E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
relapsing-remitting multiple sclerosis |
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E.1.1.1 | Medical condition in easily understood language |
relapsing-remitting multiple sclerosis |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10063399 |
E.1.2 | Term | Relapsing-remitting multiple sclerosis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 |
General Aim:
To assess the efficacy and safety of BGC20-0134 in patients with RRMS treated for 24 weeks in the double-blind period and for a further 24 weeks in the open-label period
Primary Endpoint:
Cumulative number of new gadolinium-enhanced (GdE) T1 weighted lesions developing while on treatment (specifically the sum of new GdE T1 lesions seen on MRI at weeks 12, 16, 20 and 24)
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E.2.2 | Secondary objectives of the trial |
• MRI endpoints:
Cumulative number of total GdE T1 weighted lesions
Cumulative number of new T2 weighted lesions
Patients free of GdE (T1-weighted) lesions
Change in volume of GdE T1 weighted, and new T2-weighted lesions
Brain atrophy
Cumulative number of new T1 hypointense lesions (black holes)
• Disease burden, T1 and T2 lesion activity at week 48
• Number of clinical relapses from baseline
• Change on the Expanded Disability Status Scale (EDSS)
• Number of patients receiving methylprednisolone treatment for a relapse
• Serum levels of cytokines during the first 24 weeks
• Quality of life (MSQOL-54) assessment
• PK for determination of circulating levels of BGC20-0134 and plasma concentrations of DHGLA during the first 24 weeks.
• Safety assessment
All secondary variables will be analysed in the double-blind period, however, all variables with the exception of some of the week 24 specific MRI variables will be analysed in the open-label phase. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients aged ≥18 with relapsing-remitting multiple sclerosis (EDSS score 0-5.5), with evidence of disease activity defined as at least one relapse or the presence of active MRI lesion/s consistent with MS during the year prior to inclusion
Patients who have refused to be treated with approved disease modifying therapies available for MS, for any reason and once the investigator has fully informed the patient about the related benefits and potential adverse events associated with such treatments. Also, patients for whom such treatments have proved to be intolerable. |
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E.4 | Principal exclusion criteria |
MS-related exclusion criteria:
• MS relapse or systemic corticosteroids in the previous 1 month
• Secondary progressive (SPMS), progressive relapsing (PRMS), or primary progressive MS (PPMS)
• Treatment with other agents for MS within the previous 3 months (intertferon beta, glatiramer acetate, intravenous immunoglobulin or plasmapherese) or within the previous 12 months (other MS drugs)
• Treatment with agents for the non-symptomatic treatment of MS within the previous 3 months
General exclusion criteria
• Pregnant or breast feeding
• Participation in a clinical study of an unlicensed drug in the previous 6 months
• Has a clinically significant abnormal serum biochemistry, haematology or urine examination values within 14 days prior to the start of the study.
• Has a 12-lead ECG with abnormal QTc interval within 14 days prior to the start of the study.
• Presence of pacemakers or foreign metal objects in the body which would contraindicate an MRI scan, or renal impairment which would contraindicate gadolinium injection
• Has any systemic disease, which can influence his/her safety and compliance, or the evaluation of disability.
• Any finding on medical history or physical examination which would prevent the patient being able to fully comply with the requirements of the study.
• Any finding on medical history or physical examination which would affect absorption of the study drug; e.g. metabolic disorders.
• Serious concomitant medical conditions: e.g. HIV infection, Hepatitis B or C, uncontrolled diabetes, malignancy.
• Current history of cancer, excluding localised non-melanoma skin cancer
• Known allergies to the study drug, its constituents or gadolinium (MRI contrast).
• Has suffered from major depression or any other psychiatric disorder
• Incapability of giving informed legal consent
• Co-worker, student, relative or spouse of the investigator
• Patients unable to swallow oral medications
• Previous participation in this study |
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E.5 End points |
E.5.1 | Primary end point(s) |
Cumulative number of new gadolinium-enhanced (GdE) T1 weighted lesions developing while on treatment (specifically the sum of new GdE T1 lesions seen on MRI at weeks 12, 16, 20 and 24). |
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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) |
see E.2.2 Secondary objectives
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
All secondary variables will be analysed in the double-blind period, however, all variables with the exception of some of the week 24 specific MRI variables will be analysed in the open-label phase. |
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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 | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
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 | Yes |
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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
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
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 11 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 29 |
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 |
European Union |
Russian Federation |
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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
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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 | 2 |
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 | 2 |
E.8.9.2 | In all countries concerned by the trial days | 15 |