Clinical trial · Interventional
Phase III Study of Rindopepimut/GM-CSF in Patients With Newly Diagnosed Glioblastoma
An International, Randomized, Double-Blind, Controlled Study of Rindopepimut/GM-CSF With Adjuvant Temozolomide in Patients With Newly Diagnosed, Surgically Resected, EGFRvIII-positive Glioblastoma
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This 2-arm, randomized, phase III study will investigate the efficacy and safety of the addition of rindopepimut (an experimental cancer vaccine that may act to promote anti-cancer effects in patients who have tumors that express the EGFRvIII protein) to the current standard of care (temozolomide) in patients with recently diagnosed glioblastoma, a type of brain cancer. All patients will be administered temozolomide, the standard treatment for glioblastoma. Half the patients will be randomly assigned to receive rindopepimut and half the patients will be randomly assigned to receive a control called keyhole limpet hemocyanin. Patients will be treated in a blinded fashion (neither the patient or the doctor will know which arm of the study the patient is on). Patients will be treated until disease progression or intolerance to therapy and all patients will be followed for survival.
Conditions
Conditions (5)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Giant Cell Glioblastoma | Giant Cell Glioblastoma | ONTOLOGY_EXACT | 0.90 |
| Glioblastoma | Glioblastoma | CURATED_BROADER | 0.80 |
| Glioblastoma With Oligodendroglial Component | Brain Neoplasm | PROBABILISTIC | 0.70 |
| Gliosarcoma | Gliosarcoma | ONTOLOGY_EXACT | 0.90 |
| Small Cell Glioblastoma | Small Cell Glioblastoma | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (3)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| KLH | Drug | — | UNRESOLVED |
| Rindopepimut (CDX-110) with GM-CSF | Drug | — | UNRESOLVED |
| Temozolomide | Drug | Temozolomide | ALIAS |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Rindopepimut/GM-CSF plus Temozolomide
- interventionNames
- Drug: Rindopepimut (CDX-110) with GM-CSF
- Drug: Temozolomide
- type
- ACTIVE_COMPARATOR
- label
- KLH plus Temozolomide
- interventionNames
- Drug: Temozolomide
- Drug: KLH
Primary outcomes (1)
- measure
- Overall Survival
- timeFrame
- During treatment and every three months from end of treatment through end of study or approximately up to 5 years.
- description
- The primary efficacy endpoint of Overall Survival is defined as the number of months from randomization to the date of death (whatever the cause), and will be censored for patients who remain alive at completion of the study for patients with a gross total resection (\~n=374). The overall survival of patients will be monitored and compared between the two study arms until the end of the study.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria- Among other criteria, patients must meet the following conditions to be eligible for the study: 1. Adult patients, ≥ 18 years old 2. Newly diagnosed glioblastoma 3. Attempted surgical resection followed by conventional chemoradiation 4. Documented EGFRvIII positive tumor status by a Sponsor designated laboratory 5. No evidence of progressive disease from the post-operative period to the post-chemoradiation period 6. Candidate for, and agrees to receive, adjuvant (maintenance) temozolomide therapy 7. Systemic corticosteroid therapy at ≤2 mg of dexamethasone or equivalent per day for at least 3 days prior to randomization 8. WHO-ECOG Performance Status ≤ 2 9. Patients of childbearing/reproductive potential will be instructed to use birth control as defined by your doctor. Exclusion Criteria- Among other criteria, patients who meet the following conditions are NOT eligible for the study: 1. Stereotactic biopsy only (without further surgical resection) 2. Presence of diffuse leptomeningeal disease, gliomatosis cerebri, or infratentorial disease. 3. History, presence, or suspicion of metastatic disease 4. Patients who have received any additional treatment for glioblastoma, aside from surgical resection and chemoradiation with temozolomide 5. Active systemic infection requiring treatment 6. History of any malignancy (other than glioblastoma) during the last three years except non-melanoma skin cancer, in situ cervical cancer, treated superficial bladder cancer, cured, early-stage prostate cancer in a patient with PSA level less than ULN,or other carcinoma in situ that has been adequately treated and cured. 7. Planned major surgery 8. Evidence of current drug or alcohol abuse 9. Known allergy or hypersensitivity to keyhole limpet hemocyanin (KLH), GM-CSF (sargramostim; LEUKINE®), polysorbate 80 or yeast derived products, or a history of anaphylactic reactions to shellfish proteins 10. Severe acute or chronic medical or psychiatric condition or laboratory abnormality that could increase the risk associated with participating in a clinical trial 11. Women who are pregnant or lactating
References
Publications (2)
- RESULTWeller M, Butowski N, Tran DD, Recht LD, Lim M, Hirte H, Ashby L, Mechtler L, Goldlust SA, Iwamoto F, Drappatz J, O'Rourke DM, Wong M, Hamilton MG, Finocchiaro G, Perry J, Wick W, Green J, He Y, Turner CD, Yellin MJ, Keler T, Davis TA, Stupp R, Sampson JH; ACT IV trial investigators. Rindopepimut with temozolomide for patients with newly diagnosed, EGFRvIII-expressing glioblastoma (ACT IV): a randomised, double-blind, international phase 3 trial. Lancet Oncol. 2017 Oct;18(10):1373-1385. doi: 10.1016/S1470-2045(17)30517-X. Epub 2017 Aug 23. PMID 28844499
- DERIVEDSeliger C, Oppong FB, Lefranc F, Chinot O, Stupp R, Nabors B, Gorlia T, Weller M; EORTC Brain Tumor Group. Association of antidepressant drug use with outcome of patients with glioblastoma. Int J Cancer. 2023 Apr 1;152(7):1348-1359. doi: 10.1002/ijc.34344. Epub 2022 Nov 17. PMID 36346112