Behcet SyndromePhase 2NCT03554161
Tocilizumab for the Treatment of Refractory Behcet's Uveitis
A Phase 2 study testing Tocilizumab (TCZ) in people with Behcet Syndrome and Uveitis, run by Wenjie Zheng at 1 site in China.
Trial summary
The aim of this single-center prospective study is to evaluate the efficacy and safety of Tocilizumab (TCZ), humanized monoclonal antibody against IL-6receptor, 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… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Behcet Syndrome, Uveitis
- What is being tested?
- Tocilizumab (TCZ)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteChina
- Study dates
- 10 May 2018to 1 Feb 2021
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- All participants fulfill the the proposed International Criteria for BD, either ISG criteria(1990) or ICBD 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.
Who cannot join
- Patients with impaired hepatic and renal function, other severe ocular diseases, active tuberculosis, viral hepatitis, malignancy, pregnancy will be excluded.
Trial locations and contacts
1 site in 1 country.
China1 site
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
About this study
Official title: Efficacy and Safety of Tocilizumab in the Treatment of Refractory Uveitis in Patients With Behcet's Disease
The aim of this single-center prospective study is to evaluate the efficacy and safety of Tocilizumab (TCZ), humanized monoclonal antibody against IL-6receptor, 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.
Read the 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 treatment strategy of the disease as well as to reduce blindness.
Tocilizumab (TCZ) is a humanized antibody against IL-6 receptor, clinical trials on non-infectious uveitis and retrospective study on BDU has obtained expected results.
This single-center prospective study is to evaluate the efficacy and safety of TCZ in the treatment of refractory BDU, to verify its effects on decreasing the dose of corticosteroids, and to determine whether it can reduce BDU recurrence.The investigators aim to enroll nine refractory BDU patients with acute onset uveitis, with difficulty tapering corticosteroids while being treated with at least one of the commonly used immunosuppressants. All participants will receive TCZ , oral corticosteroids and immunosuppressants will remain unchanged, intraocular injection will be avoided. During monthly follow-up, the investigators will measure and record intraocular inflammation status，as well as extraocular manifestations and the acute-phase reactants. The participants will continue to receive TCZ for six months, the primary end point is the efficacy four weeks after first dose of TCZ, secondary end points other than intraocular inflammation will be achieved on each follow-up visit, as 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, the study has been reviewed by the PUMCH ethics committee, and drug clinical research liability insurance was prepared.
- Conditions
- Behcet Syndrome, Uveitis
- Being tested
- Tocilizumab (TCZ)
- Keywords
- Tocilizumab, Refractory uveitis
- Medical subject headings
- Behcet Syndrome, Uveitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tocilizumab (TCZ)Biological
Patients with refractory BD associated uveitis will receive Tocilizumab therapy, the efficacy and corticosteroids-tapering effects will be evaluated.
| Study group | Role | What they receive |
|---|---|---|
| Tocilizumab for refractory BDU | Experimental | This study is a self-control study and all the participants will be enrolled in the interventional arm. Receives: Biological: Tocilizumab (TCZ) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of Tocilizumab on BD Uveitis
Remission or deterioration of uveitis，based on intraocular inflammation evaluation
Measured: four weeks
Secondary outcomes (7)
Intraocular inflammation evaluation BOS24 index
Visual acuity, anterior chamber cells, vitreous opacities, retinal inflammation, macular thickness, etc.
Measured: each follow-up visit / every four weeks, up to six months
Corticosteroid-tapering effects
Whether corticosteroid dosage could be tapered to minimum dose.
Measured: six months
Reduction of uveitis recurrence
Differences of recurrence between before and after treatment
Measured: each follow-up visit / every four weeks, up to six months
Severity of uveitis on recurrence
Differences of severity of recurrence between before and after treatment
Measured: each follow-up visit / every four weeks, up to six months
Improvement of quality of life
Record quality of life on questionnaire: BDCAF
Measured: each follow-up visit / every four weeks, up to six months
Improvement of quality of life
Record quality of life on questionnaire: SF-36
Measured: each follow-up visit / every four weeks, up to six months
Side effects of treatment
Record any side effects during intervention
Measured: each follow-up visit / every four weeks, up to six months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jun 2018
- Study start10 May 2018
- Primary completion1 Feb 2021
- Study completion1 Feb 2021
Why it stopped: The study was terminated based on its primary and secondary outcome measures in three consecutive patients.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Behcet SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Tocilizumab is approved for the treatment of several inflammatory disease, this study tends to evaluate the response to TCZ in refractory BDU, thus it is a self-control study.
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wenjie Zheng
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Wenjie Zheng, Peking Union Medical College Hospital
- Investigators
- Wenjie Zheng, M.D., Principal investigator, Department of Rheumatology, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03554161
- Sponsor's protocol ID
- TCZ-BDU-PUMCH
- Status last verified
- Mar 2021
- First submitted
- 21 Apr 2018
- Last updated
- 22 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Atienza-Mateo B, Calvo-Rio V, Beltran E, Martinez-Costa L, Valls-Pascual E, Hernandez-Garfella M, Atanes A, Cordero-Coma M, Miquel Nolla J, Carrasco-Cubero C, Loricera J, Gonzalez-Vela MC, Vegas-Revenga N, Fernandez-Diaz C, Demetrio-Pablo R, Dominguez-Casas LC, Luis Martin-Varillas J, Palmou-Fontana N, Hernandez JL, Gonzalez-Gay MA, Blanco R. Anti-interleukin 6 receptor tocilizumab in refractory uveitis associated with Behcet's disease: multicentre retrospective study. Rheumatology (Oxford). 2018 May 1;57(5):856-864. doi: 10.1093/rheumatology/kex480.
PubMed 29471416Lopalco G, Fabiani C, Sota J, Lucherini OM, Tosi GM, Frediani B, Iannone F, Galeazzi M, Franceschini R, Rigante D, Cantarini L. IL-6 blockade in the management of non-infectious uveitis. Clin Rheumatol. 2017 Jul;36(7):1459-1469. doi: 10.1007/s10067-017-3672-z. Epub 2017 May 20.
PubMed 28528519
Study at a glance
From the registry record, last updated by the sponsor on 22 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Tocilizumab in the Treatment of Refractory Uveitis in Patients With Behcet's Disease
- ClinicalTrials.gov ID
- NCT03554161
- Acronym / sponsor ID
- TCZ-BDU-PUMCH
- Conditions
- Behcet Syndrome, Uveitis
- Keywords
- Tocilizumab, Refractory uveitis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was terminated based on its primary and secondary outcome measures in three consecutive patients.
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Wenjie ZhengUniversity, hospital or other
- Study start
- 10 May 2018 (actual)
- Primary completion
- 1 Feb 2021 (actual)
- Study completion
- 1 Feb 2021 (actual)
- First posted
- 13 Jun 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this single-center prospective study is to evaluate the efficacy and safety of Tocilizumab (TCZ), humanized monoclonal antibody against IL-6receptor, 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 treatment strategy of the disease as well as to reduce blindness.
Tocilizumab (TCZ) is a humanized antibody against IL-6 receptor, clinical trials on non-infectious uveitis and retrospective study on BDU has obtained expected results.
This single-center prospective study is to evaluate the efficacy and safety of TCZ in the treatment of refractory BDU, to verify its effects on decreasing the dose of corticosteroids, and to determine whether it can reduce BDU recurrence.The investigators aim to enroll nine refractory BDU patients with acute onset uveitis, with difficulty tapering corticosteroids while being treated with at least one of the commonly used immunosuppressants. All participants will receive TCZ , oral corticosteroids and immunosuppressants will remain unchanged, intraocular injection will be avoided. During monthly follow-up, the investigators will measure and record intraocular inflammation status，as well as extraocular manifestations and the acute-phase reactants. The participants will continue to receive TCZ for six months, the primary end point is the efficacy four weeks after first dose of TCZ, secondary end points other than intraocular inflammation will be achieved on each follow-up visit, as 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, the study has been reviewed by the PUMCH ethics committee, and drug clinical research liability insurance was prepared.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Tocilizumab is approved for the treatment of several inflammatory disease, this study tends to evaluate the response to TCZ in refractory BDU, thus it is a self-control study.
| Arm | Type | Receives |
|---|---|---|
| Tocilizumab for refractory BDU | Experimental | Biological: Tocilizumab (TCZ)This study is a self-control study and all the participants will be enrolled in the interventional arm. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- All participants fulfill the the proposed International Criteria for BD, either ISG criteria(1990) or ICBD 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.
Key exclusion criteria (1 listed)
- Patients with impaired hepatic and renal function, other severe ocular diseases, active tuberculosis, viral hepatitis, malignancy, pregnancy will be excluded.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Tocilizumab (TCZ)Patients with refractory BD associated uveitis will receive Tocilizumab therapy, the efficacy and corticosteroids-tapering effects will be evaluated. | Biological | Tocilizumab for refractory BDU |
Primary outcome
Efficacy of Tocilizumab on BD Uveitis
Time frame: four weeks
Remission or deterioration of uveitis，based on intraocular inflammation evaluation
7 secondary outcomes
Intraocular inflammation evaluation BOS24 index
Time frame: each follow-up visit / every four weeks, up to six months
Corticosteroid-tapering effects
Time frame: six months
Reduction of uveitis recurrence
Time frame: each follow-up visit / every four weeks, up to six months
Severity of uveitis on recurrence
Time frame: each follow-up visit / every four weeks, up to six months
Improvement of quality of life
Time frame: each follow-up visit / every four weeks, up to six months
Improvement of quality of life
Time frame: each follow-up visit / every four weeks, up to six months
Side effects of treatment
Time frame: each follow-up visit / every four weeks, up to six months
Results and safety
The study was stopped early (reason given: The study was terminated based on its primary and secondary outcome measures in three consecutive patients.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Mar 2021.
- Results publication: PubMed 29471416Atienza-Mateo B, Calvo-Rio V, Beltran E, Martinez-Costa L, Valls-Pascual E, Hernandez-Garfella M, Atanes A, Cordero-Coma M, Miquel Nolla J, Carrasco-Cubero C, Loricera J, Gonzalez-Vela MC, Vegas-Revenga N, Fernandez-Diaz C, Demetrio-Pablo R, Dominguez-Casas LC, Luis Martin-Varillas J, Palmou-Fontana N, Hernandez JL, Gonzalez-Gay MA, Blanco R. Anti-interleukin 6 receptor tocilizumab in refractory uveitis associated with Behcet's disease: multicentre retrospective study. Rheumatology (Oxford). 2018 May 1;57(5):856-864. doi: 10.1093/rheumatology/kex480.
- Results publication: PubMed 28528519Lopalco G, Fabiani C, Sota J, Lucherini OM, Tosi GM, Frediani B, Iannone F, Galeazzi M, Franceschini R, Rigante D, Cantarini L. IL-6 blockade in the management of non-infectious uveitis. Clin Rheumatol. 2017 Jul;36(7):1459-1469. doi: 10.1007/s10067-017-3672-z. Epub 2017 May 20.
- SpringerlinkLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Wenjie Zheng, M.D. principal investigator
Department of Rheumatology, Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Tocilizumab (TCZ) in people with Behcet Syndrome and Uveitis, run by Wenjie Zheng at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 22 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: The study was terminated based on its primary and secondary outcome measures in three consecutive patients.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of Tocilizumab on BD Uveitis (time frame: four weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03554161, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Mar 2021.