RecruitingRare disease

Study on Dapagliflozin for Slowing Kidney Disease in Adolescents and Young Adults with Alport Syndrome

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a medication called Dapagliflozin on a condition known as Alport syndrome. Alport syndrome is a genetic disorder that affects the kidneys, leading to chronic kidney disease. The trial aims to see if Dapagliflozin can help slow down the progression of kidney disease in adolescents and young adults who have this condition. Dapagliflozin is a type of medication known as a sodium-glucose co-transporter-2 inhibitor, which is often used to manage blood sugar levels in people with diabetes but is being tested here for its potential benefits on kidney health.

Participants in the study will be randomly assigned to receive either Dapagliflozin or a placebo, which is a tablet that looks like the medication but does not contain the active ingredient. The study is designed to be "double-blind," meaning neither the participants nor the researchers will know who is receiving the actual medication and who is receiving the placebo. This helps ensure that the results are not influenced by expectations about the treatment. The trial will last for about 48 weeks, during which time participants will take the tablets orally and have regular check-ups to monitor their kidney function and overall health.

The main goal of the study is to determine if Dapagliflozin can prevent the worsening of kidney disease in people with Alport syndrome by reducing the amount of protein in the urine, a condition known as albuminuria. If successful, this could lead to new treatment recommendations for young people with Alport syndrome, potentially improving their quality of life by delaying the progression to more severe kidney disease. Participants will be closely monitored throughout the study to ensure their safety and to gather important data on the effects of the medication.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, eligibility is confirmed based on specific criteria. This includes having an early stage of chronic kidney disease (CKD) with a diagnosis of Alport syndrome.
Participants are adolescents aged 10 to 17 years with a specific level of albumin in the urine or adults aged 18 to 39 years with a higher level of albumin in the urine. Kidney function must be at a certain level, and a stable treatment background is required.
Step 2
Screening visit
A screening visit is conducted to ensure all criteria are met. This includes a molecular-genetic diagnosis or a diagnosis established by kidney biopsy.
Participants must have a stable treatment background with no changes in medication dosage for the three months prior to the baseline visit.
Step 3
Baseline visit
At the baseline visit, initial measurements are taken. This includes the level of albumin in the urine and kidney function.
Participants are randomly assigned to receive either a placebo or dapagliflozin.
Step 4
Medication administration
Participants take the assigned medication orally. The medication is either a placebo or dapagliflozin in the form of a tablet.
The dosage for dapagliflozin is 5 mg, taken as a film-coated tablet.
Step 5
Follow-up visits
Regular follow-up visits are scheduled to monitor progress. These visits include assessments of kidney function and the level of albumin in the urine.
The primary goal is to observe changes from the baseline over a period of 48 weeks.
Step 6
End of study
The study concludes after 52 weeks. Final assessments are conducted to evaluate the effects of the medication on kidney function and quality of life.
The study aims to determine if dapagliflozin can prevent the progression of Alport syndrome and improve quality of life.

Who can join the trial?

6 criteria

The patient must be in the early stages of **CKD** (chronic kidney disease) and have a confirmed diagnosis of **Alport syndrome** at the first visit, which is the screening.
For adolescents aged 10 to less than 18 years, they must have **albuminuria** (a condition where there is too much protein called albumin in the urine) with a **UACR** (urine albumin-to-creatinine ratio) of 300 mg/g or more.
For adults aged 18 to less than 40 years, they must have albuminuria with a UACR of 500 mg/g or more and an **eGFR** (estimated glomerular filtration rate, a test to check how well the kidneys are working) of 60 ml/min/1.73 m² or more.
The patient must have a **molecular-genetic diagnosis** (a diagnosis based on genetic testing) of any type of Alport gene variant or a diagnosis confirmed by a **kidney biopsy** (a procedure where a small piece of kidney tissue is removed and examined).
The patient must be on a stable **RAS blockade** (a type of medication that helps protect the kidneys) as part of their standard care, with no changes to the dose in the last three months before the first visit.
The patient or their guardian must provide signed and dated written informed consent, which means they agree to participate in the study after being informed about it.

Who cannot join the trial?

7 criteria

Patients who are not diagnosed with Alport syndrome cannot participate. Alport syndrome is a genetic condition that affects the kidneys, ears, and eyes.
Patients who are not within the specified age range for the study cannot participate. The study is for children and young adults.
Patients who are not able to take the study medication, such as those with allergies to the medication, cannot participate.
Patients who are currently participating in another clinical trial cannot participate in this study.
Patients with other serious health conditions that might interfere with the study cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who are unable to follow the study procedures or attend study visits cannot participate.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Charite Universitaetsmedizin Berlin KöR
Berlin, Germany
Verified
Klinikum der Universitaet Muenchen AöR
Munich, Germany
Responsive
Ludwig Maximilian University Of Munich
Munich, Germany

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Investigated drugs

Dapagliflozin is a medication used in this clinical trial to assess its effect on the progression of chronic kidney disease in patients with Alport syndrome. It belongs to a class of drugs known as sodium-glucose co-transporter-2 inhibitors (SGLT2i). The goal of using dapagliflozin in this study is to prevent the worsening of kidney function by reducing the amount of protein in the urine, which can help delay the onset of end-stage kidney failure and improve the quality of life for patients.

What is already known about the treatment

Dapagliflozin – Dapagliflozin is administered orally in tablet form. It is currently being studied in clinical trials for its potential to slow the progression of chronic kidney disease in patients with Alport syndrome, a genetic condition affecting the kidneys. The main therapeutic indication for dapagliflozin in this context is to prevent the progression of kidney damage by reducing albuminuria, which is a marker of kidney disease. At the molecular level, dapagliflozin works by inhibiting the sodium-glucose co-transporter 2 (SGLT2) in the kidneys, which reduces glucose reabsorption and promotes glucose excretion in the urine. This medication is classified as an SGLT2 inhibitor, a class of drugs primarily used to manage blood sugar levels in diabetes but now being explored for kidney protection.

Investigated diseases

Alport syndrome – Alport syndrome is a genetic condition characterized by kidney disease, hearing loss, and eye abnormalities. It primarily affects the kidneys, leading to progressive loss of kidney function. The disease is caused by mutations in genes responsible for producing type IV collagen, a protein essential for the normal function of the basement membranes in the kidneys, ears, and eyes. As the condition progresses, individuals may experience blood and protein in the urine, which can lead to chronic kidney disease. Hearing loss typically begins in childhood or early adolescence, while eye problems may include changes in the lens shape or corneal erosion. The severity and progression of symptoms can vary widely among affected individuals.