E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary Sjogren's Syndrome |
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E.1.1.1 | Medical condition in easily understood language |
Primary Sjogren's Syndrome |
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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 | 21.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10040766 |
E.1.2 | Term | Sjogren's disease |
E.1.2 | System Organ Class | 100000004859 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the efficacy of nipocalimab in participants with primary Sjogren’s syndrome (pSS) versus placebo
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E.2.2 | Secondary objectives of the trial |
- To evaluate the safety of nipocalimab treatment versus placebo in participants with pSS
- To evaluate the pharmacokinetics (PK) and immunogenicity of nipocalimab in participants with pSS
- To evaluate the effect of nipocalimab versus placebo on levels of serum biomarkers related to pSS |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
optional labial salivary gland biopsy substudy |
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E.3 | Principal inclusion criteria |
- Meets classification criteria for primary sjogren's syndrome (pSS) by the 2016 American College of Rheumatology (ACR) / European League Against Rheumatism (EULAR) at the time of screening, (results either obtained during screening or documented in the participant's medical history are acceptable to fulfill these criteria for Schirmer's test, unstimulated salivary flow test, ocular staining score, or labial salivary gland biopsy), and was diagnosed with pSS no less than 26 weeks prior to screening - At screening is seropositive for antibodies to pSS-associated antigen A Ro/sjogren's syndrome-related antigen [SSA]) - Total Clinical European League Against Rheumatism Sjogren's Syndrome Disease Activity Index (clinESSDAI) score greater than or equal to (>=) 6 - At least one abnormal laboratory marker of pSS-related inflammatory disease activity, and at least low activity in one or more specified European League Against Rheumatism Sjogren's Syndrome Disease Activity Index (ESSDAI) domains - It is recommended to be up to date on all age-appropriate vaccinations prior to screening as per routine local medical guidelines. It is strongly recommended that participants will have completed a locally-approved (or emergency use-authorized) coronavirus disease 2019 (COVID-19) vaccination regime at least 2 weeks prior to study related visits or procedures. Study participants should follow applicable local vaccine labelling, guidelines, and standards-of-care for patientss receiving immune-targeted therapy will be followed when determining an appropriate interval between vaccination and study enrollment. |
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E.4 | Principal exclusion criteria |
-Has any confirmed or suspected clinical immunodeficiency syndrome not related to treatment of his/her pSS or has a family history of congenital or hereditary immunodeficiency unless confirmed absent in the participant - Comorbidities (for example, asthma, chronic obstructive pulmonary disease) which have required 3 or more courses of systemic glucocorticoids within the previous 12 months - Has any unstable or progressive manifestation of pSS that is likely to warrant escalation in therapy beyond permitted background medications and/or has severely active pSS - Has received oral cyclophosphamide within 3 months or intravenous (IV) cyclophosphamide within 6 months prior to first administration of study intervention - Has sjogren's syndrome overlap syndromes where another confirmed autoimmune rheumatic or systemic inflammatory condition (that is, rheumatoid arthritis [RA], systemic lupus erythematosus [SLE], scleroderma, inflammatory bowel disease [IBD]) is the primary diagnosis or has clinical manifestations that, in the opinion of the investigator, or the sponsor or sponsor's representative are likely to interfere with the investigator's ability to assess pSS manifestations |
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E.5 End points |
E.5.1 | Primary end point(s) |
Change from baseline in Clinical European League Against Rheumatism (EULAR) Sjögren's Syndrome Disease Activity Index (clinESSDAI) score at Week 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) |
1. Change from baseline in EULAR Sjögren Syndrome Patient Reported Index (ESSPRI) score at Week 24 2. Improvement of ≥4 points from baseline in EULAR Sjögren Syndrome Disease Activity Index (ESSDAI) score (minimal clinically important improvement) at Week 24 3. Improvement of ≥4 points from baseline in clinESSDAI score (minimal clinically important improvement) at Week 24 4. ESSPRI response at Week 24 5. Disease response according to the STAR composite score at Week 24 6. Improvement in disease activity level by ≥1 level in at least one clinESSDAI or ESSDAI domain at Week 24 7. Percentage of Participants with Treatment-emergent Adverse Events (TEAEs) 8. Percentage of Participants with Adverse Events of Special interests (AESIs) 9. Percentage of Participants with Treatment-emergent Serious Adverse Events (SAEs) 10. Percentage of Participants with Clinically Significant Abnormalities in Vital Signs 11. Percentage of Participants with Clinically Significant Abnormalities in Laboratory Parameters 12. Serum Concentration of Nipocalimab Over Time 13. Number of Participants with Antibodies to Nipocalimab (Anti-drug Antibodies [ADAs] and Neutralizing Antibodies [NAbs]) 14. Number of Participants with Change from Baseline in Biomarkers 15. Number of Participants with Change from Baseline in Autoantibodies 16. Percentage of Participants with TEAEs leading to treatment discontinuation |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
secondary endpoint 1-6 at week 24 secondary endpoint 8,10,11,13, up to 36 weeks secondary endpoint 7,9,12,16 up to 30 weeks secondary endpoint 14-15 baseline to week 36 |
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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 | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | Yes |
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 | 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 | 3 |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 37 |
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 |
Japan |
Taiwan |
United States |
France |
Poland |
Netherlands |
Spain |
Germany |
Italy |
Portugal |
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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 follow-up assessment (8 weeks after the last infusion of study intervention) for the last participant. |
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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 | 7 |
E.8.9.1 | In the Member State concerned days | |
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 | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |