Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1-Mediated Proteinuric Kidney Disease
This study is looking into a new medication called VX-147, for children and adults aged 10 to 65 who have a kidney condition called APOL1-mediated proteinuric kidney disease. This type of kidney disease is linked to specific changes in a gene called APOL1. The main goals are to find out if VX-147 can effectively treat the kidney disease, if it's safe to use, and how well people tolerate it. The study will also explore how the body processes the medicine. Participants will either receive VX-147 or a placebo (a dummy medicine) to compare their effects. This is a crucial step in understanding if VX-147 could become a new treatment option.
At a glance
What is this study about?
This study is investigating a new medicine called VX-147 for a specific type of kidney disease. This condition is known as APOL1-mediated proteinuric kidney disease and affects how your kidneys filter waste. It is linked to certain changes in a gene called APOL1. Proteinuric means that your body is losing too much protein through your urine, which is a sign that the kidneys are not working as well as they should.
The main purpose of this research is to see how well VX-147 works in treating this kidney disease. The study will also closely monitor whether the medicine is safe and easy for people to take. Researchers will also learn how the body uses and processes VX-147. This is a 'Phase 2/3 adaptive study,' which means it's a later stage of research, combining two phases to get information more efficiently.
Developing new treatments is very important for conditions like APOL1-mediated proteinuric kidney disease. By understanding if VX-147 can help reduce protein loss and improve kidney function, this study aims to offer hope for better management and outcomes for those affected. Your participation could contribute significantly to medical knowledge and potentially help many others in the future.
Key takeaways
- Tests a new medicine (VX-147) for a specific kidney disease.
- Aimed at people aged 10-65 with APOL1-mediated proteinuric kidney disease.
- Checks how well the medicine works, its safety, and how the body handles it.
- Participants will receive either VX-147 or a placebo.
- Involves regular clinic visits for health checks and tests.
- You have the right to withdraw from the study at any time.
Who may be eligible?
To join this study, people generally need to be between 10 and 65 years old and have a specific gene type (G1/G1, G2/G2, or G1/G2) related to their APOL1 gene. They must also have proteinuric kidney disease, which means their kidneys are letting too much protein pass into their urine.
There are also some reasons why someone might not be able to join. For example, if you have had an organ transplant (like a kidney or bone marrow transplant), or if you have very high blood pressure that isn't under control. People with diabetes, or other known causes of kidney disease like sickle cell disease, would also not be able to take part.
If you have already started the first part of this study, you might be able to continue to the second part as long as you haven't stopped taking the study medicine permanently and your kidney disease hasn't progressed to its final stage (End Stage Kidney Disease). The study team will check all your health details and blood tests to make sure it's safe for you to participate.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you between 10 and 65 years old?
- Have you been diagnosed with proteinuric kidney disease?
- Do you have the specific APOL1 gene type (G1/G1, G2/G2, or G1/G2)?
- Have you NOT had an organ or bone marrow transplant?
- Do you NOT have uncontrolled high blood pressure or diabetes?
What does participation involve?
If you join this study, you will either receive the new medicine, VX-147, or a 'placebo.' A placebo looks like the study medicine but contains no active ingredients. This helps researchers compare the effects of the real medicine. You won't know whether you are receiving VX-147 or the placebo, and neither will your study doctor, to ensure fairness in the results.
You will need to attend regular appointments at the study clinic. These visits will involve various assessments, such as blood tests, urine tests, physical examinations, and questions about your health and how you are feeling. These tests are important for monitoring your kidney function, checking for any side effects, and seeing how your body is reacting to the treatment.
The total duration of your participation in the study, including follow-up appointments, will be clearly explained by the study team. It's important to understand that you would be expected to take the study medicine as instructed and attend all scheduled visits throughout this period. The team will provide more details on the exact timeline.
Potential risks and benefits
Locations (318)
- Alabama Kidney ResearchVerified postcodeAlabaster, United States· Recruiting
- EmVenio Research - Mobile Unit - BirminghamVerified postcodeBirmingham, United States· Recruiting
- Cardiology, P.C.Verified postcodeBirmingham, United States· Completed
- Children's Hospital of AlabamaVerified postcodeBirmingham, United States· Recruiting
- The Kirklin Clinic - NephrologyVerified postcodeBirmingham, United States· Recruiting
- Nephrology AssociatesVerified postcodeFairhope, United States· Recruiting
- Heart Center Research LLCVerified postcodeHuntsville, United States· Recruiting
- Nephrology Consultants, LLCVerified postcodeHuntsville, United States· Recruiting
- Renal Associates of Alabama, LLCVerified postcodeMontgomery, United States· Recruiting
- Arizona Kidney Disease and Hypertension Center - Banner Desert OfficeVerified postcodeMesa, United States· Withdrawn
- Arizona Kidney Disease and Hypertension Center - Thomas OfficeVerified postcodePhoenix, United States· Recruiting
- Phoenix Children's Hospital, Inc. - NephrologyVerified postcodePhoenix, United States· Recruiting
Common questions
What is proteinuric kidney disease?
It's a condition where your kidneys let too much protein leak into your urine, which can be a sign that your kidneys are not working properly.
What does 'APOL1-mediated' mean?
This refers to kidney disease that is linked to specific changes or variations in a gene called APOL1.
Will I definitely get the new medicine?
You might get the new medicine (VX-147) or a placebo (a dummy medicine) to compare the effects. You and your doctor won't know which one you are getting.
How long will I be in the study?
The exact length of time will be explained by the study team, including how long you'll take the medicine and attend follow-up appointments.
Can I leave the study at any time?
Yes, you are free to withdraw from the study at any point without it affecting your medical care.
How to find out more
Medical Information
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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