UCB Proof of Concept Study in Patients With Primary Sjögren's Syndrome

A Randomized, Double-blind, Placebo-controlled, Proof-of-concept Study to Evaluate the Efficacy of UCB5857 Over 12 Weeks in Subjects With Primary Sjögren's Syndrome

This is a Phase 2, multicenter, double-blind, placebo-controlled, 12-week proof-of-concept study to assess the efficacy, safety, and tolerability of UCB5857 in subjects with primary Sjögren's Syndrome (pSS).

The primary objective of this study is to evaluate the efficacy on overall disease activity and safety of UCB5857 added to current treatment relative to placebo in subjects with pSS.

Study Overview

• Status: Terminated
• Conditions: Primary Sjögren's Syndrome
• Intervention / Treatment: Drug: UCB5857; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 27
• Phase: Phase 2

Contacts and Locations

Study Locations

• France
  • Brest, France
    • Ss0004 34
  • Le Kremlin-Bicêtre, France
    • Ss0004 30
  • Strasbourg, France
    • Ss0004 35
• Italy
  • L'Aquila, Italy
    • Ss0004 20
  • Palermo, Italy
    • Ss0004 21
  • Udine, Italy
    • Ss0004 22
• Spain
  • Cordoba, Spain
    • Ss0004 42
  • Villajoyosa, Spain
    • Ss0004 40
• Sweden
  • Stockholm, Sweden
    • Ss0004 50
• United Kingdom
  • Birmingham, United Kingdom
    • Ss0004 01
  • Essex, United Kingdom
    • Ss0004 05
  • Leeds, United Kingdom
    • Ss0004 04
  • Newcastle upon Tyne, United Kingdom
    • Ss0004 03
  • Swindon, United Kingdom
    • Ss0004 02

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 73 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Subject must be between 18 years and 75 years of age
• Women of childbearing potential must agree to use a highly effective method of birth control during the study and for a period of 3 months after their final dose of study drug. Women not agreeing to use birth control must be of non-childbearing potential defined as; permanently sterile, congenitally sterile or postmenopausal for at least 2 years prior to Screening (Visit 1). Women of childbearing potential are required to have a serum pregnancy test taken at Screening (Visit 1), which is confirmed to be negative by urine testing prior to the first dose of study drug at Week 1 (Visit 2) Male subjects with a partner of childbearing potential must be willing to use a condom when sexually active during the study and for 3 months after the last dose of study drug In addition female partner of childbearing potential of male subject must be willing to use a highly effective method of contraception for duration of study and for 3 months after last dose of study drug
• Subject must meet the 2002 AECG (American-European Consensus Group) criteria for Primary Sjӧgren's Syndrome
• Subject must have a serum test positive for anti-SSA/Ro (Ro-52 or Ro-60) and/or anti SSB/La autoantibodies

Exclusion Criteria:

• Subject has a diagnosis of any other autoimmune disease, ie, secondary Sjögren's syndrome (eg, rheumatoid arthritis, systemic lupus erythematosus
• Subject has a diagnosis of any other sicca syndrome (eg, history of head and neck radiation treatment, sarcoidosis chronic graft-versus-host disease)
• Subject has significant fibromyalgia syndrome as defined by the American College of Rheumatology 2010 classification criteria
• Subject has significant depression as defined by the 5th edition of the Diagnostic and Statistical Manual of Mental Disorders
• Subject has oral candidiasis
• Subject is female and is breast-feeding, pregnant, or plans to become pregnant or to start breastfeeding during the study or within 3 months after the final dose of the investigational medicinal product (IMP)

• Subject has evidence of an immunosuppressive state, including human immunodeficiency virus (HIV) infection, hypogammaglobulinemias, T-cell deficiencies, or human T-cell leukemia virus type 1 (HTLV-1)

  o Positive testing for HIV-1/2 at Screening (Visit 1)

• Subject has a history of chronic infections, including but not limited to concurrent acute or chronic viral hepatitis B (HBV) or hepatitis C (HCV)

  • Positive testing for HBV at Screening (Visit 1)
  • Positive testing for HCV at Screening (Visit 1)
• A subject with a history of a recent serious or life-threatening infection or any current signs or symptoms that may indicate a significant infection at Screening (Visit 1) to randomization, as per the Investigator's clinical judgment is also excluded Subject must have completed any prior anti-infective therapy prior to the first dose of study drug with the exception of anti-infectives taken specifically for the treatment of acne, rosacea, onychomycosis, or vaginal yeast infections; for the prophylaxis of urinary tract infections; or prophylaxis for pre-surgical or pre-procedural reasons (including dental procedures). Note: minocycline may not be used for these purposes
• Subject is at a particularly high risk of significant infection due to their lifestyle and/or occupation
• Subject has received intranasal influenza vaccine within the 8 weeks prior to Screening (Visit 1)
• Subject has significant hematologic abnormalities of hemoglobin <10.0 g/dL, or white blood cell (WBC) <2000 /mm^3, or absolute neutrophil count <1000 /mm^3, or platelets <100,000 /mm^3 at Screening (Visit 1)
• Subject has a history of cancer

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: UCB5857; UCB5857 once daily for 12 weeks	Drug: UCB5857; Active Substance: UCB5857 Pharmaceutical form: Capsule Concentration: 5 mg, 10 mg, 30 mg Route of administration: oral
Placebo Comparator: Placebo; Placebo once daily for 12 weeks	Drug: Placebo; Active substance: Placebo Pharmaceutical Form: Capsule Route of administration: oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline to Week 12 in the European League Against Rheumatism Sjögren's Syndrome Disease Activity Index (ESSDAI)	The ESSDAI is a physician administered questionnaire containing 12 organ-specific domains designed to measure disease activity	Week 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline to Week 4 in the European League Against Rheumatism Sjögren's Syndrome Disease Activity Index (ESSDAI)	The ESSDAI is a physician administered questionnaire containing 12 organ-specific domains designed to measure disease activity	Week 4
Change from Baseline to Week 8 in the European League Against Rheumatism Sjögren's Syndrome Disease Activity Index (ESSDAI)	The ESSDAI is a physician administered questionnaire containing 12 organ-specific domains designed to measure disease activity	Week 8
Change from Baseline to Week 4 in the EULAR Sjögren's Syndrome Patient Response Index (ESSPRI)	The ESSPRI is a patient completed questionnaire to assess subjective patient symptoms, which includes 3 domains (dryness, limb pain and fatigue)	Week 4
Change from Baseline to Week 8 in the EULAR Sjögren's Syndrome Patient Response Index (ESSPRI)	The ESSPRI is a patient completed questionnaire to assess subjective patient symptoms, which includes 3 domains (dryness, limb pain and fatigue)	Week 8
Change from Baseline to Week 12 in the EULAR Sjögren's Syndrome Patient Response Index (ESSPRI)	The ESSPRI is a patient completed questionnaire to assess subjective patient symptoms, which includes 3 domains (dryness, limb pain and fatigue)	Week 12
Change from Baseline to Week 4 in the stimulated salivary flow	The stimulated salivary flow test evaluates the status of salivary glands and the production of saliva. Saliva is collected into a graduated container after gustatory provocation with a stimulant	Week 4
Change from Baseline to Week 8 in the stimulated salivary flow	The stimulated salivary flow test evaluates the status of salivary glands and the production of saliva. Saliva is collected into a graduated container after gustatory provocation with a stimulant	Week 8
Change from Baseline to Week 12 in the stimulated salivary flow	The stimulated salivary flow test evaluates the status of salivary glands and the production of saliva. Saliva is collected into a graduated container after gustatory provocation with a stimulant	Week 12
Change from Baseline to Week 4 in the unstimulated salivary flow	The unstimulated salivary flow test evaluates the status of salivary glands and the production of saliva. Saliva is collected into a graduated container without gustatory provocation	Week 4
Change from Baseline to Week 8 in the unstimulated salivary flow	The unstimulated salivary flow test evaluates the status of salivary glands and the production of saliva. Saliva is collected into a graduated container without gustatory provocation.	Week 8
Change from Baseline to Week 12 in the unstimulated salivary flow	The unstimulated salivary flow test evaluates the status of salivary glands and the production of saliva. Saliva is collected into a graduated container without gustatory provocation	Week 12
Change in sum total tear secretion from Baseline to Week 12 measured by Schirmer´s I test(without anesthesia)	The Schirmer's test measures basic tear function. A 35 mm x 5mm size paper strip is inserted into each eye for a period of 5 minutes to measure the production of tears.	Week 12

Collaborators and Investigators

• Sponsor: UCB Celltech
• Collaborators: PRA Health Sciences
• Investigators: Study Director: UCB Cares, +18445992273 (UCB)

Publications and helpful links

General Publications

• Juarez M, Diaz N, Johnston GI, Nayar S, Payne A, Helmer E, Cain D, Williams P, Devauchelle-Pensec V, Fisher BA, Giacomelli R, Gottenberg JE, Guggino G, Kvarnstrom M, Mariette X, Ng WF, Rosas J, Sanchez Burson J, Triolo G, Barone F, Bowman SJ. A phase 2 randomized, double-blind, placebo-controlled, proof-of-concept study of oral seletalisib in primary Sjogren's syndrome. Rheumatology (Oxford). 2021 Mar 2;60(3):1364-1375. doi: 10.1093/rheumatology/keaa410.

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2015
• Primary Completion (Actual): September 7, 2017
• Study Completion (Actual): December 5, 2017

Study Registration Dates

• First Submitted: November 18, 2015
• First Submitted That Met QC Criteria: November 19, 2015
• First Posted (Estimate): November 20, 2015

Study Record Updates

• Last Update Posted (Actual): December 1, 2020
• Last Update Submitted That Met QC Criteria: November 26, 2020
• Last Verified: November 1, 2020

More Information

Terms related to this study

• Keywords: Primary Sjögren's Syndrome
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Eye Diseases; Disease; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Arthritis; Stomatognathic Diseases; Mouth Diseases; Lacrimal Apparatus Diseases; Arthritis, Rheumatoid; Xerostomia; Salivary Gland Diseases; Dry Eye Syndromes; Syndrome; Sjogren's Syndrome
• Other Study ID Numbers: SS0004; 2014-004523-51 (EudraCT Number)