Belimumab Treatment for IgG4-related Disease
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|ClinicalTrials.gov Identifier: NCT04660565|
Recruitment Status : Recruiting
First Posted : December 9, 2020
Last Update Posted : January 8, 2021
|Condition or disease||Intervention/treatment||Phase|
|IgG4-related Disease||Drug: Prednisone and Belimumab Drug: Prednisone||Phase 4|
IgG4-RD is associated with substantial morbidity and mortality, but there is no established therapy other than GCs for an acute flare. Up to date, no randomized prospective controlled studies have been performed for this disease, and no approved therapy is available. Although GCs are widely and effectively used for treatment of initial disease and flare, they are associated with substantial toxicity which limits their long-term use. Disease flares also occur in many patients either during the GC taper or after GC discontinuation. Patients need a treatment that will more effectively control their disease and avoid GC toxicity. This study will establish the safety and tolerability of Belimumab in IgG4-RD and its ability to reduce the risk of disease flares. There are currently no medicinal products approved for the treatment of IgG4-RD. The majority of cases follow a relapsing course that can lead to permanent tissue damage with attendant morbidity and potential mortality. Glucocorticoids are widely and effectively used for treatment of initial disease and of flare, but they do not prevent recurrence of active disease after their discontinuation and are associated with substantial toxicity. Patients also continue to relapse on the off-label steroid-sparing immunosuppressive medications used by some physicians to manage patients with IgG4-RD; thus, there is a high unmet medical need for more effective therapies in this patient population.
The pathogenesis of IgG4-RD suggests that B-cell depletion may be an effective avenue for therapeutic intervention. Therapeutic depletion of B cells with rituximab reduces disease-relevant biomarkers and appears to have clinical benefit in uncontrolled, retrospective and prospective clinical studies. This study aims to define the efficacy and safety of Belimumab for the prevention of flares of this rare disease. In addition, the potential of Belimumab for minimizing GC exposure could limit the well-known adverse effects of GCs on bone, skin, muscle, adrenal gland, and eyes, and GC association with weight gain, diabetes, hypertension, and neuropsychiatric effects.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Belimumab Treatment for IgG4-related Disease, a Prospective, Open-label Clinical Trial|
|Estimated Study Start Date :||January 2021|
|Estimated Primary Completion Date :||December 2022|
|Estimated Study Completion Date :||December 2023|
Experimental: Glucocorticoid monotherapy Group
Patients treated with single glucocorticoid
Prednisone/prednisolone: started at 0.6-0. 8mg/kg.d for 2 to 4 weeks, tapered at 5mg per 1-2 weeks to equal to or less than 5mg per day in 3 months.
Other Name: Prednisone Monotherapy
Experimental: Combination therapy Group
Patients treated with Belimumab and glucocorticoid
Drug: Prednisone and Belimumab
Prednisone/prednisolone: started at 0.6-0. 8mg/kg.d for 2 to 4 weeks, tapered at 5mg per 1-2 weeks to equal to or less than 5mg per day in 3 months. Belimumab will start at the same time as the prednisone induction.
Other Name: Belimumab treatment
- Evaluate the efficacy of Belimumab in reducing the risk of flare in patients with IgG4-RD. [ Time Frame: Twelve months ]Relapse was defined as the new progress or recurrence of clinical symptoms or imaging findings with or without IgG4 level re-elevation. the new progress or recurrence of imaging findings were evaluated by MRI, CT or Ultrasound. Primary endpoint is the difference of disease relapse rate between two groups.
- The difference of time to first flare between two groups; [ Time Frame: Twelve months ]The date of disease flare is defined as the date of initiation of any flare treatment. The date of disease flare is defined as the date of initiation of any flare treatment (new or increased GC treatment, other immunotherapy, or interventional procedure) deemed necessary by the Investigator for the flare.
- The effect of Belimumab on disease activity in IgG4-RD patients. [ Time Frame: Twelve months ]The estimated treatment effect (ie, the rate ratio of Belimumab versus control), including complete response rate, partial response rate, corresponding 95% CI, and two-sided p-value for the rate ratio will be presented.
- Safety and tolerability of Belimumab in patients with IgG4-RD. [ Time Frame: Twelve months ]Side effects of Belimumab in patients with IgG4-RD will be evaluated, such as infection, hypersensitivity reactions and infusion reactions etc. Incidence of Side effects of Belimumab in patients with IgG4-RD will be recorded.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04660565
|Contact: Yunyun Fei, Dr.||+8613681125226||Feiyunyun2013@sina.com|