Tocilizumab for the Treatment of Behcet's Syndrome

NCT01693653 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: 12-01182
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Behcet Syndrome

Keywords

Behcet's syndrome; oral ulcers

Outcome Measures

Primary Outcomes (1)

• Primary Outcome

  The study was terminated. No data were collected for this Outcome Measure.

  9 months

Secondary Outcomes (9)

• Genital Ulcers

  9 months

• Oral Ulcers

  9 months

• Treatment Failures

  9 months

• Oral Ulcer Pain

  9 months

• Gential Ulcer Pain

  9 months

Study Arms (2)

Tocilizumab

ACTIVE COMPARATOR

tocilizumab infusion every 4 weeks over 3 months

Drug: Tocilizumab

Placebo

PLACEBO COMPARATOR

placebo infusion 0.9% sodium chloride every 4 weeks over 3 months

Drug: Tocilizumab

Interventions

Tocilizumab DRUG

Intravenous infusions every 4 weeks for 3 doses.

Also known as: actemra

Placebo; Tocilizumab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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)

Sponsors & Collaborators

• NYU Langone Health: lead
• Genentech, Inc.: collaborator

Study Sites (1)

NYU Center for Musculoskeletal Care

New York, New York, 10016, United States

Location

MeSH Terms

Conditions

Behcet Syndrome; Oral Ulcer

Interventions

tocilizumab

Condition Hierarchy (Ancestors)

Mouth Diseases; Stomatognathic Diseases; Uveitis, Anterior; Panuveitis; Uveitis; Uveal Diseases; Eye Diseases; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vascular

Results Point of Contact

• Title: Yusuf Yazici
• Organization: NYU Langone Medical Center, Center for Musculoskeletal Care

yusuf.yazici@nyumc.org

646-501-7400

Study Officials

• Yusuf Yazici, MD

  NYU Langone Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 19, 2012
• First Posted: September 26, 2012
• Study Start: September 1, 2012
• Primary Completion: November 1, 2013
• Study Completion: November 1, 2013
• Last Updated: February 15, 2019
• Results First Posted: June 2, 2016

Record last verified: 2019-02

Locations

US (1)