Safety and Efficacy Study of Safusidenib in Participants With IDH1-Mutant Glioma Who Discontinued Vorasidenib Treatment Due to Progressive Disease

The film gives the main points. The full list below is copied from the registry; the study team makes the final decision.

• Age18 Years to no maximum
• SexAll
• Healthy volunteersNo

You may be able to join if

• Key Inclusion Criteria:
• Histologically confirmed Grade 2 or 3 IDH-mutant astrocytoma or IDH-mutant and 1p19q co-deleted oligodendroglioma, according to WHO CNS 2021 criteria
• IDH1 mutation (e.g., R132H/C/G/S/L) based on immunohistochemistry (IHC) (R132H only), PCR, or next generation sequencing (NGS).
• Have had at least 1 prior surgery for glioma (biopsy, sub-total resection, or gross total resection).
• Measurable disease per modified RANO 2.0 confirmed by BICR during Screening.
• Previously treated with vorasidenib and experienced radiographic disease progression during treatment and are not in need of immediate chemotherapy or radiotherapy in the opinion of the Investigator. Disease progression must be confirmed by BICR using modified RANO 2.0.
• Adequate hematologic and organ function
• Expected survival of ≥12 months
• Key Exclusion Criteria:
• Any prior anticancer therapy other than surgery (biopsy, sub-total, or gross total resection) and vorasidenib for treatment of glioma.
• Discontinued vorasidenib for toxicity or any reason other than radiographic disease progression
• Prior treatment with anti-angiogenic agents such as Avastin (bevacizumab) or investigational agents for glioma.
• Brainstem or spinal cord involvement either as primary location, site of multifocal involvement, or by significant tumor extension.
• Significant functional or neurocognitive deficits, including uncontrolled seizures
• Evidence of leptomeningeal disease.
• Use of therapeutic doses of steroids for signs/symptoms of glioma. Participants taking physiologic doses (defined as equivalent of <1.5 mg of dexamethasone equivalent) for medical conditions not related to glioma will be permitted.

This is how the treatment is meant to work. Whether it helps, and how safe it is, is what this trial is testing.

safusidenib. An orally available inhibitor of isocitrate dehydrogenase type 1 (IDH1; IDH-1; IDH1 [NADP+] soluble) mutant forms, including substitution mutations at the arginine in position 132, IDH1(R132) (IDH1-R132), with potential antineoplastic activity. Upon oral administration, safusidenib specifically binds to and inhibits certain mutant forms of IDH1, thereby inhibiting the formation of the oncometabolite 2-hydroxyglutarate (2HG) from alpha-ketoglutarate (a-KG). This prevents 2HG-mediated signaling and leads to both an induction of cellular differentiation and an inhibition of cellular proliferation in tumor cells expressing IDH1 mutations. IDH1(R132) mutations are highly expressed in certain malignancies, including gliomas; they initiate and drive cancer growth by both blocking cell differentiation and catalyzing the formation of 2HG. Safusidenib minimally targets and affects wild-type IDH1, which is expressed in normal, healthy cells. NCI Drug Dictionary

Treatments listed in the registry

• Safusidenib (Drug): Safusidenib administered twice daily as a single agent orally on Days 1 to 28 of a 28-day cycle. Participants may continue treatment until disease progression or another reason for discontinuation occurs.

Na · Single Group · masking: None

Main goals

• Objective Response Rate (ORR) per modified Response Assessment in Neuro-Oncology (RANO) 2.0 assessed by Blinded Independent Central Review (BICR) (From the date of first dose of study drug until the date of first documented disease progression, approximately 18 months)

Other goals

• ORR per modified RANO 2.0 assessed by Investigator
• Volumetric Tumor Growth Rate (TGR) by BICR
• Duration of Response (DOR) per modified RANO 2.0 assessed by BICR and by Investigator
• Time to Next Intervention (TTNI)
• Progression Free Survival (PFS) per modified RANO 2.0 assessed by BICR and by Investigator
• Time to Response (TTR) per modified RANO 2.0 assessed by BICR and by Investigator
• Overall Survival (OS)
• Safety and tolerability
• Seizure Activity

Planned enrollment: 40 (estimated).

• NYU Langone Health, New York, New York

Site status as of Oct 30, 2026. Check the live record before contacting a site.

• ClinicalTrials.gov NCT07703436
• NCI Drug Dictionary: C148134

Registry record reviewed as fetched Oct 30, 2026, last updated by the sponsor Oct 19, 2026. Public record; not medical advice.