What is the mechanism of action of this treatment?

The most common treatments for Alport Syndrome primarily aim to reduce proteinuria and slow the progression of kidney damage. Angiotensin-converting enzyme (ACE) inhibitors and angiotensin II receptor blockers (ARBs) are frequently used to lower blood pressure and reduce proteinuria by decreasing the pressure within the glomeruli, thereby minimizing kidney damage. These treatments are crucial for Alport Syndrome patients as they help to preserve kidney function and delay the onset of end-stage renal disease. The R3R01 trial likely explores additional or alternative therapies that further target proteinuria and kidney pathology, potentially offering new avenues to manage this genetic disorder more effectively.

What side effects are associated with this type of intervention?

Common treatments for Alport Syndrome, particularly those aimed at reducing proteinuria and addressing kidney pathology, include ACE inhibitors, ARBs, and immunosuppressive therapies. ACE inhibitors and ARBs are generally well-tolerated but can cause side effects such as hyperkalemia, hypotension, and renal function deterioration. Immunosuppressive therapies, such as corticosteroids and cyclophosphamide, can lead to a range of adverse effects including increased infection risk, liver enzyme elevation, gastrointestinal distress, and bone marrow suppression. Monitoring and managing these side effects are crucial for patient safety.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved drugs exclusively for Alport Syndrome. However, treatments commonly used to manage the condition include angiotensin-converting enzyme (ACE) inhibitors and angiotensin II receptor blockers (ARBs), which help reduce proteinuria and slow the progression of kidney disease. These treatments are similar to the ones being studied in the trial R3R01, which aims to address uncontrolled proteinuria in Alport Syndrome patients.

What other types of research exist?

Promising novel alternatives to R3R01 for Alport Syndrome patients include TRF-budesonide, which has shown efficacy in reducing proteinuria and stabilizing eGFR in clinical trials. Additionally, calcineurin inhibitors (CNIs) like cyclosporine and tacrolimus, used in combination with glucocorticoids, have demonstrated higher rates of complete remission of proteinuria in small studies. These treatments represent potential options for managing proteinuria and kidney pathology in Alport Syndrome.

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Unknown

R3R01 for Alport Syndrome

Phase 2

Recruiting

Research Sponsored by River 3 Renal Corp.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

For countries that are enrolling pediatric patients: 12 to 75 years old at the time of signing the informed consent. For countries that are not enrolling pediatric patients: 18 to 75 years old at the time of signing the informed consent.

ACEi/ARB therapy at maximum tolerated dose stable for at least 4 weeks prior to screening. ACEi/ARB dose should remain stable over the course of the study.

Must not have

Patient has had an organ transplant, is currently on an organ transplant waiting list or there is a reasonable possibility that the patient will have an organ transplant in the 6 months after screening.

Focal Segmental Glomerulosclerosis Patients Exclusion Criteria (in addition): Patient has collapsing variant of FSGS on renal biopsy.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial tests R3R01, a new drug that lowers fat in kidney cells to improve kidney function and reduce damage. It targets patients with Alport Syndrome and steroid-resistant FSGS who have high protein levels in their urine because their conditions are not improving with current treatments. Another drug has shown promise in reducing protein levels and kidney damage in animal studies.

Who is the study for?

This trial is for patients with Alport Syndrome or Focal Segmental Glomerulosclerosis who have uncontrolled proteinuria despite ACE/ARB therapy. Participants must be willing to follow study procedures and use contraception. Exclusions include uncontrolled diabetes, hypertension, recent investigational drug use, organ transplant recipients, active infections including COVID-19, and certain medication restrictions.

What is being tested?

The trial is testing R3R01's safety, tolerability, efficacy, and how the body processes it in patients with Alport Syndrome or Focal Segmental Glomerulosclerosis. It's an open-label Phase 2 study where all participants will receive the experimental drug without a placebo comparison.

What are the potential side effects?

Potential side effects of R3R01 are not specified here but typically could include reactions at the injection site if applicable, gastrointestinal symptoms like nausea or diarrhea, potential kidney function changes given the patient population involved in this study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 12 and 75 years old, or I am between 18 and 75 if my country does not enroll children.

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I have been on a stable dose of ACEi/ARB for at least 4 weeks.

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My kidney disease (FSGS) is confirmed by biopsy or linked to a genetic mutation.

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My kidney function is within the required range.

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My kidney function is good based on a specific test.

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I am at least 12 years old in a country enrolling children, or 18 otherwise.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had an organ transplant, am waiting for one, or might get one in the next 6 months.

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I do not have the collapsing variant of FSGS according to my kidney biopsy.

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I am currently taking bardoxolone, rituximab, cyclophosphamide, abatacept, or sparsentan.

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I haven't taken any experimental drugs in the last 30 days or 5 half-lives.

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I have a genetic condition that affects how my body processes certain sugars.

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I do not have any active or uncontrolled infections, including COVID.

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I am HIV positive.

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My FSGS is caused by another health condition like obesity or an autoimmune disorder.

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I haven't taken Rituximab, cyclophosphamide, or abatacept in the last 120 days.

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I am using or agree to use effective birth control if I can have children.

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I have not had cancer, except for treated skin cancer, in the last 5 years.

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I only have Alport Syndrome and no other kidney diseases.

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My blood pressure is not controlled and is very high.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Assess change in urine creatinine protein ratio

Incidence of adverse events (Safety and Tolerability)

Secondary study objectives

Change in quality-of-life assessment from baseline to end of treatment and to the end of the follow-up period by cohort for adults

Change in quality-of-life assessment from baseline to end of treatment and to the end of the follow-up period by cohort for children

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 3 (Focal Segmental Glomerulosclerosis Patients)Experimental Treatment1 Intervention

R3R01 administered orally as 200 mg tablets twice daily for the 84 days.

Group II: Cohort 2 (Alport Syndrome Patients)Experimental Treatment1 Intervention

R3R01 administered orally as 200 mg tablets twice daily for 84 days.

0 / 19Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Alport Syndrome primarily aim to reduce proteinuria and slow the progression of kidney damage. Angiotensin-converting enzyme (ACE) inhibitors and angiotensin II receptor blockers (ARBs) are frequently used to lower blood pressure and reduce proteinuria by decreasing the pressure within the glomeruli, thereby minimizing kidney damage. These treatments are crucial for Alport Syndrome patients as they help to preserve kidney function and delay the onset of end-stage renal disease. The R3R01 trial likely explores additional or alternative therapies that further target proteinuria and kidney pathology, potentially offering new avenues to manage this genetic disorder more effectively.

Can immunosuppressive drugs slow the progression of IgA nephropathy?Neonatal nephrotic presentation of a child with heterozygous NPHS1 mutation.