idiopathic retroperitoneal fibrosis MedDRA version: 20.1 Level: LLT Classification code 10021244 Term: Idiopathic retroperitoneal fibrosis System Organ Class: 100000004856
Conditions
Interventions
Trade Name: ROACTEMRA - 20 MG/ML CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO (VETRO) 4ML 1 FLACONCINO
Product Name: TOCILIZUMAB
Product Code: N.A.
Pharmaceutical Form: Concen
Sponsors
AZIENDA OSPEDALIERO-UNIVERSITARIA DI PARMA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Written signed Informed consent - patients with clinically active IRF, who have refractory disease: disease unresponsive to standard glucocorticoid therapy (initial dose 1 mg/kg/d) +/- conventional immunosuppressants (MTX, MMF). - patients with clinically active IRF who previously reported serious adverse events related to the use of standard-dose glucocorticoids with or without common immunosuppressive drugs -age 18-80 years - Men and women of reproductive potential must agree to use a highly efficient means of contraception during the study. Such means include: a) intrauterine device (IUD) b) hormonal methods (such methods were used for at least three months prior to study entry and that they are used with a condom in the male partner); c) Essure micro-insert system ; d)complete abstinence from intercourse of reproductive potential; e) for male patients with female partners of childbearing potential, use of condoms is required. Female partners of male study subjects are asked to select one of the above methods. 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 12 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 6
Exclusion criteria
Exclusion criteria: -Major surgery (including joint surgery) within 8 weeks prior to screening or planned major surgery within 6 months following enrolment. -secondary forms of retroperitoneal fibrosis -Treatment-naïve patients -Treatment with the 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 include: CAMPATH, anti-CD4, anti-CD5, anti-CD3, anti-CD19 and anti-CD20. -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). -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.) - Any history of recent serious bacterial, viral, fungal, or other opportunistic infections. -Primary or secondary immunodeficiency (history of or currently active) unless related to primary disease under investigation. -Any medical or psychological condition that in the opinion of the principal investigator would interfere with safe completion of the trial. - History of other malignancy within 5 years prior to screening, except for appropriately treated carcinoma in situ of the cervix, non-melanoma skin carcinoma, or Stage I uterine cancer. - 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: to assess the rate of remission at the end of treatment (month 6). Remission denotes absence of disease-related symptoms, resolution or improvement of hydronephrosis (without indwelling stents), near-normalization (i.e. normalization or reduction to <30% of baseline values) of erythrocyte sedimentation rate and C-reactive protein levels, as previously described; prednisone dose must be =5 mg/die for the definition of remission ;Secondary Objective: • to assess the rate of remission at the end of month 12 • to assess percentage reduction in IRF thickness on CT/MRI and of FDG uptake on PET scans • to assess treatment-related toxicity throughout the study period ;Primary end point(s): - We will assess the remission rate at month 6, defined as the proportion of treated patients who achieve and maintain remission at the end of month 6 (=end of the treatment period). Remission is a composite end-point which includes absence of disease-related symptoms, resolution or improvement of hydronephrosis (without indwelling stents), near-normalization (i.e. normalization or reduction to <30% of basal values) of erythrocyte sedimentation rate and C-reactive protein levels, as described in a previous randomised controlled trial; prednisone dose must be =5 mg/die for the definition of remission. We expect that at least 60% of the treated patients will experience remission by the end of treatment;Timepoint(s) of evaluation of this end point: 6 months (stop treatment) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - We will assess the remission rate at month 12, defined as the proportion of treated patients who are in remission at the end of the 12th month of study (=6 months after the end of treatment). Remission is a composite end-point which includes absence of disease-related symptoms, resolution or improvement of hydronephrosis (without indwelling stents), near-normalization (i.e. normalization or reduction to <30% of basal values) of erythrocyte sedimentation rate and C-reactive protein levels, as described in a previous randomised controlled trial; prednisone dose must be =5 mg/die for the definition of remission. - Reduction in IRF thickness will be assessed as the percentage reduction in maximal peri-aortic or peri-iliac thickness of the retroperitoneal tissue (end of treatment vs baseline) on CT or MRI, as previously described - Reduction in FDG uptake will be assessed as percentage reduction in maximum standardized uptake value (SUVmax) on FDG-PET (end of treatment vs baseline) - In order to assess treatment-related toxicity or other types of adverse events, we will record all adverse events occurred during the study and divide them in two periods, the 6 months of treatment and the subsequent 6 months of follow-up. ;Timepoint(s) of evaluation of this end point: 12 months (rate remission ant drug toxicity), 6 months (reduction up-take of PET-TC) | — |
Countries
Italy
Contacts
Public ContactSegreteria Comitato Etico
Az. Ospedaliero-Universitaria di Parma
Outcome results
None listed