A Phase 2, multicenter, clinical study to evaluate the efficacy and safety of safusidenib erbumine in patients with isocitrate dehydrogenase 1 [IDH1]-mutant glioma (NCT05303519)

This is a 3-part study. The purpose of Part 1 of the study is to evaluate the efficacy, safety, and pharmacokinetic (PK) characteristics of safusidenib in participants with recurrent/progressive IDH1-mutant World Health Organization (WHO) Grade 2 or Grade 3 glioma. Part 1 has been complete. The purpose of Part 2 will be to evaluate the efficacy of maintenance safusidenib treatment versus placebo in IDH1-mutant Grade 2 or Grade 3 astrocytoma with high-risk features or IDH1-mutant Grade 4 astrocytoma, following standard-of-care radiation or chemoradiation and adjuvant temozolomide. Part 2 will be randomized, double-blind, and placebo-controlled. The purpose of Part 3 will be to evaluate the efficacy of safusidenib in participants with residual or recurrent IDH1-mutant Grade 3 oligodendroglioma who have received surgery as their only treatment. Part 3 will be an open-label single-arm cohort and will enroll participants concurrently with Part 2.

Trial phase

Phase 3

Trial participation type

This trial has been designed for the Clinical Research of a Drug.

Principal investigator

Dr Lucy Gately

Key inclusion data

Expected survival of ≥6 months. Diagnosis of histologically confirmed IDH1-mutant Grade 3 or Grade 4 astrocytoma, per WHO 2021 classification and Investigator Assessment. Have received a resection and/or biopsy; additional resections are only allowed for reasons not relating to disease recurrence or progression (e.g. removal of residual tumor). Participants may not have received any anticancer treatments other than surgery, radiation, concurrent/adjuvant temozolomide, and tumor-treating fields. Tumor-treating fields must be discontinued prior to randomization. Participants with other malignancies must have received curative treatment and been disease-free for at least 3 years. Curatively resected non-melanoma skin cancer or curatively treated carcinoma in situ is allowed.