066 is an analogue of caffeic acid that is a potent inhibitor of p-STAT3. It will be taken by mouth 2 times per day on Monday, Wednesday, and Friday of Weeks 1 and 2 of each 28-day cycle.

Timeline

• Start date: 2020-05-04
• Primary completion: 2023-02-03
• Completion: 2023-02-03
• First posted: 2020-04-06
• Last updated: 2023-02-21

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study