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

Phase 1 Completed 10 enrolled Emory University CURE Childhood Cancer, Inc. · collab Peach Bowl LegACy Fund · collab

NaSingle-groupOpen-labelTreatment

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.

Timeline

Start: 2020-05-04
Primary completion: 2023-02-03
Completion: 2023-02-03

Publications

Castellino RC, Mumme H, Franson A, Yu B, Robinson H, Dhodapkar K, Aguilera D, Schniederjan M, Keesari R, He Z, Bhasin M, Priebe W, Heimberger AB, MacDonald TJ. First-in-child phase I trial of p-STAT3 inhibitor WP1066 in pediatric brain tumor patients. JCI Insight. 2025 Dec 11;11(3):e194823. doi: 10.1172/jci.insight.194823. eCollection 2026 Feb 9.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	WP1066	Small molecule	4 mg/kg	Oral
Subject	WP1066	Small molecule	6 mg/kg	Oral
Subject	WP1066	Small molecule	8 mg/kg	Oral
Subject	WP1066	Small molecule	16 mg/kg	Oral

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

Summary

Timeline

Publications

Drugs

Indications