Berubicin in Adult Patients With Recurrent Glioblastoma Multiforme (WHO Grade IV)
- Conditions
- Recurrent Glioblastoma Multiforme
- Interventions
- Registration Number
- NCT04915404
- Lead Sponsor
- WPD Pharmaceuticals Sp. z o.o.
- Brief Summary
This is a multicenter, open-label, Phase 1b/2 efficacy and safety study of Berubicin utilizing a Simon's 2-stage design to confirm the efficacy (or futility) of a single arm of Berubicin treatment, administered at the recommended Phase 2 dose (RP2D) identified in Phase 1 studies (7.5 mg/m2 Berubicin HCl), on the endpoint of ORR in up to approximately 61 patients. A central reader will determine the radiologic responses for each patient according to m RANO criteria. The responder criteria for this Simon's design will be based on objective response criteria defined as individual patients achieving CR or PR per m-RANO criteria within 6 months from baseline.
- Detailed Description
The planned minimum duration of the study for an individual patient is approximately 11 weeks as follows:
* A 4-week Screening Period
* A 3-week Treatment Period (ie, one 3 week treatment cycle)
* A 4-week End-of-Treatment follow-up Period after the last dose The 3-week treatment cycles may be repeated in the absence of clinical and/or neurological deterioration, or unacceptable toxicity, and as long as both the patient and investigator agree that further therapy is in the patient's best interest.
After the End-of-Treatment follow-up visit, patients will enter a Post-Study follow-Up Period of up to approximately 2 years.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 5
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description pK Assessment of Berubicin and its active metabolite Berubicin Hydrochloride The first 18 patients will undergo a pK assessment of Berubicin and it's active metabolite Berubicinol during the dosing days of the first two cycles. After 18 patients are done n intern analysis will new performed.
- Primary Outcome Measures
Name Time Method objective response rate (ORR) 6 months To confirm the efficacy (or futility) of Berubicin treatment on objective response rate (ORR) defined as CR or PR per modified Response Assessment in Neuro-Oncology (m RANO) criteria in patients with GBM (World Health Organization \[WHO\] Grade IV) that has recurred after standard initial therapy, based on Simon's 2-stage design
- Secondary Outcome Measures
Name Time Method • To confirm the safety profile of Berubicin that was characterized during Phase 1 studies and assess the effect of Berubicin on event-free survival (EFS) 6 months • To assess the effect of Berubicin on event-free survival (EFS) defined as the length of time from the initiation of study drug administration to stable disease , disease progression, death, or discontinuation of treatment for any reason (eg, toxicity, intolerance, disease-related conditions, or failure to respond). Safety will be monitored by a safety review committee (SRC). The SRC will review PK and safety data from the first 2 cycles of treatment for the first approximately 18 patients (i.e., Stage 1) of this study
• To confirm the pharmacokinetic (PK) profile of Berubicin and its metabolite, berubicinol, that was characterized during Phase 1 studies 6 months intensive pK assessment of Berubicin and it's active metabolite will be performed during the dosing days of the first two cycles , this will be folowed by an interim analysis at 18 patients time point.Plasma samples will be collected from the first approximately 18 patients enrolled, and concentrations of Berubicin and its metabolite, berubicinol, will be measured
• To assess the effect of Berubicin on disease control rate (DCR) defined as CR or PR or stable disease [SD] per m-RANO criteria in patients with GBM after failure of standard first line therapy 12 months Screening/baseline MRI scans will be evaluated by a central reader for validation of eligibility and documentation of initial measurements. During the Treatment Period, the baseline scan will be compared with scans at 6-week (2-cycle) intervals for evaluation of disease by the investigator as well as the central reader according to m-RANO criteria.
Trial Locations
- Locations (2)
Uniwersyteckie Centrum Kliniczne Klinika Onkoligii i Radioterapii
🇵🇱Gdańsk, Poland
Narodowy Instytut Onkologii im. Marii Skłodowskiej-Curie,Państwowy Instytut Badawczy w Warszawie,Klinika Nowotworów Głowy i Szyi
🇵🇱Warszawa, Poland