Tocilizumab for the Treatment of Familial Mediterranean Fever

Phase 1

• Conditions: Adult patients with Familial Mediterranean Fever, who have active disease; MedDRA version: 20.0Level: PTClassification code 10016207Term: Familial mediterranean feverSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2016-004505-13-DE
• Lead Sponsor: niversity Hospital Tuebingen

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-08-21
• Last Update Posted: 5 July 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

-Age = 18 years and written informed consent
-FMF according to the Tel Hashomer Criteria (Appendix); with at least one heterozygous or homozygous mutation of the MEFV gene
-Inadequate response or intolerance to colchicine
-Attack during the last 12 weeks, defined as episodes of fever and/or pericarditis and/or serositis and/or testis involvement and/or arthritis and/or erysipelas-like rash and
-CRP >0.5mg/dl and/or ESR >20mm/h and/or SAA >10mg/dl
-Physician Global Assessment (PGA) >2
-Able and willing to provide written informed consent and to comply with the study protocol

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Subjects presenting with any of the following criteria will not be included in the trial:
-Age < 18 years
-Major surgery within 8 weeks prior to screening or planned major surgery within 12 months after randomization
-Transplanted organs (except corneal transplant performed more than 3 months prior to screening)
Exclusions Related to Prior or Concomitant Therapy
-Previous treatment with TCZ
-Treatment with glucocorticosteroide > 10mg/day within 1 week; prednisolone = 10mg/day can be given on a stable dose throughout the study
-Analgesic medication other than paracetamol or ibuprofen or diclofenac, which can be used at a stable dose and for treatment of FMF attacks.
-Treatment with any investigational agent within 12 weeks (or 5 half-lives of the investigational drug, whichever is longer) of screening
-Treatment with Anakinra within the last 1 week prior to baseline (ptb), Canakinumab within the last 8 week prior to baseline
-Treatment with etanercept within 2 weeks; certolizumab pegol, abatacept or adalimumab within 6 weeks; golimumab and infliximab within 8 weeks ptb
-Rituximab within 24 weeks ptb
-Leflunomide within 12 weeks ptb (washout possible),
-azathioprine, cyclophosphamide within 12 weeks ptb
-Immunization with a live/attenuated vaccine within = 4 weeks ptb
-Previous treatment with cell-depleting therapies, including investigational agents or approved therapies: anti-CD33, anti-CD52, anti-CD4, anti-CD5, anti- CD3 and anti-CD19
-Treatment with intravenous gamma globulin within 6 months of baseline
-Treatment with plasmapheresis within 6 months of baseline
-Any previous treatment with alkylating agents such as chlorambucil, or with total lymphoid irradiation
Exclusions Related to General Safety
-History of severe allergic or anaphylactic reactions to human, humanized, or murine antibodies
-Evidence of serious uncontrolled concomitant cardiovascular, nervous system, pulmonary (including obstructive pulmonary disease), renal, hepatic, psychiatric or GI disease
-History of diverticulitis, diverticulosis requiring antibiotic treatment, or chronic ulcerative lower GI disease such as Crohn's disease, ulcerative colitis, or other symptomatic lower GI conditions that might predispose a patient to perforations
-Known active current or history of recurrent bacterial, viral, fungal, mycobacterial, or other infections (including but not limited to tuberculosis [TB] and atypical mycobacterial disease, hepatitis B and C, and herpes zoster, but excluding fungal infections of the 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 of screening
-Active TB requiring treatment within the previous 3 years; Patients should be screened for latent TB and, if positive, treated according to local practice guidelines prior to initiating TCZ treatment; Patients treated for TB with no recurrence within 3 years and patients treated for latent TB within 3 years are eligible.
-Primary or secondary immunodeficiency (history of or currently active)
-Evidence of malignant disease or malignancies diagnosed within the previous 5 years (except basal and squamous cell carcinoma of the skin or carcinoma in situ of the cervix uteri that have been excised and cured)
-Females of childbearing potential who are not willing to use an effective method of contraception, such as condom, sterilization, or true abstinence throughout stu

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified