Pilot Trial of Ustekinumab for Primary Sjögren's Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 8 July 2024
• Main ID: NCT04093531
• Date of registration: 05/08/2019
• Prospective Registration: Yes
• Primary sponsor: University of Rochester
• Public title: Pilot Trial of Ustekinumab for Primary Sjögren's Syndrome
• Scientific title: Pilot Trial of Ustekinumab for Primary Sjögren's Syndrome
• Date of first enrolment: January 15, 2020
• Target sample size: 12
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT04093531
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Ummara Shah, MD
• Affiliation: Assistant Professor of Medicine

Key inclusion & exclusion criteria

A subject who has met all of the following criteria is eligible for participation in the
study:

- Has provided written informed consent

- Between the ages of 18-75 years (inclusive)

- Body weight = 40 kg

- Meets the 2016 ACR EULAR criteria (score >4)

- 3 points- Labial salivary gland with focal lymphocytic sialadenitis and focus
score of >1 foci/4 mm2‡

- 3 Points- Anti-SSA/Ro positive

- 1 Point- Ocular Staining Score >5 in at least 1 eye

- 1 Point- Schirmer's test <5 mm/5 minutes in at least 1 eye

- 1 Point- Unstimulated whole saliva flow rate <0.1 ml/minute

- If taking prednisone (or equivalent corticosteroid), the dose must be = 10 mg/day
and stable for at least 4 weeks prior to baseline visit

- If taking hydroxychloroquine, the dose must be stable for at least 12 weeks prior to
baseline.

- If taking a cholinergic stimulant (e.g. pilocarpine, cevimeline), the dose must be
stable for at least 4 weeks prior to baseline.

- If a male of reproductive potential, must agree to practice two highly effective
forms of contraception during the study (one of which must be a barrier method) and
be able to continue contraception for 20 weeks after his last dose of study agent
Subject must also agree not to donate sperm up to 20 weeks after his last dose of
study agent.

- If a female of childbearing potential, must agree to practice two highly effective
forms of contraception during the study (one of which must be a barrier method) and
able to continue contraception for 20 weeks after her last dose of study agent.

A subject who meets any of the following criteria is disqualified from participation in
the study:

- Has a chronic or persistent infection that might be worsened by immunosuppressive
treatment (e.g., HIV, hepatitis B, hepatitis C, or tuberculosis).

- History of untreated TB or positive QuantiFERON TB-Gold during screening period. If
a subject has previously received an adequate course of therapy for either latent (9
months of isoniazid in a locale where rates of primary multi-drug resistant TB
infection are <5%) or active TB infection, a QuantiFERON TB-Gold test need not be
obtained, but a chest radiograph or other appropriate image must still be obtained
if not done so within the prior 3 months.

- History of recurrent significant infections or occurrence of a serious local
infection (e.g., cellulitis, abscess) or systemic infection (e.g., pneumonia,
septicemia) within twelve weeks prior to Day 0.

- Active symptomatic infection within two weeks prior to Day 0.

- Receipt of live vaccine within four weeks prior to Day 0.

- History or presence of primary or secondary immunodeficiency.

- History of any life-threatening allergic reactions to pilocarpine or any components
of ustekinumab. Pilocarpine will be used to stimulate salivary flow in order to
assess flow rate.

- Is currently pregnant or nursing.

- Concurrent use of anticholinergic agents, such as tricyclic antidepressants,
antihistamines, phenothiazines, antiparkinsonian drugs, anti-asthmatic medications,
or gastrointestinal (GI) medications that cause xerostomia in more than 10% of
patients.

- Treatment with any of the following within the defined period prior to the screening
and Day 0 visits:

- 12 months for rituximab

- 24 weeks for cyclophosphamide

- 8 weeks for azathioprine, cyclosporine, methotrexate, and mycophenolate mofetil

- 4 weeks for intravenous immunoglobulin

- 4 weeks for etanercept

- 8 weeks for adalimumab

- 12 weeks for infliximab

- 8 weeks Golimumab

- 8weeks Certolizumab pegol

- 16 weeks Abatacept

- 4 weeks Tocilizumab SQ

- 16 weeks Tocilizumab IV

- 4 weeks Tofacitinib and Tofacitinib XR

- Prednisone (or equivalent corticosteroid) > 10 mg/day.

- A definite diagnosis of RA, SLE, systemic sclerosis, or dermatomyositis.

- A history of alcohol or substance abuse.

- A history of head and neck radiation therapy, sarcoidosis, or graft-versus-host
disease.

- A history of malignancy, except for a resected basal or major squamous cell
carcinoma, cervical dysplasia, or in situ cervical cancer Grade I, within the last
five years.

- Abnormal laboratory results for the following parameters at the baseline visit:

- Absolute neutrophil count (ANC): < 1500/mm3

- Platelets: < 100,000/mm3

- Hemoglobin: < 9 grams (g)/deciliter (dL)

- Serum creatinine: = 2.0 mg/dL

- AST: > 1.5x upper limit of normal

- ALT: > 1.5x upper limit of normal.

- A psychiatric disorder rendering the subject incapable of providing informed
consent.

- Plans for foreign travel to countries other than Canada or Western Europe within the
treatment period.

- Inability or unwillingness to follow the protocol

- Any condition or treatment that, in the opinion of the investigator, places the
subject at an unacceptable risk as a participant in the trial.


Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Primary Sjögren Syndrome

Intervention(s)

Drug: Ustekinumab

Primary Outcome(s)

Change in The European League Against Rheumatism (EULAR) Sjogren's Syndrome Patient Reported Index (ESSPRI) Score From Baseline to Week 24 [Time Frame: baseline to 24 weeks]

Secondary Outcome(s)

Change in Mean Serum Biomarkers of Inflammation From Baseline to Week 24 [Time Frame: baseline to 24 weeks]

Change in the Short Form Health Survey (SF-36) Patient Reported Outcome Measure Between Day 0 and Week 24 [Time Frame: baseline to 24 weeks]

Total Score of the EULAR Sjogren's Syndrome Disease Activity Index (ESSDAI) [Time Frame: 24 weeks.]

Secondary ID(s)

Ustekinumab for PSS

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Janssen, LP

Results

• Results available: Yes
• Date Posted: 25/06/2024
• URL: https://clinicaltrials.gov/ct2/show/results/NCT04093531