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Clinical Trials/NCT04968912
NCT04968912
Completed
Phase 2

A Randomized, Placebo-controlled, Double-blind, Multicenter Study to Assess the Efficacy and Safety of Nipocalimab in Adults With Primary Sjogren's Syndrome (pSS)

Janssen Research & Development, LLC69 sites in 7 countries163 target enrollmentSeptember 21, 2021

Overview

Phase
Phase 2
Intervention
Placebo
Conditions
Sjogren's Syndrome
Sponsor
Janssen Research & Development, LLC
Enrollment
163
Locations
69
Primary Endpoint
Change from Baseline in Clinical European League Against Rheumatism Sjogren's Syndrome Disease Activity Index (clinESSDAI) Score at Week 24
Status
Completed
Last Updated
5 months ago

Overview

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of nipocalimab in participants with primary Sjogren's syndrome (pSS) versus placebo.

Detailed Description

Sjogren's syndrome is a chronic, progressive autoimmune disease of unclear etiology typically originating in exocrine glands and capable of affecting the function of almost any organ system in the body. Nipocalimab is a fully human aglycosylated immunoglobulin (Ig)G1 monoclonal antibody designed to selectively bind, saturate, and block the IgG binding site on the endogenous neonatal fragment crystallizable receptor (FcRn). Nipocalimab blocks the binding site for IgG on FcRn, leads directly to increased IgG catabolism and a decrease in circulating IgG antibody concentrations, including pathogenic IgG antibody concentrations, and directly inhibits inflammatory cellular responses to these pathogenic IgG. Therefore, Nipocalimab may successfully treat pSS and improve disease manifestations. The study will consist of Screening Period (less than or equal to \[\<=\] 6 Weeks), Double-blind Treatment Period (24 Weeks), and a Follow-up Period (6 Weeks). Key safety assessments will include adverse events (AEs), serious adverse events (SAEs), adverse events of special interests (AESIs), clinical laboratory safety tests (hematology, chemistry, urinalysis, and lipid profile) and vital signs. The total duration of the study is up to 36 weeks.

Registry
clinicaltrials.gov
Start Date
September 21, 2021
End Date
November 30, 2023
Last Updated
5 months ago
Study Type
Interventional
Study Design
Parallel
Sex
All

Investigators

Responsible Party
Sponsor

Eligibility Criteria

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 A \[SSA\])
  • 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 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 regimen 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 participants receiving immune-targeted therapy will be followed when determining an appropriate interval between vaccination and study enrollment

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 (for example, 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

Arms & Interventions

Group 1: Placebo

Participants will receive placebo intravenously (IV) every 2 weeks (q2w) through Week 22 along with standard of care treatments (\[including ophthalmic drops, artificial tears and saliva, punctum plugs, and secretagogues\], and/or one immunomodulator with or without low-dose glucocorticosteroids).

Intervention: Placebo

Group 1: Placebo

Participants will receive placebo intravenously (IV) every 2 weeks (q2w) through Week 22 along with standard of care treatments (\[including ophthalmic drops, artificial tears and saliva, punctum plugs, and secretagogues\], and/or one immunomodulator with or without low-dose glucocorticosteroids).

Intervention: Standard of Care Treatment

Group 2: Nipocalimab Dose 1

Participants will receive nipocalimab dose 1 IV q2w through Week 22 along with standard of care treatments (\[including ophthalmic drops, artificial tears and saliva, punctum plugs, and secretagogues\], and/or one immunomodulator with or without low-dose glucocorticosteroids).

Intervention: Nipocalimab

Group 2: Nipocalimab Dose 1

Participants will receive nipocalimab dose 1 IV q2w through Week 22 along with standard of care treatments (\[including ophthalmic drops, artificial tears and saliva, punctum plugs, and secretagogues\], and/or one immunomodulator with or without low-dose glucocorticosteroids).

Intervention: Standard of Care Treatment

Group 3: Nipocalimab Dose 2

Participants will receive nipocalimab dose 2 IV q2w through Week 22 along with standard of care treatments (\[including ophthalmic drops, artificial tears and saliva, punctum plugs, and secretagogues\], and/or one immunomodulator with or without low-dose glucocorticosteroids).

Intervention: Nipocalimab

Group 3: Nipocalimab Dose 2

Participants will receive nipocalimab dose 2 IV q2w through Week 22 along with standard of care treatments (\[including ophthalmic drops, artificial tears and saliva, punctum plugs, and secretagogues\], and/or one immunomodulator with or without low-dose glucocorticosteroids).

Intervention: Standard of Care Treatment

Outcomes

Primary Outcomes

Change from Baseline in Clinical European League Against Rheumatism Sjogren's Syndrome Disease Activity Index (clinESSDAI) Score at Week 24

Time Frame: Baseline to Week 24

The clinESSDAI is a validated tool used in clinical studies to measure the systemic disease activity in participants with primary Sjogren's syndrome. The clinESSDAI includes 11 domains divided into 3-4 activity levels, where zero represents no activity and low, medium, and high scores can vary in numerical value depending on the domain.

Secondary Outcomes

  • Improvement of Greater than or Equal to (>=) 4 Points from Baseline in ESSDAI Score (Minimal Clinically Important Improvement) at Week 24(Baseline to Week 24)
  • Improvement of >= 4 Points from Baseline in clinESSDAI Score (Minimal Clinically Important Improvement) at Week 24(Baseline to Week 24)
  • ESSPRI Response at Week 24(Week 24)
  • Improvement in Disease Activity Level by >= 1 Level in at Least One clinESSDAI or ESSDAI Domain at Week 24(Week 24)
  • Percentage of Participants with Clinically Significant Abnormalities in Laboratory Parameters(Up to 36 weeks)
  • Serum Concentration of Nipocalimab Over Time(Up to Week 30)
  • Number of Participants with Antibodies to Nipocalimab (Anti-drug Antibodies [ADAs] and Neutralizing Antibodies [NAbs])(Up to Week 36)
  • Number of Participants with Change from Baseline in Autoantibodies(Baseline to Week 36)
  • Change from Baseline in European League Against Rheumatism (EULAR) Sjogren Syndrome Patient Reported Index (ESSPRI) Score at Week 24(Baseline to Week 24)
  • Percentage of Participants with Adverse Events of Special interests (AESIs)(Up to 36 weeks)
  • Percentage of Participants with TEAEs Leading to Treatment Discontinuation(Up to 30 weeks)
  • Percentage of Participants with Clinically Significant Abnormalities in Vital Signs(Up to 36 weeks)
  • Number of Participants with Change from Baseline in Biomarkers(Baseline to Week 36)
  • Disease Response as Assessed by Sjögren's Tool for Assessing Response (STAR) Composite Score at Week 24(Week 24)
  • Improvement from Baseline in >= 3 of 5 Composite of Relevant Endpoints for Sjogren's Syndrome (CRESS) Categories at Week 24(Baseline to Week 24)
  • Percentage of Participants with Treatment-emergent Serious Adverse Events (SAEs)(Up to 30 weeks)
  • Percentage of Participants with Treatment-emergent Adverse Events (TEAEs)(Up to 30 weeks)

Study Sites (69)

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Nipocalimab Demonstrates Significant Efficacy in Phase 2 Sjögren's Disease Trial Published in The Lancet- Johnson & Johnson's nipocalimab, a first-in-class FcRn blocker, met its primary endpoint in the Phase 2 DAHLIAS study with statistically significant improvement in disease activity scores at Week 24 versus placebo. - The treatment showed favorable biomarker changes including lower rheumatoid factor levels, fewer circulating immune complexes, and decreased inflammatory markers in patients with moderate-to-severe Sjögren's disease. - Nipocalimab demonstrated a tolerable safety profile with preserved immune function and no increase in serious infections, while more than twice as many patients showed improved salivary flow compared to placebo. - The drug is the only investigational treatment granted Breakthrough Therapy Designation by the FDA for Sjögren's disease, with Phase 3 DAFFODIL study currently enrolling patients.Nipocalimab Receives FDA Breakthrough Therapy Designation for Sjögren's Disease- The FDA has granted Breakthrough Therapy Designation to nipocalimab for moderate-to-severe Sjögren's disease, marking it as the first therapy with this designation for the condition. - The designation is supported by Phase 2 DAHLIAS trial data, which demonstrated statistically significant improvements in disease activity compared to placebo at 24 weeks. - Nipocalimab, an investigational FcRn blocker, showed improvements in multiple organ assessments and patient-reported symptoms, with safety and tolerability consistent with previous trials. - A Phase 3 study is underway to further investigate nipocalimab's efficacy and safety in treating Sjögren's disease, addressing a critical unmet need for targeted therapies.