A Phase 2 Study to Investigate Efficacy and Safety of HZN-1116 in Participants With Sjögren’s Syndrome

Phase 2

Recruiting

• Conditions: Sjögren’s Syndrome

• Registration Number: 2023-507680-19-00
• Lead Sponsor: Horizon Therapeutics Ireland Designated Activity Company

Brief Summary

"Population #1:

- To evaluate the effect of HZN-1116 on systemic manifestations of Sjӧgren’s Syndrome (SS) in participants with moderate-to-severe systemic disease activity

Population #2:

- To evaluate the effect of HZN-1116 on patient-reported symptoms of SS in participants with unsatisfactory symptom state"

Detailed Description

The study will enroll 2 SS populations: Population 1 will include participants with moderate to high systemic disease activity; Population 2 will include participants with moderate to severe subjective symptoms. This study will include 3 periods: screening (5 weeks), treatment period (48 Weeks) and follow-up period (12 weeks). In the treatment period, participants from each of the populations will be randomized to receive subcutaneous (SC) dose of HZN-1116 or placebo.

Acquired from Horizon in 2024.

Study Timeline

• Study First Submitted: 2024-02-16
• Study First Submitted QC: 2024-03-07
• Study First Posted: 2024-03-15
• Study Start: 2024-05-09
• Status Verified: 2025-09
• Last Update Submitted: 2025-09-24
• Last Update Posted: 2025-09-25
• Primary Completion: 2026-06-02
• Study Completion: 2027-02-09

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Not specified
• Target Recruitment: 151

Inclusion Criteria

Adults, ≥ 18 and ≤ 75 years of age at time of informed consent (the minimum age for adult participants can be > 18 years of age based on country-specific regulations). Participants must be capable of providing their own informed consent.

Diagnosed with SS by meeting the 2016 ACR/EULAR Classification Criteria. If SS diagnosis based on positive anti-Ro autoantibody, anti-Ro positivity must be confirmed by central lab.

Population #1 only: a. Have an ESSDAI score of ≥ 5 at screening despite symptomatic (eg, nonsteroidal anti-inflammatory drugs [NSAIDs]) or local therapy at screening. The following domains will be scored but they will not contribute to the minimum ESSDAI score of 5 required for inclusion as these domains may have lower sensitivity to change over duration of trial: peripheral nervous system, central nervous system, and pulmonary. Population #2 only: a. Have an ESSPRI score of ≥ 5 at screening. b. Have an ESSDAI score of < 5 at screening.

Positive for either anti-Ro autoantibodies or rheumatoid factor (RF), or both at screening (as per the definition of the standard central laboratory test).

Residual salivary gland function as defined by whole stimulated salivary flow > 0.1 mL/min or with residual lacrimal gland function defined as an unanesthetized Schirmer’s test ≥ 5 mm/5 min.

Females of childbearing potential who are sexually active with a nonsterilized male partner must use a highly effective method of contraception from signing the informed consent form (ICF) and must agree to continue using such precautions through the end of the study (or 4 months after IP administration, if participant withdraws from study) and refrain from ova donation during this period; cessation of contraception after this point should be discussed with a responsible physician.

Nonsterilized male participants who are sexually active with a female partner of childbearing potential must use a condom with spermicide (unless spermicide is not available or restricted per local regulations) till the end of the study and refrain from sperm donation during this period and for a minimum of 90 days after last IP administration.

Written informed consent and any locally required authorization (eg, Health Insurance Portability and Accountability Act in the United States [US], European Union [EU] Data Privacy Directive in the EU) obtained from the participant prior to performing any protocolrelated procedures, including screening evaluations.

Fully vaccinated against severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2)according to current local authority guidelines, if any, at least 2 weeks prior to screening unless individual refuses vaccination. Initial or subsequent coronavirus disease 2019 (COVID-19) vaccine administration is permitted during the study as long as it is not administered during the screening period or within 2 weeks after Dose 1; if vaccine is to be administered during this window, screening should be delayed to complete vaccination

Exclusion Criteria

Concomitant systemic sclerosis.

Individuals who have received live (attenuated) vaccine within the 4 weeks prior to randomization. Non-live vaccines are permitted during the study (see Inclusion Criterion 9for COVID-19 vaccines).

Last administration of experimental biologic or oral agents < 6 months or 5 half-lives, whichever is longer, before screening.

Active malignancy or history of malignancy within the last 5 years, except as follows: a. In situ carcinoma of the cervix treated with apparent success with curative therapy > 12 months prior to screening; OR b. Cutaneous basal cell carcinoma following presumed curative therapy.

Individuals who are pregnant or lactating or planning to become pregnant during the study.

Individuals with known history of severe allergy or reaction to any component of the IP formulation or to any other biologic therapy.

Individuals with any severe or life-threatening cardiovascular (including vasculitis and uncontrolled hypertension), respiratory, endocrine, gastrointestinal, hematological, neurological, psychiatric, or systemic disorder or any other condition that in the opinion of the Investigator, would place the individual at unacceptable risk of complications, interfere with evaluation of the IP, or confound the interpretation of participant safety or study results.

Individuals who, in the opinion of the Investigator, are unable or unwilling to comply with protocol requirements (eg, active drug or alcohol abuse or for other reasons), including the completion of the Diary for Assessing Sjögren’s Patient Reported Index (DASPRI).

Individuals who have a positive test for, or have been treated for, hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) infection. A positive test for hepatitis B infection at screening is defined as: (1) positive for hepatitis B surface antigen (HbsAg); OR (2) positive for hepatitis B core antibody (HbcAb). Patients HbsAg negative, hepatitis B surface antibody (HbsAb) positive and HbcAb negative due to vaccination are eligible for the study. Individuals with a positive test for or a history oftreatment for hepatitis C are excluded unless they have a documented sustained viral response to antiviral drugs approved for the treatment of hepatitis C, defined as an undetectable viral level of hepatitis C RNA at least 24 weeks following completion of therapy. Individuals with advanced fibrosis or cirrhosis due to hepatitis C should not be enrolled.

Individuals with a positive test for SARS-CoV-2 on the day of randomization or symptoms suggestive of SARS-CoV-2 at randomization or significant exposure to COVID-19 within 10 days prior to randomization. Individuals with COVID-19 or COVID-19 exposure can delay randomization up to a maximum of 10 days and randomize once recovered; otherwise, they will need to rescreen.

Individuals with: a. A history of more than one episode of herpes zoster and/or opportunistic infections (see Section 10.3 [Appendix 3]) in the last 12 months, with the exception of noninvasive herpes simplex at any site, oral candidiasis, vaginal candidiasis, or cutaneous fungal infections, which are permitted within the prior 12 months unless of unusual severity. Individuals with a prior history of ophthalmic herpes zoster will be excluded. b. Active infections requiring systemic treatment at the time of screening or through randomization, or history of more than 2 infections requiring IV antibiotics within 12 months prior to screening

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Population #1: - Change from baseline in European Alliance of Associations for Rheumatology Sjögren’s Syndrome Disease Activity Index (ESSDAI) at Week XX		Population #1: - Change from baseline in European Alliance of Associations for Rheumatology Sjögren’s Syndrome Disease Activity Index (ESSDAI) at Week XX
"Population #2: - Change from baseline in European Alliance of Associations for Rheumatology Sjögren’s Syndrome Patient Reported Index (ESSPRI) at Week XX"		"Population #2: - Change from baseline in European Alliance of Associations for Rheumatology Sjögren’s Syndrome Patient Reported Index (ESSPRI) at Week XX"

Secondary Outcome Measures

Name	Time	Method
Population #2: - Change from baseline in PROMIS-Fatigue SF-10a at Week XX		Population #2: - Change from baseline in PROMIS-Fatigue SF-10a at Week XX
Population #1 and #2: - PK of HZN-1116		Population #1 and #2: - PK of HZN-1116
Population #1: - Change from baseline in ESSDAI at Week XX		Population #1: - Change from baseline in ESSDAI at Week XX
Population #1: - Proportion of participants achieving ESSDAI[5] response at the Week XX and XX time points without premature discontinuation from treatment and without receiving rescue or potentially confounding therapy		Population #1: - Proportion of participants achieving ESSDAI[5] response at the Week XX and XX time points without premature discontinuation from treatment and without receiving rescue or potentially confounding therapy
Population #1: - Change from baseline in ESSPRI- and Diary for Assessing Sjögren’s Patient Reported Index (DASPRI)-pain, -fatigue, and -dryness domains at the Week XX and XX time points.		Population #1: - Change from baseline in ESSPRI- and Diary for Assessing Sjögren’s Patient Reported Index (DASPRI)-pain, -fatigue, and -dryness domains at the Week XX and XX time points.
Population #1: - Change from baseline in tender joint count (TJC) and swollen joint count (SJC) at the Week XX and XX time points		Population #1: - Change from baseline in tender joint count (TJC) and swollen joint count (SJC) at the Week XX and XX time points
Population #1: - Change from baseline in 36-item Short Form Survey (SF-36) physical component score (PCS) and mental component score (MCS) at the Week XX and XX time points		Population #1: - Change from baseline in 36-item Short Form Survey (SF-36) physical component score (PCS) and mental component score (MCS) at the Week XX and XX time points
Population #1: - Change from baseline in PROMIS-Fatigue SF-10a at Week XX and Week XX		Population #1: - Change from baseline in PROMIS-Fatigue SF-10a at Week XX and Week XX
Population #2: - Change from baseline in DASPRI at Week XX		Population #2: - Change from baseline in DASPRI at Week XX
Population #2: - Proportion of participants achieving ESSPRI[1.5] response without premature discontinuation from treatment and without receiving rescue or potentially confounding therapy at Week XX		Population #2: - Proportion of participants achieving ESSPRI[1.5] response without premature discontinuation from treatment and without receiving rescue or potentially confounding therapy at Week XX
Population #2: - Change from baseline in ESSPRI -pain, -fatigue, and -dryness domains at Week XX		Population #2: - Change from baseline in ESSPRI -pain, -fatigue, and -dryness domains at Week XX
Population #2: - Change from baseline in SF-36 PCS and MCS at Week XX		Population #2: - Change from baseline in SF-36 PCS and MCS at Week XX
Population #1 and #2: - Proportion of participants who develop anti-drug antibodies (ADA) over time		Population #1 and #2: - Proportion of participants who develop anti-drug antibodies (ADA) over time

Trial Locations

Locations (67)

ULS de Santo António, EPE - Hospital de Santo António; 🇵🇹 Porto, Portugal

Arizona Arthritis & Rheumatology Research-S Vineyard Ave; 🇺🇸 Mesa, Arizona, United States

Arizona Arthritis and Rheumatology Associates -4550 E Bell Rd; 🇺🇸 Phoenix, Arizona, United States

Neurovations Research; 🇺🇸 Napa, California, United States

Life Arc Research Centers Corp; 🇺🇸 Miami, Florida, United States

IRIS Research and Development LLC; 🇺🇸 Plantation, Florida, United States

Clinical Research of West Florida Inc - Tampa; 🇺🇸 Tampa, Florida, United States

Augusta University Medical Center-Augusta-1120 15th St; 🇺🇸 Augusta, Georgia, United States

Tufts Medical Center - 800 Washington St - PPDS; 🇺🇸 Boston, Massachusetts, United States

Pioneer Clinical Research NY; 🇺🇸 New York, New York, United States

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