New hope for brain tumor patients: targeted drug shows promise in phase 3 trial

NCT ID NCT05303519

Disease control Sponsor: Nuvation Bio Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 28, 2026 · Updated 3 times

Summary

This study tests a drug called safusidenib in people with a type of brain tumor (glioma) that has a specific gene mutation (IDH1). The trial has three parts: one for recurrent tumors, one for high-risk tumors after standard treatment, and one for a specific tumor type after surgery alone. The goal is to see if the drug can control the tumor and improve survival.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 365 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2023

Expected to finish

Dec 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria for Part 1: 1. Patient must be ≥ 18 years of age at the time of signing the informed consent form (ICF). 2. Patient must have histologically confirmed recurrent or progressive WHO Grade 2 glioma or Grade 3 glioma with IDH1 R132H or R132C mutation confirmed by immunohistochemistry or molecular genetic testing. 3. The IDH mutation, and other applicable gene/molecular alterations (see Table 10-2) are determined by a validated assay as performed in Clinical Laboratory Improvement Amendments (CLIA)-certified/College of American Pathologists (CAP)-accredited or locally equivalent clinical laboratories. Prior clinical pathology report fulfilling the diagnosis criteria prior to screening with tumor samples collected is acceptable for patient enrollment in both Part 1 and Part 2. 4. Patient has received no more than 2 prior therapies for disease recurrence/progression. 5. Patient has disease recurrence or progression or cannot tolerate the most recent therapy. 6. Patient must have a measurable lesion(s) as per the RANO-HGG criteria for primarily enhancing lesions or RANO-LGG criteria for primarily non-enhancing lesions. The lesion (s) must be visible on 2 or more axial slices and have perpendicular diameters of at least 10 × 10 mm. The definition of primarily enhancing lesions or primarily non-enhancing lesions is referred to Section 8.3.1. Key Inclusion Criteria for Part 2 and 3: 1. Must be ≥18 years old at the time of signing the ICF. 2. Must agree to submit sufficient tumor tissue for retrospective biomarker and histological analyses. This requirement may be waived in rare circumstances with approval by the Sponsor. 3. Has adequate hematologic and organ function Key Inclusion Criteria for Part 2: 1. Diagnosis of histologically confirmed IDH1-mutant Grade 2, Grade 3 with high risk features or Grade 4 astrocytoma, per WHO 2021 classification and Investigator Assessment. 2. Have an IDH1 mutation (R132H/C/G/S/L) based on IHC (R132H only), polymerase chain reaction (PCR), or next-generation sequencing (NGS). CDKN2A/B status and at least 1 of the following must be confirmed: absence of 1p19q co-deletion by fluorescence in situ hybridization, array comparative genomic hybridization, or NGS; presence of an ATRX loss of function mutation by NGS; or loss of normal ATRX expression by IHC. A validated assay performed in a CLIA-certified/CAP-accredited (or local equivalent) clinical laboratory must be used for all of the aforementioned results. Documentation of biomarker status, including redacted molecular pathology and NGS reports, must be provided during Screening. 3. Must not have experienced tumor recurrence or progression between first day of radiotherapy and randomization by local assessment per RANO 2.0. 4. Participants must have completed radiation therapy with a minimum of 80% of planned treatment completed (with or without concurrent temozolomide) and between 6 and 12 cycles of adjuvant . Randomization must occur at least 28 days and not more than 75 days after the final dose of temozolomide. Key Inclusion Criteria for Part 3: 1. Have had 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 and no longer than 5 years before the date of enrollment, 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. 2. Have histologically confirmed Grade 3 IDH-mutant oligodendroglioma according to WHO 2021 criteria per local assessment. 3. Have residual or recurrent measurable disease per RANO 2.0 and confirmed by BICR, at the time of enrollment. 4. Have an IDH1 mutation (R132H/C/G/S/L). The presence of 1p19q co-deletion must also be confirmed. All results must be generated using a validated assay performed in a CLIA-certified/CAP-accredited (or local equivalent) clinical laboratory. Key Exclusion Criteria for Part 1: 1. Prior anti-cancer therapy, within the applicable periods shown below, before the start of the protocol treatment: 2. Systemic drug therapies: within 3 weeks (lomustine within 6 weeks) 3. Surgery: within 3 weeks 4. Radiation therapy: within 12 weeks 5. Investigational agents: within 5 half-lives for other investigational agents 6. Patient did receive the prior therapy targeted to IDH1 mutation.. 7. Known hypersensitivity to safusidenib or to any drug with similar chemical structure or to any other excipient present in the pharmaceutical form of safusidenib. Key Exclusion Criteria for Part 2 and 3: 1. Participants with prior or anticipated treatment with anti-angiogenic agents such as Avastin (bevacizumab), agents known to target IDH1 or IDH2, or investigational agents for glioma are excluded. 2. Have brainstem or spinal cord involvement either as primary location, site of multifocal involvement, or by significant tumor extension. 3. Significant functional or neurocognitive deficits, including uncontrolled seizures, that would preclude participation in protocol-defined study activities, as assessed by Investigator. 4. Evidence of diffuse leptomeningeal disease. 5. History of significant cardiac disease within 12 months prior to randomization (if applicable) or first dose of study drug (if randomization does not apply). 6. If taking corticosteroids, must be on a stable or decreasing dose for the 14 days prior to randomization (if applicable) or first dose of study drug (if randomization does not apply). 7. Participants with other malignancies must have received curative treatment and been disease-free for at least 3 years. Curatively resected skin cancer or curatively treated carcinoma in situ is allowed. 8. Have a condition that would interfere with, or increase the risk of, study participation. Key Exclusion Criteria for Part 2 1. 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. Key Exclusion Criteria for Part 3: 1\. Participants may not have received any prior anticancer therapy other than surgery (biopsy, sub-total, or gross total resection) for treatment of glioma, including radiotherapy.

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Conditions

The condition(s) this trial relates to.

astrocytoma (excluding glioblastoma) astrocytoma, IDH-mutant, grade 2 astrocytoma, IDH-mutant, grade 3 astrocytoma, IDH-mutant, grade 4 follicular lymphoma glioblastoma glioma IDH-mutant anaplastic astrocytoma IDH-mutant and 1p/19q-codeleted oligodendroglioma oligodendroglioma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Astrocytoma, grade IV Astrocytoma, IDH-mutant, grade 2 Astrocytoma, IDH-mutant, grade 3 Astrocytoma, IDH-mutant, grade 4 GLIOMA IDH1-mutant glioma Oligodendroglioma Oligodendroglioma, IDH-mutant and 1p/19q-codeleted

How to take part

Only the study team decides who joins. These are the ways to reach them.

The study's own enquiry address

This study publishes an address for enquiries. See it below .
The places running it

61 sites in 3 countries. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

Open the record ↗
A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Show contact details

Locations

Atrium Health Wake Forest Baptist Comprehensive Cancer Center
NOT_YET_RECRUITING

Winston-Salem, North Carolina, 27157, United States
Beijing Tiantan Hospital, Capital Medical University
RECRUITING

Beijing, Beijing Municipality, 100070, China
Billings Clinic
RECRUITING

Billings, Montana, 59101, United States
Cleveland Clinic
RECRUITING

Cleveland, Ohio, 44106, United States
Columbia University Medical Center
RECRUITING

New York, New York, 10032, United States

Contact Email: •••••@•••••
Dana-Farber Cancer Institute
RECRUITING

Boston, Massachusetts, 02215, United States
Duke Cancer Institute
RECRUITING

Durham, North Carolina, 27710, United States
Emory University
RECRUITING

Atlanta, Georgia, 30322, United States
Fred Hutch Cancer Center
RECRUITING

Seattle, Washington, 98195, United States
Henry Ford Hospital
RECRUITING

Detroit, Michigan, 48202, United States
Hoag Memorial Hospital Presbyterian
RECRUITING

Newport Beach, California, 92663, United States
Huashan Hospital Affiliated to Fudan University
RECRUITING

Shanghai, Shanghai Municipality, 200040, China
Huntsman Cancer Insititute, University of Utah
RECRUITING

Salt Lake City, Utah, 84112, United States
Icahn School of Medicine at Mount Sinai
NOT_YET_RECRUITING

New York, New York, 10029, United States
Indiana University
RECRUITING

Indianapolis, Indiana, 46202, United States
Inova Schar Cancer Institute
RECRUITING

Fairfax, Virginia, 22031, United States
MD Anderson Cancer Center
RECRUITING

Houston, Texas, 77030, United States
Massachusetts General Hospital
RECRUITING

Boston, Massachusetts, 02214, United States
Mayo Clinic - Arizona
RECRUITING

Phoenix, Arizona, 85013, United States
Mayo Clinic - Florida
RECRUITING

Jacksonville, Florida, 32224, United States

Contact Email: •••••@•••••
Mayo Clinic - Rochester
RECRUITING

Rochester, Minnesota, 55905, United States

Contact Email: •••••@•••••
Memorial Sloan Kettering Cancer Center
RECRUITING

New York, New York, 10065, United States
NYU Langone Health
RECRUITING

New York, New York, 10016, United States
Oregon Health Science University
RECRUITING

Portland, Oregon, 97239, United States

Contact Email: •••••@•••••
Orlando Health Cancer Institute
RECRUITING

Orlando, Florida, 32806, United States
Peking Union Medical College Hospital
RECRUITING

Beijing, Beijing Municipality, 100730, China
Peter MacCallum Cancer Centre
RECRUITING

Melbourne, Victoria, Australia
Royal Brisbane and Women's Hospital
RECRUITING

Herston, Queensland, Australia
Royal North Shore Hospital
RECRUITING

Saint Leonards, New South Wales, Australia
Rutgers Cancer Institute
RECRUITING

New Brunswick, New Jersey, 08901, United States
Sanbo Brain Hospital, Capital Medical University
RECRUITING

Beijing, Beijing Municipality, 100093, China
Sir Charles Gairdner Hospital
NOT_YET_RECRUITING

Nedlands, Western Australia, 6009, Australia
St Vincents Hospital Sydney (Kinghorn)
RECRUITING

Darlinghurst, New South Wales, Australia
St. Joseph's Hospital and Medical Center
RECRUITING

Phoenix, Arizona, 85013, United States
Stanford University
RECRUITING

Palo Alto, California, 94304, United States
Sun Yat-sen University Cancer Center
RECRUITING

Guangzhou, Guangdong, 510060, China
The Alfred
RECRUITING

Melbourne, Victoria, Australia
The First Affiliated Hospital of Fujian Medical University
RECRUITING

Fuzhou, Fujian, 350005, China
Thomas Jefferson University Hospital
RECRUITING

Philadelphia, Pennsylvania, 19107, United States

Contact Email: •••••@•••••
UPMC Hillman Cancer Center
RECRUITING

Pittsburgh, Pennsylvania, 15232, United States
UVA Health, Emily Couric Clinical Cancer Cente
RECRUITING

Charlottesville, Virginia, 22903, United States
University of Alabama
RECRUITING

Birmingham, Alabama, 35294, United States
University of California
RECRUITING

San Francisco, California, 94143, United States
University of California San Diego
RECRUITING

La Jolla, California, 92093, United States
University of California, Los Angeles
RECRUITING

Los Angeles, California, 90095, United States
University of Chicago
NOT_YET_RECRUITING

Chicago, Illinois, 60637, United States

Contact Email: •••••@•••••
University of Colorado Health Cancer Care
RECRUITING

Aurora, Colorado, 80045, United States
University of Florida Health
RECRUITING

Gainesville, Florida, 32608, United States
University of Kansas Medical Center
RECRUITING

Kansas City, Kansas, 66160, United States
University of Kentucky
RECRUITING

Lexington, Kentucky, 40536, United States
University of Miami Health
RECRUITING

Miami, Florida, 33136, United States
University of Rochester Medical Center
RECRUITING

Rochester, New York, 14642, United States
University of Texas Health Mays Cancer Center
RECRUITING

San Antonio, Texas, 78229, United States
University of Texas Southwestern Medical Center
RECRUITING

Dallas, Texas, 75390, United States
University of Wisconsin Health
RECRUITING

Madison, Wisconsin, 53792, United States
Vanderbilt-Ingram Cancer Center
RECRUITING

Nashville, Tennessee, 37203, United States
Washington University
RECRUITING

St Louis, Missouri, 63110, United States
West China Hospital of Sichuan University
RECRUITING

Chengdu, Sichuan, 610041, China
Xiangya Hospital of Central South University
RECRUITING

Changsha, Hunan, 410008, China
Xuanwu Hospital, Capital Medical University
RECRUITING

Beijing, Beijing Municipality, 10053, China
Yale University
RECRUITING

New Haven, Connecticut, 06510, United States

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