A Phase 1/2 interventional study of ReposMBZ in Cancer of the Gastrointestinal Tract and Cancer of Unknown Origin, sponsored by Repos Pharma. Terminated at 1 site in Sweden. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-01-22.
Sponsored by Repos Pharma · Phase 1/2, Interventional, and Treatment
This study will evaluate the safety and efficacy of mebendazole (ReposMBZ) in patient with advanced gastrointestinal cancer or cancer of unknown origin. All patients will be given ReposMBZ for 16 weeks continuous treatment, individually dosed based on the serum concentration of mebendazole.
Mebendazole has been used extensively during long time for local gut helminthic infections at low dose but also at considerably higher doses during months to years against invasive echinococcus infections. Recent research has now clearly indicated that mebendazole has anticancer effect. Given these observations and the experience of excellent tolerance to mebendazole the current clinical trial protocol is based on the repositioning strategy to more extensively investigate whether mebendazole could be developed into a useful anticancer drug.
779 studies on the registry are indexed under Gastrointestinal Neoplasms; 230 are open to participants now.
This study's enrollment of 11 is below the median of 61 across 568 interventional studies indexed under Gastrointestinal Neoplasms.
Browse Gastrointestinal Neoplasms studies →This is the only study on the registry with Repos Pharma as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Females of childbearing potential should use adequate contraception throughout the study;
Exclusion Criteria:
Inadequate laboratory parameters reflecting major organ function i.e.:
ReposMBZ 100 mg capsule by mouth followed by 8h PK sampling to decide the initial daily dose. Treatment: Repos MBZ capsules by mouth twice daily for 16 weeks, daily dose 50mg-4g, based on the serum level of mebendazole.
Drug: ReposMBZ
Capsules 50mg, 100mg, 200mg
Also known as: Mebendazole
Incidence of adverse events (AEs) probably or possibly related to ReposMBZ
AEs graded according to CTCAE 4.03.
Time frame: From date of first dose, up to 30 days after last dose of ReposMBZ or start of new treatment, whatever comes first, assessed up to 20 weeks after start of treatment phase.
Changes in plasma Albumin over time
Blood Chemistry (plasma): Albumin (g/L)
Time frame: From date of first dose, up to 30 days after last dose of ReposMBZ or start of new treatment, whatever comes first, assessed up to 20 weeks after start of treatment phase.
Changes in C-reactive protein (CRP) over time
Blood chemistry (plasma): CRP (mg/L)
Time frame: From date of first dose, up to 30 days after last dose of ReposMBZ or start of new treatment, whatever comes first, assessed up to 20 weeks after start of treatment phase.
Changes in plasma Sodium, Potassium, Calcium and Glucose over time
Blood chemistry (plasma): Sodium, Potassium, Calcium, Glucose (mmol/L)
Time frame: From date of first dose, up to 30 days after last dose of ReposMBZ or start of new treatment, whatever comes first, assessed up to 20 weeks after start of treatment phase.
Changes in plasma Bilirubin over time
Blood chemistry (plasma): Bilirubin (µmol/L)
Time frame: From date of first dose, up to 30 days after last dose of ReposMBZ or start of new treatment, whatever comes first, assessed up to 20 weeks after start of treatment phase.
Changes in plasma ALAT (alanine aminotransferase), ASAT (aspartate aminotransferase), LDH (lactate dehydrogenase), ALP (alkaline phosphatase) over time
Blood chemistry (plasma): ALAT, ASAT, LDH, ALP (µkat/L)
Time frame: From date of first dose, up to 30 days after last dose of ReposMBZ or start of new treatment, whatever comes first, assessed up to 20 weeks after start of treatment phase.
Changes in Haemoglobin over time
Haematology: Haemoglobin (g/L)
Time frame: From date of first dose, up to 30 days after last dose of ReposMBZ or start of new treatment, whatever comes first, assessed up to 20 weeks after start of treatment phase.
Changes in red, white and platelet blood cell count over time
Haematology: RBC (red blood cell count), White blood cells with differential count and platelets (absolute count/L)
Time frame: From date of first dose, up to 30 days after last dose of ReposMBZ or start of new treatment, whatever comes first, assessed up to 20 weeks after start of treatment phase.
Changes in Activated Partial Thromboplastin Time (APTT) over time
Coagulation (plasma): APTT (s)
Time frame: From date of first dose, up to 30 days after last dose of ReposMBZ or start of new treatment, whatever comes first, assessed up to 20 weeks after start of treatment phase.
Changes in Prothrombin complex (PK/INR) over time
Coagulation (plasma): Prothrombin complex (INR)
Time frame: From date of first dose, up to 30 days after last dose of ReposMBZ or start of new treatment, whatever comes first, assessed up to 20 weeks after start of treatment phase.
Changes in blood pressure over time
Systolic and diastolic blood pressure (mmHg) Weight (kg)
Time frame: From date of first dose, up to 30 days after last dose of ReposMBZ or start of new treatment, whatever comes first, assessed up to 20 weeks after start of treatment phase.
Changes in heart rate over time
Supine heart rate (beats per minute)
Time frame: From date of first dose, up to 30 days after last dose of ReposMBZ or start of new treatment, whatever comes first, assessed up to 20 weeks after start of treatment phase.
Changes in body temperature over time.
Body temperature (Celsius degrees)
Time frame: From date of first dose, up to 30 days after last dose of ReposMBZ or start of new treatment, whatever comes first, assessed up to 20 weeks after start of treatment phase.
Tumour response: CT/MRI assessed according to RECIST 1.1
Best overall radiological response Time to tumour progression (TTP) compared with TTP on the treatment just preceding this protocol.
Time frame: From date of first dose ReposMBZ until the date of first documented progression or date of death from any cause, whichever comes first, assessed up to 24 months (end of study).
The peak serum concentration (Cmax) of ReposMBZ after single dose administration.
Cmax will be used to decide the starting dose in the treatment phase.
Time frame: Pre-dose, 30 minutes, 60 minutes, 2 hours, 3 hours, 4 hours, 6 hours, 8 hours.
Area under the serum concentration versus time curve (AUC) for ReposMBZ
Analysis of serum concentration after single dose administration of ReposMBZ.
Time frame: Pre-dose, 30 minutes, 60 minutes, 2 hours, 3 hours, 4 hours, 6 hours, 8 hours.
The Cmax serum concentration of ReposMBZ after repeated dose administration.
Cmax will be used to adjust the dose until target S-mebendazole level is reached and to show the individual variation of S-mebendazole concentration over time
Time frame: Pre-dose, 30 minutes, 60 minutes, 2 hours, 3 hours, 4 hours, 6 hours, 8 hours (only up to the time point for Cmax after single dose), assessed up to 16 weeks after start of treatment phase.
Target S-mebendazole concentration after repeated dose administration.
Number of patients that reach the steady state S-mebendazole target concentration.
Time frame: From first dose in treatment phase and assessed up to 16 weeks after start of treatment phase.
Time to reach the steady state S-mebendazole target concentration after repeated dose administration..
Time from first dose in treatment phase until target S-mebendazole concentration is reached
Time frame: From first dose in treatment phase and assessed up to 16 weeks after start of treatment phase.
Systemic immune activation.
Change of cytokine levels in blood, evaluated by cytokine array.
Time frame: From baseline up to 20 weeks after start of treatment phase, assessed up to 24 months (end of study).
Immune cell activation.
Up-regulation of activation markers compared to baseline, evaluated by flow cytometry.
Time frame: From baseline and up to 20 weeks after start of treatment phase, assessed up to 24 months (end of study).
Overall survival.
Months of survival from first dose until death of any cause.
Time frame: From date of first dose ReposMBZ to date of death, assessed up to 24 months (end of study).
Change in tumour load and TTP according to irRECIST
Best over all radiological response according to irRECIST 1.1.
Time frame: From date of first dose ReposMBZ until the date of first documented progression or date of death from any cause, whichever comes first, assessed up to 24 months (end of study).
Association between ReposMBZ efficacy and properties of the diagnostic tumour tissue (optional)
Genetic changes and infiltration of immune cells, grade, molecular subtype, gene and protein expression and tumour infiltration and subtypes of macrophages and lymphocytes.
Time frame: Assessed up to 24 months (end of study).
Comparison of diagnostic tissue and a fresh tumour biopsy after 4 weeks of treatment at the target S-mebendazole concentration (optional).
Changes in the properties analysed in the archived tissue (baseline) and the fresh biopsy.
Time frame: Collected up to 20 weeks after start of treatment phase, assessed up to 24 months (end of study).
Association between S-mebendazole and efficacy and safety
Mean S-mebendazole concentration during treatment phase in relation to safety (CTCAE 4.03 grade 3 and 4 toxicity) and efficacy (tumour response and TTP) respectively.
Time frame: From date of first dose until date of first documented progression or date of death, whichever comes fist, assessed up to 24 months (end of study).
Plan to share: No
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