NCT04164901

Brief Summary

Study AG881-C-004 is a phase 3, multicenter, randomized, double-blind, placebo-controlled study comparing the efficacy of vorasidenib to placebo in participants with residual or recurrent Grade 2 glioma with an IDH1 or IDH2 mutation who have undergone surgery as their only treatment. Participants will be required to have central confirmation of IDH mutation status prior to randomization. Approximately 340 participants are planned to be randomized 1:1 to receive orally administered vorasidenib 40 mg QD or placebo.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
331

participants targeted

Target at P50-P75 for phase_3

Timeline
24mo left

Started Jan 2020

Longer than P75 for phase_3

Geographic Reach
11 countries

84 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress76%
Jan 2020May 2028

First Submitted

Initial submission to the registry

November 11, 2019

Completed
4 days until next milestone

First Posted

Study publicly available on registry

November 15, 2019

Completed
2 months until next milestone

Study Start

First participant enrolled

January 5, 2020

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 6, 2022

Completed
1.2 years until next milestone

Results Posted

Study results publicly available

November 24, 2023

Completed
4.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2028

Expected
Last Updated

April 3, 2025

Status Verified

April 1, 2025

Enrollment Period

2.7 years

First QC Date

November 11, 2019

Results QC Date

September 5, 2023

Last Update Submit

April 1, 2025

Conditions

Keywords

AG-881

Outcome Measures

Primary Outcomes (1)

  • Progression-Free Survival (PFS)

    PFS is defined as the time from date of randomization to date of first documented radiographic PD (as assessed by the blinded independent review committee (BIRC) per modified Response Assessment for Neuro-oncology for Low-Grade Gliomas or date of death due to any cause, whichever occurs earlier.

    Up to approximately 30 months

Secondary Outcomes (11)

  • Time to Next Intervention (TTNI)

    Up to approximately 3 years

  • Tumor Growth Rate (TGR)

    every 6 months, up to 2 years and 9 months

  • Objective Response (OR) as Assessed by the Blinded Independent Review Committee (BIRC)

    approximatively 30 months

  • Complete Response (CR) and Partial Response (PR) by BIRC

    Approximatively 30 months

  • Time to Response (TTR) by BIRC

    Approximatively 30 months

  • +6 more secondary outcomes

Study Arms (2)

Vorasidenib

EXPERIMENTAL

Vorasidenib 40 mg, continuous daily dosing.

Drug: Vorasidenib

Matching Placebo

PLACEBO COMPARATOR

Matching placebo 40 mg, continuous daily dosing.

Drug: Matching Placebo

Interventions

Vorasidenib oral film-coated tablets

Also known as: AG-881
Vorasidenib

Matching Placebo oral tablets

Matching Placebo

Eligibility Criteria

Age12 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Be at least 12 years of age and weigh at least 40 kg.
  • Have Grade 2 oligodendroglioma or astrocytoma per WHO 2016 criteria.
  • Have had at least 1 prior surgery for glioma (biopsy, sub-total resection, gross-total resection), with the most recent surgery having occurred at least 1 year (-1 month) and not more than 5 years (+3 months) before the date of randomization, and no other prior anticancer therapy, including chemotherapy and radiotherapy and not be in need of immediate chemotherapy or radiotherapy in the opinion of the Investigator.
  • Have confirmed IDH1 (IDH1 R132H/C/G/S/L mutation variants tested) or IDH2 (IDH2 R172K/M/W/S/G mutation variants tested) gene mutation status disease by central laboratory testing during the Prescreening period and available 1p19q status by local testing (eg, fluorescence in situ hybridization \[FISH\], comparative genomic hybridization \[CGH\] array, sequencing) using an accredited laboratory.
  • Have MRI-evaluable, measurable, non-enhancing disease, as confirmed by the BIRC.
  • Have a Karnofsky Performance Scale (KPS) score (for participants ≥16 years of age) or Lansky Play Performance Scale (LPPS) score (for participants \<16 years of age) of ≥80%.

You may not qualify if:

  • Have had any prior anticancer therapy other than surgery (biopsy, sub-total resection, gross-total resection) for treatment of glioma including systemic chemotherapy, radiotherapy, vaccines, small-molecules, IDH inhibitors, investigational agents, laser ablation, etc.
  • Have features assessed as high-risk by the Investigator, including brainstem involvement either as primary location or by tumor extension, clinically relevant functional or neurocognitive deficits due to the tumor in the opinion of the Investigator (deficits resulting from surgery are allowed), or uncontrolled seizures (defined as persistent seizures interfering with activities of daily life AND failed 3 lines of antiepileptic drug regimens including at least 1 combination regimen).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (84)

University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

City of Hope

Duarte, California, 91010, United States

Location

University of California San Diego

La Jolla, California, 92093, United States

Location

UCLA Oncology Center

Los Angeles, California, 90095, United States

Location

University of California Irvine - Hospital

Orange, California, 92868, United States

Location

University of California San Francisco

San Francisco, California, 94143, United States

Location

Stanford Cancer Center

Stanford, California, 94305, United States

Location

University of Colorado Hospital - Anschutz Cancer Pavilion

Aurora, Colorado, 80045, United States

Location

Yale University, Yale Cancer Center

New Haven, Connecticut, 06510, United States

Location

Mayo Clinic Jacksonville

Jacksonville, Florida, 32224, United States

Location

Sylvester Comprehensive Cancer Center - University of Miami Hospital and Clinics

Miami, Florida, 33136, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

Indiana University Medical Center

Indianapolis, Indiana, 46202, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

University of Kentucky

Lexington, Kentucky, 40536, United States

Location

Maine Medical Partners Neurology

Scarborough, Maine, 04074, United States

Location

John Hopkins Cancer Center

Baltimore, Maryland, 21231, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02111, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, 48109, United States

Location

Henry Ford Hospital

Detroit, Michigan, 48202, United States

Location

Metro Minnesota Community Oncology

Minneapolis, Minnesota, 55416, United States

Location

Mayo Comprehensive Cancer

Rochester, Minnesota, 55905, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Ohio State University Comprehensive Cancer Center

Columbus, Ohio, 43210, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pittsburgh Hillman Cancer Center

Pittsburgh, Pennsylvania, 15232, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Tennessee Oncology

Nashville, Tennessee, 37203, United States

Location

Baylor University Medical Center

Dallas, Texas, 75246, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

The University of Utah, Huntsman Cancer Hospital

Salt Lake City, Utah, 84112, United States

Location

Seattle Cancer Care Alliance

Seattle, Washington, 98109, United States

Location

BC Cancer Agency

Vancouver, British Columbia, V5Z 4E6, Canada

Location

London Health Sciences Centre

London, Ontario, N6A 5W9, Canada

Location

Sunnybrook Health Sciences Centre

Toronto, Ontario, M4N 3M5, Canada

Location

Princess Margaret Hospital

Toronto, Ontario, Canada

Location

McGill University Health Center

Montreal, Quebec, H3A 2B4, Canada

Location

Centre Hospitalier Universitaire de Lille

Lille, 59037, France

Location

Hôpital Pierre Wertheimer

Lyon, 69394, France

Location

Hopitaux de La Timone

Marseille, 13385, France

Location

Hospitalier Pitié Salpétrière

Paris, 75013, France

Location

Universitätsklinikum Essen

Essen, 45122, Germany

Location

Universitätsklinikum Hamburg Eppendorf

Hamburg, 20246, Germany

Location

Universitätsklinikum Heidelberg

Heidelberg, 69120, Germany

Location

Klinikum Mannheim Universitätsklinikum

Mannheim, 68135, Germany

Location

Hadassah Medical Center

Jerusalem, 91120, Israel

Location

Rabin Medical Center

Petah Tikva, 49100, Israel

Location

Chaim Sheba Medical Center

Ramat Gan, 52621, Israel

Location

Tel Aviv Sourasky Medical Center

Tel Aviv, 6423906, Israel

Location

Azienda Ospedaliera Città della Salute e della Scienza di Torino

Turin, Piedmont, 10126, Italy

Location

Ospedale Bellaria

Bologna, 40139, Italy

Location

Istituto Oncologico Veneto - I.R.C.C.S.

Padua, 35128, Italy

Location

Istituto Nazionale Tumori Regina Elena

Roma, 144, Italy

Location

Istituto Clinico Humanitas

Rozzano, 20089, Italy

Location

Nagoya University Hospital

Nagoya, Aichi-ken, 466-8560, Japan

Location

Fujita Health University Hospital

Toyoake, Aichi-ken, 470-1192, Japan

Location

Hiroshima University Hospital

Minami-Ku, Hiroshima, 734-8551, Japan

Location

The University of Tokyo Hospital

Bunkyō-Ku, Tokyo, 113-8655, Japan

Location

National Cancer Center Hospital

Chuo Ku, Tokyo, 104-0045, Japan

Location

Kumamoto University Hospital

Kumamoto, 860-8556, Japan

Location

University Hospital, Kyoto Prefectural University of Medicine

Kyoto, 602-8566, Japan

Location

Kyoto University Hospital

Kyoto, 606-8507, Japan

Location

Okayama University Hospital

Okayama, 700-8558, Japan

Location

Haaglanden MC, Antoniushove

Leidschendam, South Holland, 2262 BA, Netherlands

Location

Erasmus Medical Center

Rotterdam, 3015 GD, Netherlands

Location

Universitair Medisch Centrum Utrecht

Utrecht, 3584 CX, Netherlands

Location

Hospital Universitario Vall d'Hebrón

Barcelona, 08035, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Hôpitaux Universitaire de Genève

Geneva, 1211, Switzerland

Location

Centre Hospitalier Universitaire Vaudois

Lausanne, 1011, Switzerland

Location

Universitätsspital Zürich

Zurich, 8006, Switzerland

Location

Freeman Hospital

Newcastle upon Tyne, England, NE7 7DN, United Kingdom

Location

The Royal Marsden NHS Foundation Trust

Sutton, Surrey, SM2 5PT, United Kingdom

Location

Western General Hospital Edinburgh - PPDS

Edinburgh, EH4 2XY, United Kingdom

Location

The Christie NHS Foundation Trust

Manchester, M20 4BX, United Kingdom

Location

Related Publications (2)

  • Mellinghoff IK, van den Bent MJ, Blumenthal DT, Touat M, Peters KB, Clarke J, Mendez J, Yust-Katz S, Welsh L, Mason WP, Ducray F, Umemura Y, Nabors B, Holdhoff M, Hottinger AF, Arakawa Y, Sepulveda JM, Wick W, Soffietti R, Perry JR, Giglio P, de la Fuente M, Maher EA, Schoenfeld S, Zhao D, Pandya SS, Steelman L, Hassan I, Wen PY, Cloughesy TF; INDIGO Trial Investigators. Vorasidenib in IDH1- or IDH2-Mutant Low-Grade Glioma. N Engl J Med. 2023 Aug 17;389(7):589-601. doi: 10.1056/NEJMoa2304194. Epub 2023 Jun 4.

  • Cloughesy TF, van den Bent MJ, Touat M, Blumenthal DT, Peters KB, Ellingson BM, Clarke JL, Mendez J, Yust-Katz S, Welsh L, Mason WP, Ducray F, Umemura Y, Nabors B, Holdhoff M, Hottinger AF, Arakawa Y, Sepulveda JM, Wick W, Soffietti R, Perry J, Giglio P, de la Fuente M, Maher E, Bottomley A, Tron AE, Yi D, Zhao D, Pandya SS, Steelman L, Hassan I, Wen PY, Mellinghoff IK; INDIGO trial investigators. Vorasidenib in IDH1-mutant or IDH2-mutant low-grade glioma (INDIGO): secondary and exploratory endpoints from a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Oncol. 2025 Dec;26(12):1665-1675. doi: 10.1016/S1470-2045(25)00472-3. Epub 2025 Oct 29.

MeSH Terms

Conditions

Glioma

Interventions

vorasidenib

Condition Hierarchy (Ancestors)

Neoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Glandular and EpithelialNeoplasms, Nerve Tissue

Results Point of Contact

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

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: Participants randomized in a 1:1 allocation (vorasidenib vs Placebo)
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 11, 2019

First Posted

November 15, 2019

Study Start

January 5, 2020

Primary Completion

September 6, 2022

Study Completion (Estimated)

May 1, 2028

Last Updated

April 3, 2025

Results First Posted

November 24, 2023

Record last verified: 2025-04

Data Sharing

IPD Sharing
Will share

Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data. Access can be requested for all interventional clinical studies: * used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US). * where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope. In addition, access can be requested for all interventional clinical studies in patients: * sponsored by Servier * with a first patient enrolled as of 1 January 2004 onwards * for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.

Shared Documents
STUDY PROTOCOL, SAP, ICF, CSR
Time Frame
After Marketing Authorisation in EEA or US if the study is used for the approval.
Access Criteria
Researchers should register on Servier Data Portal and fill in the research proposal form. This form in four parts should be fully documented. The Research Proposal Form will not be reviewed until all mandatory fields are completed.
More information

Available IPD Datasets

Individual Participant Data Set Access

Locations