Behcet SyndromePhase 2NCT01693653
Tocilizumab for the Treatment of Behcet's Syndrome
A Phase 2 study testing Tocilizumab in people with Behcet Syndrome, run by NYU Langone Health at 1 site in United States.
Trial summary
This is a double-blind placebo controlled study targeting individuals with active Behcet's Syndrome who have oral ulcers and are resistant (have not responded after 4 weeks) to conventional treatments. Maximum allowable dose of colchicine (0.6mg twice a day) and stable dose for 4 weeks before enrollment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Behcet Syndrome
- What is being tested?
- Tocilizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2012to Nov 2013
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Any patient with BS with at least 1 active oral ulcer resistant (have not responded after 4 weeks to colchicine or local measures.
- dose (maximum allowable 0.6mg twice a day ) stable for 4 weeks and prednisone or equivalent (maximum dose < 10mg/day) stable for 6 weeks prior to enrollment.
- Patients must have Behcet's syndrome based on International Study Group criteria.
- Patients will be included in the trial based on the following criteria:
- Normal organ function, except if abnormal due to the disease under investigation such as mucocutaneous involvement or joint involvement.
- Men and women of reproductive potential must agree to use an acceptable method of birth control during treatment and for twelve months after completion of treatment.
- Subject has provided written informed consent.
Who cannot join
- Patients with eye, CNS, vascular involvement such as DVT, thrombosis, or aneurysms.
- Patients who are currently being treated or have been exposed in the last 3 months to other immunosuppressive medications (azathioprine, TNF inhibitors, methotrexate, mycophenolate mofetil). Patients who are currently being treated or have been exposed to Cyclosporine or cyclophosphamide in the past 6 months will be excluded).
- Major surgery (including joint surgery) within 8 weeks prior to screening or planned major surgery within 6 months following randomization.
- Treatment with any investigational agent within 4 weeks (or 5 half-lives of the investigational drug, whichever is longer) of screening.
- Previous treatment with any cell-depleting therapies, including investigational agents or approved therapies, some examples are CAMPATH, anti-CD4, anti-CD5, anti¬CD3, anti-CD19 and anti-CD20 (please note exceptions above).
- Treatment with intravenous gamma globulin, plasmapheresis or Prosorba column within 6 months of baseline.
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- Immunization with a live/attenuated vaccine within 4 weeks prior to baseline.
- Previous treatment with TCZ (an exception to this criterion may be granted for single dose exposure upon application to the sponsor on a case-by-case basis).
- Any previous treatment with alkylating agents such as chlorambucil, or with total lymphoid irradiation.
- History of severe allergic or anaphylactic reactions to human, humanized or murine monoclonal antibodies.
- Evidence of serious uncontrolled concomitant cardiovascular, nervous system, pulmonary (including obstructive pulmonary disease), renal, hepatic, endocrine (include uncontrolled diabetes mellitus) or gastrointestinal disease (including complicated diverticulitis, ulcerative colitis, or Crohn's disease.)
- Current liver disease as determined by principal investigator unless related to primary disease under investigation
- Known active current or history of recurrent bacterial, viral, fungal, mycobacterial or other infections (including but not limited to tuberculosis and atypical mycobacterial disease, Hepatitis B and C, and herpes zoster, but excluding fungal infections of nail beds).
- Any major episode of infection requiring hospitalization or treatment with IV antibiotics within 4 weeks of screening or oral antibiotics within 2 weeks prior to screening.
- Active TB requiring treatment within the previous 3 years. Patients should be screened for latent TB and, if positive, treated following local practice guidelines prior to initiating TCZ. Patients treated for tuberculosis with no recurrence in 3 years are permitted. (Appendix 8)
- Primary or secondary immunodeficiency (history of or currently active) unless related to primary disease under investigation.
- Evidence of active malignant disease, malignancies diagnosed within the previous 10 years (including hematological malignancies and solid tumors, except basal and squamous cell carcinoma of the skin or carcinoma in situ of the cervix uteri that has been excised and cured), or breast cancer diagnosed within the previous 20 years unless related to primary disease under investigation.
- Pregnant women or nursing (breast feeding) mothers.
- Patients with reproductive potential not willing to use an effective method of contraception.
- History of alcohol, drug or chemical abuse within 1 year prior to screening.
- Neuropathies or other conditions that might interfere with pain evaluation unless related to primary disease under investigation.
- Patients with lack of peripheral venous access.
- Laboratory Exclusion criteria (at screening):
- Serum creatinine > 1.6 mg/dL (141 µmol/L) in female patients and > 1.9 mg/dL (168 µmol/L) in male patients. Patients with serum creatinine values exceeding limits may be eligible for the study if their estimated glomerular filtration rates (GFR) are >30.
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 1.5 times upper limit of normal (ULN)
- Total Bilirubin > ULN
- Platelet count < 100 x 109/L (100,000/mm3)
- Hemoglobin < 85 g/L (8.5 g/dL; 5.3 mmol/L)
- White Blood Cells < 3.0 x 109/L (3000/mm3)
- Absolute Neutrophil Count < 2.0 x 109/L (2000/mm3)
- Absolute Lymphocyte Count < 0.5 x 109/L (500/mm3)
- Positive Hepatitis BsAg, or Hepatitis C antibody
Trial locations and contacts
1 site in 1 country.
United States1 site
NYU Center for Musculoskeletal Care
New York, New York, 10016
About this study
This is a double-blind placebo controlled study targeting individuals with active Behcet's Syndrome who have oral ulcers and are resistant (have not responded after 4 weeks) to conventional treatments. Maximum allowable dose of colchicine (0.6mg twice a day) and stable dose for 4 weeks before enrollment. Prednisone or equivalent (< 10mg/day) permitted if dose stable for 6 weeks prior to enrollment.
The study will investigate the safety of tocilizumab for this vasculitic condition in addition to its efficacy.
Read more
The planned sample size is 30 participants per arm for a total of 60 participants. The study would be for 3 months, with a safety follow up at 2 months after study termination.
Study participants will stay on their current treatments and either tocilizumab or placebo infusions will be given every 4 weeks in addition. Patients will be randomized to Actemra IV 8mg/kg Q 4 weeks X 3 doses or placebo.
Read the detailed description
Behcet's syndrome is a vasculitis that causes oral and genital ulcerations, skin lesions, eye disease and arthritis, in addition to vascular complications with thrombophlebitis, thrombosis and rarely central nervous system involvement. IL-6 activity has been suggested in the pathogenesis in some studies. Tocilizumab with its unique mode of action among biologic agents may be a good candidate in this orphan disease.
- Conditions
- Behcet Syndrome
- Being tested
- Tocilizumab
- Keywords
- Behcet's syndrome, oral ulcers
- Medical subject headings
- Behcet Syndrome, Oral Ulcer
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TocilizumabDrug
Also called actemra
Intravenous infusions every 4 weeks for 3 doses.
| Study group | Role | What they receive |
|---|---|---|
| Tocilizumab | Active comparator | tocilizumab infusion every 4 weeks over 3 months Receives: Drug: Tocilizumab |
| Placebo | Placebo comparator | placebo infusion 0.9% sodium chloride every 4 weeks over 3 months Receives: Drug: Tocilizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Outcome
The study was terminated. No data were collected for this Outcome Measure.
Measured: 9 months
Secondary outcomes (9)
Genital Ulcers
The study was terminated. No data were collected for this outcome measure.
Measured: 9 months
Oral Ulcers
The study was terminated. No data were collected for this outcome measure.
Measured: 9 months
Treatment Failures
The study was terminated. No data were collected for this outcome measure.
Measured: 9 months
Oral Ulcer Pain
The study was terminated. No data were collected for this outcome measure.
Measured: 9 months
Gential Ulcer Pain
The study was terminated. No data were collected for this outcome measure.
Measured: 9 months
BSAS
The study was terminated. No data were collected for this outcome measure.
Measured: 9 months
MDHAQ
The study was terminated. No data were collected for this outcome measure.
Measured: 9 months
BDCAF
The study was terminated. No data were collected for this outcome measure.
Measured: 9 months
Safety
The study was terminated. No data were collected for this outcome measure.
Measured: 9 moths
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Sep 2012 (estimated)
- Study startSep 2012
- Primary completionNov 2013
- Study completionNov 2013
Why it stopped: low enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Behcet SyndromeStudies of the same condition, open and under way.
- More trials run by NYU Langone HealthThe sponsor's other registered studies.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- Yusuf Yazici, MD, Principal investigator, NYU Langone Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01693653
- Sponsor's protocol ID
- 12-01182
- Status last verified
- Feb 2019
- First submitted
- 19 Sep 2012
- Last updated
- 15 Feb 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tocilizumab for the Treatment of Behcet's Syndrome
- ClinicalTrials.gov ID
- NCT01693653
- Acronym / sponsor ID
- 12-01182
- Conditions
- Behcet Syndrome
- Keywords
- Behcet's syndrome, oral ulcers
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: low enrollment
- Enrolment
- 1 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- Sep 2012
- Primary completion
- Nov 2013 (actual)
- Study completion
- Nov 2013 (actual)
- First posted
- 26 Sep 2012 (estimated)
- Results
- Posted 2 Jun 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a double-blind placebo controlled study targeting individuals with active Behcet's Syndrome who have oral ulcers and are resistant (have not responded after 4 weeks) to conventional treatments. Maximum allowable dose of colchicine (0.6mg twice a day) and stable dose for 4 weeks before enrollment. Prednisone or equivalent (< 10mg/day) permitted if dose stable for 6 weeks prior to enrollment.
The study will investigate the safety of tocilizumab for this vasculitic condition in addition to its efficacy.
The planned sample size is 30 participants per arm for a total of 60 participants. The study would be for 3 months, with a safety follow up at 2 months after study termination.
Study participants will stay on their current treatments and either tocilizumab or placebo infusions will be given every 4 weeks in addition. Patients will be randomized to Actemra IV 8mg/kg Q 4 weeks X 3 doses or placebo.
Detailed description
Behcet's syndrome is a vasculitis that causes oral and genital ulcerations, skin lesions, eye disease and arthritis, in addition to vascular complications with thrombophlebitis, thrombosis and rarely central nervous system involvement. IL-6 activity has been suggested in the pathogenesis in some studies. Tocilizumab with its unique mode of action among biologic agents may be a good candidate in this orphan disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Tocilizumab | Active comparator | Drug: Tocilizumabtocilizumab infusion every 4 weeks over 3 months |
| Placebo | Placebo comparator | Drug: Tocilizumabplacebo infusion 0.9% sodium chloride every 4 weeks over 3 months |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Any patient with BS with at least 1 active oral ulcer resistant (have not responded after 4 weeks to colchicine or local measures.
- dose (maximum allowable 0.6mg twice a day ) stable for 4 weeks and prednisone or equivalent (maximum dose < 10mg/day) stable for 6 weeks prior to enrollment.
- Patients must have Behcet's syndrome based on International Study Group criteria.
- Normal organ function, except if abnormal due to the disease under investigation such as mucocutaneous involvement or joint involvement.
- Men and women of reproductive potential must agree to use an acceptable method of birth control during treatment and for twelve months after completion of treatment.
Key exclusion criteria (32 listed)
- Patients with eye, CNS, vascular involvement such as DVT, thrombosis, or aneurysms.
- Patients who are currently being treated or have been exposed in the last 3 months to other immunosuppressive medications (azathioprine, TNF inhibitors, methotrexate, mycophenolate mofetil). Patients who are currently being treated or have been exposed to Cyclosporine or cyclophosphamide in the past 6 months will be excluded).
- Major surgery (including joint surgery) within 8 weeks prior to screening or planned major surgery within 6 months following randomization.
- Treatment with any investigational agent within 4 weeks (or 5 half-lives of the investigational drug, whichever is longer) of screening.
- Previous treatment with any cell-depleting therapies, including investigational agents or approved therapies, some examples are CAMPATH, anti-CD4, anti-CD5, anti¬CD3, anti-CD19 and anti-CD20 (please note exceptions above).
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 |
|---|---|---|
| TocilizumabAlso: actemraIntravenous infusions every 4 weeks for 3 doses. | Drug | Placebo, Tocilizumab |
Primary outcome
Primary Outcome
Time frame: 9 months
The study was terminated. No data were collected for this Outcome Measure.
9 secondary outcomes
Genital Ulcers
Time frame: 9 months
Oral Ulcers
Time frame: 9 months
Treatment Failures
Time frame: 9 months
Oral Ulcer Pain
Time frame: 9 months
Gential Ulcer Pain
Time frame: 9 months
BSAS
Time frame: 9 months
MDHAQ
Time frame: 9 months
BDCAF
Time frame: 9 months
Safety
Time frame: 9 moths
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Feb 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Behcet Syndrome
RecruitingPhase 3
A Study of Apremilast in Children With Oral Ulcers Associated With Behçet's Disease or Juvenile Psoriatic Arthritis
Behçet's Disease, Juvenile Psoriatic Arthritis
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Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
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Serum EBI-3 Protein Levels in Behçet's Disease
Behçet Disease
Not yet recruitingPhase 2
Corticotropin for Behçet's Disease: A Multicenter Randomized Controlled TrialAcronym.
Behçet's Syndrome, Behçet's Syndrome (BS)
RecruitingPhase 2
Baricitinib in the Treatment of Intestinal Behçet's Syndrome
Behcet Syndrome, Intestinal Type
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.
Yusuf Yazici, MD principal investigator
NYU Langone Medical Center
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 in people with Behcet Syndrome, run by NYU Langone Health at 1 site in United States.
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 7 inclusion and 32 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 15 Feb 2019. 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 United States. 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 2 Jun 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Primary Outcome (time frame: 9 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Feb 2019.