Phase 3 Clinical Trial with Dapagliflozin in Chronic Kidney Disease in Adolescents and Young Adult Patients
This study is for children and young adults aged 10 to 39 years who have Alport syndrome, a specific type of chronic kidney disease. It's investigating a medication called dapagliflozin, which has shown promise in slowing down kidney damage in adults. The main goal is to see if this medicine can prevent the worsening of kidney disease in younger patients, potentially delaying the need for dialysis or a kidney transplant. Researchers believe that if successful, this study could significantly improve current treatment options and the long-term health of young people living with Alport syndrome. Participants will receive either dapagliflozin or a dummy pill (placebo) alongside their usual care.
At a glance
What is this study about?
This study is all about finding a better way to help children and young adults who have a condition called Alport syndrome. This is a type of long-term kidney disease that can get worse over time, sometimes leading to the kidneys failing completely. We know that in adults with kidney problems, a medicine called dapagliflozin has been helpful in protecting their kidneys. However, we don't yet have enough information about how it works in younger people.
The main idea behind this study is to see if dapagliflozin can slow down the kidney damage caused by Alport syndrome in young patients. This could mean delaying the time when they might need drastic treatments like dialysis or a kidney transplant, and ultimately, improving their everyday lives. Currently, there aren't many specific treatments for kidney disease in children, so finding new options is really important.
Taking part in this study means you would be helping doctors understand if dapagliflozin can make a real difference for young people with Alport syndrome. If it proves to be effective, this could change how doctors treat children and young adults with this condition in the future, offering them a chance at better long-term health and a higher quality of life.
Key takeaways
- A study for young people (10-39) with Alport syndrome.
- Investigating dapagliflozin to slow down kidney disease.
- Aims to improve quality of life and delay kidney failure.
- Participants will receive either dapagliflozin or a dummy pill.
- Requires regular clinic visits for monitoring.
- Could lead to new treatments for childhood kidney disease.
Who may be eligible?
This study is looking for young people with Alport syndrome who are between 10 and 39 years old. Your kidneys should be in the early to middle stages of chronic kidney disease. Specifically, if you are aged 10 to 17, your kidney function (eGFR) should be 30 or higher, and there should be a certain amount of protein in your urine. If you are aged 18 to 39, your kidney function (eGFR) should be 60 or higher, and there should be a slightly higher amount of protein in your urine.
We need to be sure that your Alport syndrome diagnosis has been confirmed, either through a genetic test or a kidney biopsy. You also need to be taking stable blood pressure medication (called RAS blockade) as part of your usual care. Before joining, you or your parent/guardian will need to read and sign a consent form, showing you understand what the study involves.
There are also some reasons why you might not be able to join. For example, if you've recently taken certain similar medications, have very uncontrolled high blood pressure, or have a severe allergy to the study medication. Also, if you already need dialysis or have had a kidney transplant, or if you are pregnant, breastfeeding, or not using birth control, you wouldn't be able to take part.
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 39 years old?
- Do you have Alport syndrome that has been officially diagnosed?
- Are you currently taking blood pressure medicine for your kidneys?
- Is your kidney function (eGFR) at or above 30 (for 10-17s) or 60 (for 18-39s)?
- Are you free from conditions like very high blood pressure, recent similar medicine use, dialysis, or kidney transplant?
- If you are female, are you not pregnant, breastfeeding, or not using reliable contraception?
What does participation involve?
If you decide to take part, you'll be given either the study medicine, dapagliflozin, or a dummy pill (placebo). This will be alongside the blood pressure medicine you're already taking. You won't know which one you're getting, and neither will your doctor, which helps us see the true effect of the medicine. You'll need to attend regular visits at the clinic so the study team can check your health, take blood and urine samples, and record your blood pressure. These appointments will help us monitor your kidney function and see how you're responding to the treatment. The total length of your participation in the study will be discussed with you by the study team, and it involves regular follow-up over time.
Potential risks and benefits
Locations (16)
- Universitätsklinikum Heidelberg - ChildrenVerified postcodeHeidelberg, Germany· Recruiting
- LMU KlinikumVerified postcodeMünchen, Germany· Recruiting
- v. Haunersches KinderhospitalVerified postcodeMünchen, Germany· Recruiting
- Universitätsklinikum Hamburg-Eppendorf - AdultsVerified postcodeHamburg, Germany· Recruiting
- Universitätsklinikum Hamburg-Eppendorf - ChildrenVerified postcodeHamburg, Germany· Recruiting
- Clementine KinderhospitalVerified postcodeFrankfurt am Main, Germany· Recruiting
- University Medicine Goettingen - AdultsVerified postcodeGöttingen, Germany· Recruiting
- University Medicine Goettingen - Childrens HospitalVerified postcodeGöttingen, Germany· Recruiting
- Universitätsklinik Köln - AdultsVerified postcodeCologne, Germany· Recruiting
- Universitätsklinik Köln - ChildrenVerified postcodeCologne, Germany· Recruiting
- Universitätsklinik Münster - AdultsVerified postcodeMünster, Germany· Not yet recruiting
- Universitätsklinikum Münster - ChildrenVerified postcodeMünster, Germany· Recruiting
Common questions
What is Alport syndrome?
Alport syndrome is a genetic condition that causes kidney disease, and can sometimes affect hearing and vision too. It means the filters in your kidneys don't work as well as they should.
What is dapagliflozin?
Dapagliflozin is a medicine that helps the body get rid of extra sugar and salt through the urine. It has been shown to protect the kidneys in adults with certain kidney conditions.
Will I definitely get the new medicine?
No, you have a 50/50 chance. Some participants will get dapagliflozin, and others will get a placebo (a dummy pill that looks like the medicine but has no active ingredients). This helps us compare the effects.
How long will I be in the study?
The study involves regular check-ups over a period of time, which the study team will explain in detail. It's designed to track your health and treatment effects over an extended period.
Can I stop participating if I want to?
Yes, you can leave the study at any time without having to give a reason. This won't affect the care you receive from your doctors.
How to find out more
Oliver Gross, MD
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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