Vorasidenib in Combination With Temozolomide (TMZ) in IDH-mutant Glioma

NCT ID: NCT06478212

Last Updated: 2025-11-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

42 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-01-22

Study Completion Date

2028-06-30

Brief Summary

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The objective of this study is to determine the safety and tolerability of vorasidenib in combination with temozolomide (TMZ) and to establish the recommended combination dose (RCD) of vorasidenib. The study will begin as a Phase Ib study to determine the RCD and then will transition to a Phase II study to assess the clinical efficacy of vorasidenib at the RCD in combination with TMZ. During the treatment period participants will have study visits on day 1 and 22 of each cycle, with additional visits occurring during the first cycle of the Phase 1b study. Approximately 30 days after treatment has ended, a safety follow-up visit will occur and then participants will be followed for survival every 3 months. Study visits may include questionnaires, blood tests, ECG, vital signs, and a physical examination.

Detailed Description

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Conditions

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IDH1-mutant Glioma IDH2-mutant Glioma

Keywords

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IDH-mutant glioma IDH mutation vorasidenib S95032 Phase 1/2 pediatric

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Phase 1b: Vorasidenib and Temozolomide (TMZ)

Group Type EXPERIMENTAL

Vorasidenib

Intervention Type DRUG

To be taken by mouth once daily in 28-day cycles with no break between cycles

Temozolomide (TMZ)

Intervention Type DRUG

To be taken by mouth once daily for the first 5 days of each 28-day cycle, for a maximum of 12 cycles

Phase 2: Vorasidenib recommended combination dose (RCD) and Temozolomide (TMZ)

Group Type EXPERIMENTAL

Vorasidenib

Intervention Type DRUG

To be taken by mouth once daily in 28-day cycles with no break between cycles

Temozolomide (TMZ)

Intervention Type DRUG

To be taken by mouth once daily for the first 5 days of each 28-day cycle, for a maximum of 12 cycles

Interventions

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Vorasidenib

To be taken by mouth once daily in 28-day cycles with no break between cycles

Intervention Type DRUG

Temozolomide (TMZ)

To be taken by mouth once daily for the first 5 days of each 28-day cycle, for a maximum of 12 cycles

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Be ≥12 years of age with a weight at screening ≥40 kg.
* Have documented IDH1 or IDH2 mutation based on local testing of tumor tissue by an accredited laboratory
* Have adequate renal function, defined as a creatinine clearance ≥40 mL/min based on the Cockcroft-Gault glomerular filtration rate estimation: (140 - Age) × (Weight in kg) × (0.85 if female) / 72 × serum creatinine (mg/dL).
* Have adequate bone marrow function as evidenced by:

1. Absolute neutrophil count ≥1,500/mm3 or 1.5×109/L
2. Hemoglobin ≥9 g/dL or 90 g/L
3. Platelets ≥100,000/mm3 or 100×109/L
* Have expected survival of ≥3 months.
* KPS or LPPS ≥70 at the start of study treatment.
* Participants on corticosteroids for reasons related to glioma must be on a stable or decreasing dose (≤4mg/day dexamethasone or equivalent) for ≥5 days before the start of study treatment.
* Female participants of reproductive potential must have a negative serum pregnancy test before starting study treatment.

Phase 1b ONLY:

* Have histologically confirmed Grade 2, 3 or 4 IDHm (as per WHO 2021) glioma (astrocytoma or oligodendroglioma).

1. For oligodendroglioma: Have local testing at an accredited laboratory demonstrating presence of 1p19q co deletion
2. For astrocytoma: Have local testing by an accredited laboratory demonstrating lack of 1p19q co-deletion and/or documented loss of nuclear ATRX expression or ATRX mutation
* Are appropriate to receive TMZ as post-radiotherapy (RT) adjuvant therapy or as treatment for first disease recurrence after prior RT and/or chemotherapy, per Investigator judgement. For those receiving TMZ in the post-RT adjuvant setting, study treatment must begin no more than 6 weeks after completion of RT.
* Have adequate hepatic function as evidenced by:

1. Serum total bilirubin ≤1.5×upper limit of normal (ULN); if ≥1.5×ULN and due to Gilbert syndrome, total bilirubin ≤3×ULN with direct bilirubin ≤ULN,
2. AST and ALT ≤ULN, and
3. Alkaline phosphatase ≤2.5×ULN.

Phase 2 ONLY:

* Have histologically confirmed Grade 4 astrocytoma, IDHm (per 2021 WHO criteria). Those who meet the Grade 4 designation via homozygous deletion of CDKN2A/B are eligible.
* Have absence of 1p19q co-deletion (i.e., non-co-deleted, or intact) and/or documented loss of nuclear ATRX expression or ATRX mutation by local testing.
* Have received SOC RT with concurrent TMZ (RT-TMZ) before enrollment. Study treatment must begin no more than 6 weeks after completion of RT-TMZ.
* Have adequate hepatic function as evidenced by:

1. Serum total bilirubin ≤1.5×ULN; if ≥1.5×ULN and due to Gilbert syndrome, total bilirubin ≤3×ULN with direct bilirubin ≤ULN,
2. AST and ALT at or below the upper limit of normal. An elevation ≤1.5×ULN and considered not clinically significant by the Investigator may be allowed after Medical Monitor (Sponsor) approval, and
3. Alkaline phosphatase ≤2.5×ULN.

Exclusion Criteria

* Unable to swallow oral medication.
* Are pregnant or breastfeeding.
* Are participating in another interventional study at the same time; participation in non-interventional registries or epidemiological studies is allowed.
* Have leptomeningeal disease.
* Have a known coagulopathy.
* Received prior therapy with an IDH inhibitor, IDH-directed vaccine, or bevacizumab.
* Have a history of another concurrent primary cancer, with the exception of:

1. curatively resected non-melanoma skin cancer, or
2. curatively treated carcinoma in situ. Participants with other previously treated malignancies are eligible provided they have been disease-free for 3 years at Screening.
* Have a known diagnosis of replication repair-deficient glioma (e.g., a known diagnosis of constitutional mismatch repair deficiency or Lynch syndrome).
* Have a known hypersensitivity to any of the components or metabolites of vorasidenib or TMZ.
* Have significant active cardiac disease within 6 months before Screening, including New York Heart Association (NYHA) Class III or IV congestive heart failure, myocardial infarction, unstable angina, and/or stroke.
* Have heart rate corrected QT interval (using Fridericia's formula) (QTcF) ≥450 msec or have other factors that increase risk of QT prolongation or arrhythmic events (e.g., heart failure, hypokalemia, family history of long QT interval syndrome). Right bundle branch block and prolonged QTcF interval may be permitted based on local cardiology assessment.
* Have known active hepatitis B (HBV) or hepatitis C (HCV) infections, known positive human immunodeficiency virus (HIV) antibody results, or acquired immunodeficiency syndrome (AIDS) related illness. Participants with a sustained viral response to HCV treatment or immunity to prior HBV infection will be permitted. Participants with chronic HBV or HIV that is adequately suppressed per institutional practice will be permitted.

Phase 1b ONLY:

* For those receiving TMZ in the frontline post-RT adjuvant setting: Have progressive disease during RT or after completion of SOC RT and before the start of study treatment.
* For those receiving TMZ in the recurrent disease setting:

1. Have received prior systemic anti-cancer therapy (other than surgery) within 1 month (or 6 weeks for nitrosoureas and 6 months for TMZ) of the start of study treatment. In addition, the first dose of study treatment should not occur before a period of 28 days or ≥5 half-lives of any prior investigational agent have elapsed, whichever is longer.
2. Have received more than one prior line of therapy for glioma (Note: prior RT + chemotherapy is considered one line of therapy).
* Had Grade ≥2 hepatic-related toxicity (AST, ALT, and/or bilirubin elevations) during prior systemic chemotherapy
* Had Grade 4 hematologic toxicity (excluding lymphopenia) that did not recover within 7 days during a prior course of TMZ

Phase 2 ONLY:

* Have received any other glioma-directed therapy other than surgery and SOC RT-TMZ
* Have progressive disease during RT-TMZ or after completion of SOC RT-TMZ and before the start of study treatment.
* Had Grade 4 hematologic toxicity (excluding lymphopenia) that did not recover within 7 days during concurrent RT-TMZ
* Had Grade ≥2 hepatic-related toxicity (AST, ALT, and/or bilirubin elevations) during concurrent RT-TMZ
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Institut de Recherches Internationales Servier

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of California Los Angeles

Los Angeles, California, United States

Site Status RECRUITING

University of California, San Francisco (UCSF) School of Medicine

San Francisco, California, United States

Site Status WITHDRAWN

University of Miami

Miami, Florida, United States

Site Status RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status WITHDRAWN

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Site Status RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status RECRUITING

Duke University Medical Center

Durham, North Carolina, United States

Site Status RECRUITING

MD Anderson Cancer Center

Houston, Texas, United States

Site Status RECRUITING

Medical University of Vienna - AKH

Vienna, , Austria

Site Status RECRUITING

West China Hospital of Sichuan University

Chengdu, Sichuan, China

Site Status WITHDRAWN

Beijing Tiantan Hospital, Capital Medical University

Beijing, , China

Site Status RECRUITING

Huashan Hospital, Fudan University

Shanghai, , China

Site Status RECRUITING

Hôpital Pierre Wertheimer

Lyon, , France

Site Status RECRUITING

Hôpital Pitié-Salpêtrière

Paris, , France

Site Status RECRUITING

IUCT-Oncopole Institut Universitaire du Cancer

Toulouse, , France

Site Status RECRUITING

Universitätsklinikum Heidelberg

Heidelberg, , Germany

Site Status RECRUITING

Medizinische Fakultät Mannheim, Universität Heidelberg

Mannheim, , Germany

Site Status RECRUITING

Universitätsklinikum Regensburg

Regensburg, , Germany

Site Status RECRUITING

Rabin Medical Center - Davidoff Cancer Center

Petah Tikva, , Israel

Site Status WITHDRAWN

The Tel Aviv Sourasky Medical Center (TASMC) (Ichilov Hospital)

Tel Aviv, , Israel

Site Status RECRUITING

Instituto Clinico Humanitas IRCCS

Rozzano, Milan, Italy

Site Status RECRUITING

IOV - Ospedale Busonera

Padua, , Italy

Site Status RECRUITING

Ospedale Molinette - Centro Oncologico Ematologico

Turin, , Italy

Site Status RECRUITING

Kumamoto University Hospital

Kumamoto, , Japan

Site Status RECRUITING

Kyoto University Hospital

Kyoto, , Japan

Site Status RECRUITING

Nagoya University Hospital

Nagoya, , Japan

Site Status RECRUITING

National Cancer Center Hospital

Tokyo, , Japan

Site Status RECRUITING

Erasmus MC

Rotterdam, , Netherlands

Site Status RECRUITING

H. Valle de Hebron

Barcelona, , Spain

Site Status RECRUITING

Hospital 12 de Octubre

Madrid, , Spain

Site Status RECRUITING

Christie Hospital

Manchester, , United Kingdom

Site Status RECRUITING

The Royal Marsden in Sutton

Sutton, , United Kingdom

Site Status RECRUITING

Countries

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United States Austria China France Germany Israel Italy Japan Netherlands Spain United Kingdom

Central Contacts

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Institut de Recherches Internationales Servier (I.R.I.S.) Clinical Studies Department

Role: CONTACT

Phone: +33 1 55 72 60 00

Email: [email protected]

Other Identifiers

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S095032-211

Identifier Type: -

Identifier Source: org_study_id