NCT04239092

9-ING-41 in Pediatric Patients With Refractory Malignancies.

Phase 1 Study of 9-ING-41, a Glycogen Synthase Kinase 3 Beta (GSK 3β) Inhibitor, as a Single Agent or With Irinotecan, Irinotecan Plus Temozolomide, or With Cyclophosphamide Plus Topotecan in Pediatric Patients With Refractory Malignancies.

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 40 (actual)
• Sponsor: Actuate Therapeutics Inc. · Industry
• Sex: All
• Age: 22 Years
• Healthy volunteers: Not accepted

Summary

9-ING-41 has anti-cancer clinical activity with no significant toxicity in adult patients. This Phase 1 study will study its efficacy in paediatric patients with advanced malignancies.

Detailed description

9-ING-41 is a first-in-class, intravenously administered, maleimide-based, small molecule, potent selective GSK-3β inhibitor with significant pre-clinical and clinical anticancer activity. In the ongoing Actuate 1801 study in a cohort of over 90 patients with advanced refractory malignancies, 9-ING-41 has exhibited no significant toxicity, including no myelosuppression, and significant anti-tumor activity. 9-ING-41 also has significant pre-clinical ability to reverse pathologic fibrosis in multiple models of pulmonary and pleural fibrosis. 9-ING-41 is very highly active against neuroblastoma in diverse pre-clinical models. This Phase 1 study is designed to evaluate the safety and efficacy of 9-ING-41, as a single agent or in combination with irinitecan, in paediatric patients with advanced malignancies and thus to establish the recommended Phase 2 dose (RP2D) for further paediatric patient studies.

Conditions

• Refractory Cancer
• Refractory Neoplasm
• Cancer Pediatric
• Refractory Tumor
• Pediatric Cancer
• Pediatric Brain Tumor
• Neuroblastoma
• Neuroblastoma Recurrent
• Pediatric Lymphoma
• Pediatric Meningioma
• Diffuse Intrinsic Pontine Glioma

Interventions

Type	Name	Description
DRUG	9-ING-41	9-ING-41 will be administered by intravenous infusion twice weekly at an initial dose of 9.3 mg/kg. Cycle duration is 21 days.
DRUG	Irinotecan	Irinotecan 50 mg/m2/day administered over 90 minutes IV on days 1-5 every 21 days.
DRUG	Temozolomide	Temozolomide will be administered at a dose of 100 mg/m2/dose by mouth on Days 1 through 5 of a 21 day cycle.
DRUG	Cyclophosphamide	Cyclophosphamide 400 mg/m2/dose administered intravenously over 30 min on Days 1 through 5 of a 21 day cycle.
DRUG	Topotecan	Topotecan 1.2 mg/m2/dose administered intravenously over 30 min once on Days 1 through 5 of a 21 day cycle.

Timeline

• Start date: 2020-06-05
• Primary completion: 2025-07-07
• Completion: 2025-07-07
• First posted: 2020-01-23
• Last updated: 2025-07-17

Locations

9 sites across 1 country: United States

Regulatory

• FDA-regulated drug study