E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
recurrent resectable or unresectable Glioblastoma |
|
E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10018337 |
E.1.2 | Term | Glioblastoma multiforme |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the anti-tumor activity of BGJ398 for patients with GBM with an amplification, translocation, or activating mutation in FGFR1,2,3 or 4, based on PFS6 (PFS rate at 6 months as defined by RANO criteria as assessed by the investigator) |
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E.2.2 | Secondary objectives of the trial |
1) To further assess the anti-tumor activity of BGJ398 for patients with GBM with an amplification, translocation, or activating mutation in FGFR1,2,3 or 4, based on Overall Response Rate (ORR - patients with measurable disease -as defined by RANO criteria as assessed by the investigator
2) To further assess the anti-tumor activity of BGJ398 for patients with GBM with an amplification, translocation, or activating mutation in FGFR1,2,3 or 4, based on Overall Survival
3) Safety: type, frequency, and severity of AEs and SAEs; Tolerability: dose
interruptions, reductions and dose intensity, and evaluations of laboratory values |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1)Patients with histologically confirmed GBM at the time of diagnosis or prior relapse.
2) Documentation of amplification, translocation, or activating mutation to FGFR1, FGFR2, or FGFR3, or FGFR4
3) RANO defined tumor progression by MRI in comparison to a prior scan
4) Patients must have received prior external beam radiotherapy and temozolomide.
Other protocol defined criteria may apply |
|
E.4 | Principal exclusion criteria |
1) History of another primary malignancy
2) Prior or current treatment with a FGFR inhibitor
3) Neurological symptoms related to underlying disease requiring increasing doses of corticosteroids
4) Patients must not be taking Enzyme Inducing Anti-Epileptic Drug (EIAED). If previously on an EIAED, the patient must be off of it for at least two weeks prior to study treatment.
Other protocol defined criteria may apply |
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E.5 End points |
E.5.1 | Primary end point(s) |
progression free survival |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1) Overall response rate
2) Overall survival
3) safety and tolerability |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1) 8 months after LPLV
2) 8 months after LPLV
3) 8 months afterLPLV |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 12 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Belgium |
France |
Italy |
Netherlands |
Sweden |
Australia |
Germany |
Spain |
Switzerland |
United States |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
End of study will be upon completion of the follow up period for the last patient treated. This will be either upon study completion of the last patient treated or once the last patient has expired or all patients have completed the study and have been followed for at least one year after their first dose of study treatment, have been lost to follow-up, or withdrew consent, whichever occurs first. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |