Skip to main content
OpenTrials
Not Yet Recruiting

NCT Number: NCT07712757

Safety and Efficacy Study of Safusidenib in Participants With Grade 2 IDH1-Mutant Glioma

This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study comparing the efficacy and safety of safusidenib versus placebo in participants with residual or recurrent Grade 2 glioma (oligodendroglioma or astrocytoma) with an IDH1 mutation who have undergone surgery as their only treatment and are not in need of immediate chemotherapy or radiotherapy.

Not Yet Recruiting

Trial opening soon.

Get Notified

Key information

Conditions

Grade 2 Glioma (Astrocytoma or Oligodendroglioma) With an IDH1 Mutation Astrocytoma Astrocytoma IDH Mutant Grade 2 Glioma IDH-mutant Gliomas Neoplasms Neoplasms by Histologic Type Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Neoplasms, Neuroepithelial Neuroectodermal Tumors Oligodendroglioma Oligodendroglioma IDH-mutant and 1p/19q-codeleted

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • Expected survival of ≥12 months.
  • At least 1 prior surgery for glioma (biopsy, sub-total resection, or gross total resection), with the most recent surgery having occurred at least 90 days (or at least 28 days if biopsy only and postoperative changes have resolved per BICR) and no longer than 5 years before the date of randomization
  • Have not had any other prior anticancer therapy, including chemotherapy and radiotherapy; and are not in need of immediate chemotherapy or radiotherapy in the opinion of the Investigator.
  • Histologically confirmed Grade 2, IDH1-mutant oligodendroglioma or astrocytoma according to World Health Organization Central Nervous System 2021 classification criteria per Investigator assessment.
  • IDH1 mutation (R132H/C/G/S/L), identified by polymerase chain reaction, next-generation sequencing (NGS), or immunohistochemistry; and confirmed 1p19q codeletion status by fluorescence in situ hybridization, NGS, or array comparative genomic hybridization.
  • Residual or recurrent, measurable, non-enhancing disease, as confirmed by BICR per RANO 2.0, assessed at Screening.
  • Adequate hematologic and organ functions

Key Exclusion Criteria:

  • Any prior anticancer therapy other than surgery (biopsy, sub-total, or gross total resection) for treatment of glioma, including chemotherapy, investigational therapies, prior therapies targeting IDH1 or IDH2, anti-angiogenic therapies, tumor-treating fields, or radiotherapy.
  • High-risk features as assessed by the Investigator, including brainstem or spinal cord involvement either as primary location or by significant tumor extension, clinically relevant functional or neurocognitive deficits due to the tumor (deficits resulting from surgery are allowed), or uncontrolled seizures (defined as persistent seizures interfering with ability to comply with protocol requirements).
  • 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.

Treatment and study plan

Safusidenib

Drug

Safusidenib administered twice daily as a single agent dosed orally on Days 1 through 28 of a 28-day cycle. Participants may continue treatment until disease progression or another reason for discontinuation occurs.

Placebo

Drug

Placebo administered twice daily as a single agent dosed orally on Days 1 through 28 of a 28-day cycle. Participants may continue treatment with placebo until disease progression or another reason for discontinuation occurs.

Primary outcomes

  1. Progression Free Survival (PFS) assessed by Blinded Independent Central Review (BICR) per modified Response Assessment in Neuro-Oncology (RANO) 2.0

    Time frame: From the date of randomization until the date of first documented disease progression, approximately 30 months

    PFS, defined as time from date of randomization to the date of the first occurrence of radiographic disease progression per modified RANO 2.0 assessed by BICR or death from any cause, whichever occurs earlier

Secondary outcomes

  1. Objective Response Rate (ORR) assessed by BICR per modified RANO 2.0

    Time frame: From the date of randomization until the date of first documented disease progression, approximately 30 months

    ORR, defined as the proportion of participants with the confirmed best overall response of Complete Response (CR), Partial Response (PR), or Minor Response (MR) per modified RANO 2.0 assessed by BICR

  2. Time to Next Intervention (TTNI)

    Time frame: From the date of randomization until the date of the start of another anticancer treatment or date of death, approximately 30 months

    TTNI, defined as the time from randomization to the initiation of first subsequent anticancer therapy or death from any cause, whichever occurs earlier.

  3. PFS assessed by the Investigator per modified RANO 2.0

    Time frame: From the date of randomization until the date of first documented disease progression, approximately 30 months

    PFS, defined as the time from date of randomization to the date of the first occurrence of radiographic disease progression per modified RANO 2.0 assessed by the Investigator or death from any cause, whichever occurs earlier

  4. ORR assessed by the Investigator per modified RANO 2.0

    Time frame: From the date of randomization until the date of the first documented disease progression, approximately 30 months

    ORR, defined as the proportion of participants with the confirmed best overall response of CR, PR, and MR per modified RANO 2.0 as assessed by the investigator.

  5. Duration of Response (DOR) assessed by BICR and by the Investigator per modified RANO 2.0

    Time frame: From the date of randomization until the date of first documented disease progression, approximately 30 months

    DOR, the time from the first documentation of objective response (CR, PR, or MR) to the date of the first occurrence of radiographic disease progression per modified RANO 2.0 or death from any cause, whichever occurs earlier, as assessed by BICR and by the Investigator.

  6. Time to Response (TTR) assessed by BICR and by the Investigator per modified RANO 2.0

    Time frame: From randomization to the first documentation of objective response (CR, PR, or MR), approximately 30 months

    TTR, defined as the time from randomization to the first documentation of objective response (CR, PR, or MR) per modified RANO 2.0 assessed by BICR and by the Investigator.

  7. Disease Control Rate (DCR) assessed by BICR and by the Investigator per modified RANO 2.0

    Time frame: From the date of randomization until the date of first documented disease progression, approximately 30 months

    DCR, defined as the proportion of participants with a best overall response of CR, PR, MR, or stable disease (SD) per modified RANO 2.0, as assessed by BICR and by the Investigator

  8. Tumor Growth Rate (TGR) by volume assessed by BICR

    Time frame: From historical scans through the final scan in the study, approximately 30 months

    TGR defined as the percentage change in tumor volume by unit of time, assessed by BICR

  9. Overall Survival (OS)

    Time frame: From the date of randomization until the date of death, approximately 30 months

    OS, defined as the time from randomization to death from any cause

  10. Safety and tolerability

    Time frame: From the first dose of study drug until 30 days after treatment discontinuation, approximately 30 months

    The safety and tolerability of safusidenib compared with placebo evaluated based on AEs graded by NCI-CTCAE version 5.0, laboratory abnormalities as graded by NCI-CTCAE version 5.0, vital signs, physical examinations, and ECGs

  11. Safusidenib PK Profile

    Time frame: From the first dose of study drug through approximately 16 weeks

    Characterize the safusidenib concentrations

  12. Health-Related Quality of Life

    Time frame: From the first dose of study drug to treatment discontinuation, approximately 30 months

    Evaluate health-related quality of life (HRQoL) with safusidenib compared with placebo assessed by the Functional Assessment of Cancer Therapy-Brain (FACT-Br) questionnaire scores.

  13. Health-Related Quality of Life

    Time frame: From the first dose of study drug to treatment discontinuation, approximately 30 months

    Evaluate health-related quality of life (HRQoL) with safusidenib compared with placebo assessed by the Quality of Life in Epilepsy (QOLIE-10-P) scores.

  14. Health-Related Quality of Life

    Time frame: From the first dose of study drug to treatment discontinuation, approximately 30 months

    Evaluate health-related quality of life (HRQoL) with safusidenib compared with placebo assessed by the EuroQol 5-Dimension 5-Level (EQ-5D-5L) questionnaire scores.

  15. Seizure Activity

    Time frame: From the date of randomization until the date of first documented disease progression, approximately 30 months

    Evaluate seizure frequencies and severity including type of seizure, seizure-related AEs, and changes in anti-epileptic medications in participants receiving safusidenib compared with placebo

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Trials at Nuvation Bio

CONTACT

[email protected]

332-208-6102

Sponsors and collaborators

Lead sponsor

Nuvation Bio Inc.

Industry

Registry information

Official study title

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Safusidenib in Participants With Grade 2 Isocitrate Dehydrogenase 1 (IDH1)-Mutant Glioma

Important dates

Study start
2027
Primary completion
2029
Study completion
2032
First posted
Jul 17, 2026
Registry last updated
Jul 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.