Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.
This research study is looking at a new medicine, iptacopan, for patients aged 12 to 60 who have a kidney condition called C3 Glomerulopathy (C3G). C3G causes inflammation in the kidneys, which can lead to damage over time. The study wants to find out if iptacopan is safe and effective in slowing down or stopping this kidney damage. Half of the participants will receive iptacopan, and the other half will receive a dummy pill (placebo). Researchers will compare kidney function in both groups to understand if the new medicine works better than current treatments. This is a large, important study (Phase 3) hoping to make iptacopan available if it proves successful.
De un vistazo
¿De qué trata este estudio?
This study is investigating a new medication called iptacopan for people with a kidney disease known as C3 Glomerulopathy, or C3G. C3G occurs when part of your immune system, called the 'complement system,' becomes overactive and starts attacking healthy kidney tissue. This can lead to inflammation and damage in your kidneys, potentially causing them to not work as well as they should.
The main goal of this study is to see if iptacopan can help protect the kidneys and improve their function in people with C3G. Researchers will compare iptacopan to a placebo (a dummy pill with no active medicine) to understand if it's safe and truly effective. They will look at things like protein levels in the urine and how well the kidneys are filtering waste, as well as checking if the medicine helps stabilise kidney function over time. If successful, iptacopan could become an important new treatment option for C3G patients.
This study is a 'Phase 3' trial, which means it's a large and important step before a new medicine can be approved for wider use. It involves many medical centres and patients, and the results will be crucial in deciding if iptacopan can help people living with C3G. For some adult participants, kidney biopsies will also be performed to see if the medicine leads to physical improvements in the kidney tissue itself.
Puntos clave
- Tests a new medicine, iptacopan, for C3 Glomerulopathy (C3G).
- Compares iptacopan to a dummy pill (placebo).
- Aims to check if iptacopan is safe and improves kidney function.
- For people aged 12-60 with a C3G diagnosis.
- Requires regular clinic visits and tests.
- Participation is voluntary, and you can withdraw anytime.
¿Quién puede ser elegible?
To join this study, you would generally need to be between 12 and 60 years old and have a confirmed diagnosis of C3G from a recent kidney biopsy. For adults, this biopsy must have been within the last year, and for teenagers, within the last three years.
Before taking part, you must have been on a stable dose of certain blood pressure medications (like ACE inhibitors or ARBs) for at least 90 days. Other kidney-protecting medications you might be taking should also have been at a stable dose for the same period. Your kidney function, as measured by blood and urine tests, needs to be within certain limits, and your C3 levels (a specific protein related to your condition) should be lower than normal.
You would not be able to join if you have had an organ transplant, including a kidney transplant. You also wouldn't be eligible if you have a very rapidly worsening form of kidney inflammation, severe scarring in your kidneys (more than 50%), or certain blood conditions like 'monoclonal gammopathy of undetermined significance' (MGUS). You must also have received specific vaccinations to protect against infections before starting the study medicine.
¿Podría este estudio ser adecuado para ti?
Responde estas preguntas rápidas para ver si puedes ser elegible. Esto es solo una guía: el equipo de investigación toma la decisión final.
- Are you between 12 and 60 years old?
- Do you have a confirmed diagnosis of C3G?
- Are you currently on stable doses of your regular kidney medications?
- Have you had the required vaccinations?
- Have you NOT had an organ transplant?
- Do your kidney function tests meet certain requirements?
¿En qué consiste la participación?
If you decide to take part in this study, you would be randomly assigned to either receive the new medicine, iptacopan, or a placebo (a dummy pill). Neither you nor your doctor would know which one you are receiving – this is called 'double-blind.' You would continue to take your regular, stable kidney medications alongside the study treatment.
The study would involve regular visits to the clinic for medical check-ups, blood tests, and urine tests to monitor your kidney function and general health. For adult participants, there would also be a kidney biopsy at the start and potentially another one during the study to check for changes in your kidney tissue. You would also need to make sure you've had specific vaccinations to protect against certain infections before starting the study medicine. The total duration of your participation, including follow-up, would be decided by the study doctors.
Riesgos y beneficios potenciales
Locations (87)
- Childrens Hospital ColoradoCódigo postal verificadoAurora, Estados Unidos· Reclutamiento
- Nicklaus Childrens HospitalCódigo postal verificadoMiami, United States· Reclutamiento
- Georgia Nephrology Research InstCódigo postal verificadoLawrenceville, United States· Completado
- IN University School of MedCódigo postal verificadoIndianápolis, Estados Unidos· Retirado
- University of Iowa Health CareCódigo postal verificadoIowa City, Estados Unidos· Reclutamiento
- University of Iowa Health CareCódigo postal verificadoIowa City, Estados Unidos· Reclutamiento
- Johns Hopkins HospitalCódigo postal verificadoBaltimore, Estados Unidos· Retirado
- Brigham and Womens Hosp Harvard Med SchoolCódigo postal verificadoBoston, Estados Unidos· Retirado
- Hackensack Uni Medical CenterCódigo postal verificadoHackensack, Estados Unidos· Retirado
- Albany Medical CenterCódigo postal verificadoAlbany, United States· Retirado
- Col Uni Med Center New York PresbyCódigo postal verificadoNueva York, Estados Unidos· Completado
- Baylor Scott and White ResearchCódigo postal verificadoTemple, United States· Retirado
Preguntas comunes
What is C3 Glomerulopathy (C3G)?
C3G is a rare kidney disease where a part of your immune system becomes overactive and damages the filters in your kidneys, which can lead to kidney problems.
What is iptacopan, and how might it help?
Iptacopan is a new medicine being tested to see if it can calm down the overactive immune system in C3G, potentially slowing down or preventing further kidney damage.
¿Qué es un 'placebo'?
A placebo is a dummy pill that looks exactly like the study medicine but doesn't contain any active drug. This helps researchers compare the real medicine's effects accurately.
¿Sabré si estoy recibiendo el medicamento real o el placebo?
No, neither you nor your doctors will know if you're receiving iptacopan or the placebo. This is to ensure the study results are unbiased.
Are there any extra costs involved for me to participate?
Generally, clinical trials cover the costs of the study medication, related tests, and clinic visits. You should discuss any potential personal costs, such as travel, with the study team.
Cómo obtener más información
Novartis Farmacéutica
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
¿Interesado en participar?
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