In short
There are currently 2 ongoing clinical trials investigating new treatment approaches for cryoglobulinaemia. Both trials are being conducted in France and focus on different medications aimed at controlling blood vessel inflammation and reducing abnormal proteins in the blood.
Clinical trial locations
- France
Study of Belimumab versus Placebo for Treatment of Non-infectious Active Cryoglobulinemia Vasculitis in Adult Patients
This trial is evaluating a medication called belimumab for treating people with active cryoglobulinemia vasculitis. The condition occurs when abnormal proteins in the blood cause inflammation of blood vessels throughout the body, affecting organs such as the skin, joints, kidneys, nerves, and heart.
Main inclusion criteria: Participants must be at least 18 years old with active mixed cryoglobulinemia vasculitis affecting at least one organ system. They must have already received rituximab treatment within the past 6 weeks and have blood tests showing specific antibodies and protein markers. Women of childbearing age must agree to use effective birth control during the study and for 92 days afterward, with monthly pregnancy testing required. Participants must test negative for HIV and hepatitis B or meet specific monitoring requirements.
Main exclusion criteria: People who have previously received belimumab or other biologic therapies within 6 months are not eligible. The trial excludes those with active hepatitis B or C infections, severe allergic reactions to biological medications, active tuberculosis, or other serious infections. Pregnant or breastfeeding women cannot participate. Those with cancer within the past 5 years (except successfully treated non-melanoma skin cancer), severe kidney disease requiring dialysis, drug or alcohol abuse within the past year, or unstable heart disease are also excluded.
Focus of the trial: The study aims to determine whether belimumab is effective in treating non-infectious active cryoglobulinemia vasculitis. Participants will receive rituximab therapy first, followed by either belimumab injections (200 mg under the skin) or placebo. The treatment includes corticosteroids for the first 12 weeks, which are then stopped completely. The main evaluation occurs at week 25, with continued monitoring until week 48. Doctors will assess how well the treatment works in clearing abnormal proteins from the blood and reducing inflammation, while also tracking any side effects.
Investigational drug: Belimumab is a monoclonal antibody that targets and reduces specific immune system cells causing inflammation and blood vessel damage. It works by inhibiting B-lymphocyte stimulator, a protein that helps develop abnormal B cells producing autoantibodies. While already used to treat lupus, this trial studies its potential for controlling the overactive immune response in vasculitis.
Study of Isatuximab for Patients with Type I Cryoglobulinemia
This clinical trial investigates isatuximab as a treatment for Type I Cryoglobulinemia, a rare blood disorder where abnormal proteins can cause symptoms affecting the skin, joints, kidneys, and other organs. The medication is given as an intravenous infusion directly into the bloodstream.
Main inclusion criteria: Participants must be over 18 years old and provide written informed consent. They must have monoclonal gammopathy of unknown significance with a specific type of protein called monoclonal IgG, along with active cryoglobulinemia vasculitis affecting organs like skin, joints, kidneys, lungs, heart, muscles, digestive system, and nervous system. The trial accepts both newly diagnosed patients and those experiencing a return of the condition after previous treatment. Participants must be affiliated with the National French social security system and agree to use effective contraception during the study and for at least 5 months after the last dose. They must have negative test results for HIV, Hepatitis B, and Hepatitis C.
Main exclusion criteria: The trial excludes people with other serious medical conditions that might interfere with the study, pregnant or breastfeeding women, and those who have had cancer in the past 5 years (except non-melanoma skin cancer). People with active infections requiring treatment, those who received another investigational drug within the last 30 days, or those with known allergies to the study medication are not eligible. The trial also excludes individuals with severe heart disease, uncontrolled high blood pressure, severe liver or kidney disease, or a history of drug or alcohol abuse within the last year.
Focus of the trial: The study evaluates how well isatuximab works in treating symptoms and achieving a complete clinical response, meaning symptoms improve significantly. Participants receive the medication through intravenous infusion according to a specific protocol. Regular monitoring occurs at scheduled visits at weeks 4, 12, and 20, with comprehensive evaluation at week 20 to assess improvement in vasculitis symptoms and clearance of cryoglobulinemia. A follow-up phase continues for up to 48 weeks to monitor long-term effects and potential relapses.
Investigational drug: Isatuximab is a monoclonal antibody being studied for its potential to help people with Type I cryoglobulinemia. It works by targeting specific cells in the immune system that may be involved in the disease process. The goal is to reduce or eliminate symptoms, leading to a complete clinical response where symptoms are significantly reduced or gone.
Summary
Both ongoing trials for cryoglobulinaemia are being conducted exclusively in France, suggesting a concentration of research efforts in this country for this rare condition. The two studies focus on different aspects of the disease: one targets mixed cryoglobulinemia vasculitis with belimumab following rituximab therapy, while the other investigates Type I cryoglobulinemia with isatuximab. Both investigational drugs are monoclonal antibodies that work by targeting specific immune system components, representing similar therapeutic approaches but for different subtypes of the condition. The trials employ comprehensive monitoring protocols extending up to 48 weeks, reflecting the need for long-term assessment of treatment effectiveness and safety in this complex disease.
