E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Rheumatoid Arthritis (RA) |
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E.1.1.1 | Medical condition in easily understood language |
Rheumatoid Arthritis (RA) |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10021428 |
E.1.2 | Term | Immune system disorders |
E.1.2 | System Organ Class | 10021428 - Immune 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 |
To assess the efficacy and safety of dose regimens of ALX-0061 monotherapy administered s.c. to subjects with active RA |
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E.2.2 | Secondary objectives of the trial |
To assess the effects of ALX-0061 on quality of life, PK, PD, and immunogenicity of ALX 0061 and to explore potential dose regimens for ALX 0061 monotherapy, based on safety and efficacy, for further clinical development.
To obtain parallel descriptive information concerning the efficacy and safety of tocilizumab (TCZ) s.c. in the same clinical trial RA population.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
•Man or woman ≥ 18 years and < 75 years of age at the time of signing the informed consent form.
•Diagnosis of RA for at least 6 months prior to screening, and ACR functional class I-III.
•Received previous or current treatment with MTX, and is considered intolerant to MTX, or for whom continued treatment with MTX is considered inappropriate, or have contraindications for MTX use.
•Subjects must not have received MTX for at least 4 weeks before first administration of the study drug.
•Active RA |
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E.4 | Principal exclusion criteria |
•Have been treated with DMARDs/systemic immunosuppressive prior to first administration of study drug.
•Have received approved or investigational biological or targeted synthetic DMARD therapies for RA less than 6 months prior to screening.
For subjects who received prior rituximab, subjects with an inadequate recovery of B cells should be excluded regardless of when they received rituximab.
•Have a history of toxicity, non-tolerance, primary non-response or inadequate response to a biological therapy, or targeted synthetic DMARDs (including JAK inhibitors), for RA.
•Have received prior therapy blocking the interleukin-6 (IL-6) pathway at any time.
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E.5 End points |
E.5.1 | Primary end point(s) |
The proportion of subjects achieving an ACR20 response at Week 12. |
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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) |
-ACR20, ACR50, and ACR70 response over time.
-Disease activity: Disease Activity Score using 28 joint counts (DAS28
using CRP and erythrocyte sedimentation rate [ESR]), Simplified Disease
Activity Index (SDAI), Clinical Disease Activity Index (CDAI).
-EULAR DAS28 response (good, moderate, or no response).
-Remission using disease remission parameters: DAS28, SDAI, CDAI,
Boolean.
-Health Assessment Questionnaire-Disability Index (HAQ-DI).
-Physical and mental component scores of Short Form Health Survey
(SF-36).
-Functional Assessment of Chronic Illness Therapy-Fatigue (FACITFatigue).
-Pharmacokinetics
-Pharmacodynamics
-Safety
-Immunogenicity |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Time points for all secondary endpoints above are 12 weeks except Pharmacodynamics, Safety and Immunogenicity, the time point for which is 24 weeks |
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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 | Yes |
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 | 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 | 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) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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E.8.2.4 | Number of treatment arms in the trial | 4 |
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 | 7 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 27 |
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 |
Argentina |
Belgium |
Bulgaria |
Czech Republic |
Georgia |
Germany |
Hungary |
Macedonia, the former Yugoslav Republic of |
Mexico |
Moldova, Republic of |
Poland |
Romania |
Serbia |
Spain |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
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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Last visit of the last subject |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |