Clinical trial NCT04218565: Golimumab for the Treatment of Refractory Behcet's Uveitis
Official title: Efficacy and Safety of Golimumab in the Treatment of Refractory Uveitis in Patients With Behcet's Disease
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Behcet Syndrome
Uveitis
Lead sponsor: Peking Union Medical College Hospital
Sponsor class: OTHER
First posted: 2020-01-06
Enrollment: 1
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Golimumab is approved for the treatment of several inflammatory diseases, the self-control study aims to evaluate the response to GOL in refractory BDU.
Primary purpose: TREATMENT

Brief summary: The aim of this single-center prospective study is to evaluate the efficacy and safety of Golimumab （GOL）, fully humanized anti-tumor necrosis factor (TNF)-α monoclonal antibody, in the treatment of refractory Uveitis of Behçet's disease (BDU), to verify its effects on decreasing the dose of cortical steroids, and to determine whether it can reduce BDU recurrence.

Detailed description: Uveitis of Behçet's disease (BDU) is the most common form of ocular manifestations, which is also the leading cause of irreversible visual impairment. The aim of the study is to improve the treatment strategy of the disease as well as to reduce blindness.

Monoclonal anti-TNF antibodies were recommended in 2018 recommendations for treating eye involvement affecting the posterior segment (level of evidence: IIA; strength of recommendation: B), Infliximab was mentioned for the treatment of initial or recurrent episode of acute sight-threatening uveitis.

Golimumab （GOL）is a fully-humanized anti-TNF-α monoclonal antibody, retrospective study on non-infectious uveitis and BDU has obtained promising results.

This single-center prospective study is to evaluate the efficacy and safety of GOL in the treatment of refractory BDU, to verify its effects on tapering the dose of corticosteroids, and to determine whether it can reduce BDU recurrence. We aim to enroll nine refractory BDU patients with acute onset posterior uveitis, with difficulty tapering corticosteroids while being treated with at least one of the commonly used immunosuppressants.

All participants will receive GOL monthly for six months, oral immunosuppressants will remain unchanged. During follow-up, intraocular inflammation status, BCVA, as well as extraocular manifestations, will be recorded. The primary endpoints are the efficacy of the first dose of GOL, and the recurrence of uveitis after 12 months of treatment. Secondary endpoints other than intraocular inflammation will be achieved on each follow-up visit, as the quality of life improvements, side effects, recurrence of uveitis, and corticosteroids-tapering effects.

On statistical analysis, the self-control treatment efficiency and recurrence rate difference will be determined using T-test of paired samples at a significance level of 0.05(2-sided).

To ensure the interest of the participants, our study has been reviewed by the ethics committee, and drug clinical research liability insurance was prepared.

Eligibility criteria: Inclusion Criteria:

* All participants fulfill the proposed International Criteria for BD, either ISG (International Study Group) criteria(1990) or ICBD(International Conference on Behcet's Disease) criteria(2013).
* All participants present with refractory BDU, acute onset uveitis, either posterior segment involvement or panuveitis, with difficulty tapering corticosteroids while being treated with at least one of the commonly used immunosuppressants, without severe extraocular manifestations.

Exclusion Criteria:

* Patients with impaired hepatic and renal function, other severe ocular diseases, active tuberculosis, viral hepatitis, malignancy, pregnancy, congestive heart failure, or had biologics treatment within three months will be excluded.

Primary outcomes:
measure: Efficacy of Golimumab on BD Uveitis
timeFrame: four weeks
measure: Efficacy of Golimumab on BD Uveitis
timeFrame: four weeks
measure: Efficacy of Golimumab on BD Uveitis
timeFrame: four weeks
measure: Difference of recurrence rate before and after intervention
timeFrame: 12 months
Secondary outcomes:
measure: Intraocular inflammation evaluation BOS24 index
timeFrame: each follow-up visit / every four weeks, up to six months
measure: Corticosteroid-tapering effects
timeFrame: six months
measure: Changes of uveitis recurrence
timeFrame: six months
measure: Severity of uveitis on recurrence
timeFrame: six months
measure: Severity of uveitis on recurrence
timeFrame: six months
measure: Severity of uveitis on recurrence
timeFrame: six months
measure: Impact on quality of life
timeFrame: each follow-up visit / every four weeks, up to six months
measure: Impact on quality of life
timeFrame: each follow-up visit / every four weeks, up to six months
measure: Side effects of treatment
timeFrame: six months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
Study officials:
affiliation: Department of Rheumatology, Peking Union Medical College Hospital
name: Wenjie Zheng, M.D.
role: PRINCIPAL_INVESTIGATOR
References: