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Alport SyndromePhase 2NCT05267262

Study to Evaluate R3R01 in Patients With Alport Syndrome and Patients With Focal Segmental Glomerulosclerosis

A Phase 2 study testing R3R01 in people with Alport Syndrome and Focal Segmental Glomerulosclerosis, run by River 3 Renal Corp. at 22 sites in 6 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a Phase 2, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis Read more about this study

Who is it for?
Ages 12 years and older
What is being tested?
R3R01
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
43 participants
Where
22 sitesin 6 countries
Study dates
15 Jun 2022to 19 Aug 2025
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • All Patients:
  • Patient is able to communicate well with the investigator, understands and is willing to comply with all requirements of the study, and understands and signs the written informed consent form (ICF).
  • For children to be eligible, one or both parents/legal guardians must sign a parental permission form which provides information contained in the ICF. Children capable of assent must express their willingness to participate by signing an assent form.
  • If patient has received a COVID vaccination, the baseline visit must occur at least one week or more after the second/booster vaccination.
  • Patients who have had active symptoms of COVID within 3 months prior to screening and are now asymptomatic for the last 2 weeks but have tested COVID PCR positive. If a patient is asymptomatic at screening but is COVID positive, then rescreening can occur after a minimum of two weeks.
  • Both female patients, as well as, female partners of male patients who are of child-bearing potential must be willing to not become pregnant for the complete duration of the study (>180 days) (90 days after the last dose of study medication).
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  • Males (including sterilized subjects) whose female partners have child-bearing potential, must agree to use male contraception (condoms) during the period from the time of signing the informed consent form (ICF) through 90 days after the last dose of study drug. They must agree to immediately inform the investigator if their partner becomes pregnant during the study.
  • Alport Syndrome Patients Inclusion Criteria (in addition):
  • Males and females with X-Linked AS and males and females with autosomal inherited AS.
  • For countries that are enrolling pediatric patients: patients from age 12 years and older.
  • For countries that are not enrolling pediatric patients: patients from age 18 years and older.
  • Confirmed diagnosis of AS by genetic testing and /or kidney biopsy. For patients enrolled in the US who meet all inclusion and exclusion criteria but have not had their diagnosis confirmed by genetic testing or kidney biopsy, the Sponsor will provide for patient's genetic testing.
  • UPCR ≥1.0 g/g.
  • eGFR ≥ 30 mL/min/1.73m2 (using CKD-EPI equation for adults and Bedside Schwartz equation for children).
  • ACEi/ARB therapy at maximum tolerated dose stable for at least 4 weeks prior to enrollment and during the study.
  • Focal Segmental Glomerulosclerosis Patients Inclusion Criteria (in addition):
  • Male or female patients,
  • 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
  • Primary FSGS, (without any identifiable cause, and where the FSGS is confirmed by renal biopsy) or FSGS where there is documentation of a genetic mutation in a podocyte protein associated with FSGS.
  • If on steroids, the dose should remain stable for at least 4 weeks prior to enrollment and during the study. Subjects who are steroid-resistant, defined as failure to achieve partial or complete remission, or subjects who experienced adverse events without acceptable clinical benefit after at least 8 weeks of adequate corticosteroid therapy for children and 12 weeks for adults are eligible.
  • UPCR between 1.5g/g and 12.0g/g.
  • eGFR > 30 mL/min/1.73m2 (using CKD-EPI equation for adults and Bedside Schwartz equation for children).
  • If taking concomitant ACEi and/or ARB treatment, it should remain at a stable dose for at least 4 weeks prior to enrollment and during the study.

Who cannot join

  • All Patients:
  • Uncontrolled diabetes mellitus as evidenced by an HbA1c ≥ 11%. For Germany: HbA1c ≥ 8.5%.
  • Uncontrolled hypertension
  • Adults: (SBP ≥ 180mmHg and/or DBP ≥ 100mmHg). For Germany: (SBP ≥ 140mmHg and/or DBP ≥ 100mmHg).
  • Children: ≥ 95th percentile or ≥ 130/80 mm Hg, whichever is lower
  • Moderate or severe hepatic impairment (Child-Pugh B or C), except if (a) decreased serum albumin is directly related to the renal disease (resulting in a Child Pugh score of 7), and (b) no other Child-Pugh Score parameters are increased and (c) patient has no liver pathology in medical history.
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  • Presence of any active (i.e., with symptoms) and/or uncontrolled infection (including COVID).
  • Presence of Human immunodeficiency virus (HIV).
  • BMI > 40. Note - For Germany: BMI > 35 (Obesity Class II).
  • History of malignancy other than treated basal cell or squamous cell skin cancer within the past 5 years.
  • History of alcohol abuse in the last 5 years or currently drinks in excess of 21 and 14 units per week for males and females, respectively.
  • Received an investigational agent within 30 days or 5 half-lives prior to screening (whichever is longer).
  • History of non-compliance such that patient is unlikely to be compliant with study visits, procedures or drug administration.
  • 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.
  • Participation in an interventional trial within the previous 3 months prior to screening or concurrent participation in a research trial.
  • Patient is not suitable to participate in the study for any reason (including, but not limited to co-morbidities, history of non-compliance with study visits, procedures, or drug administration) in the opinion of the investigator.
  • Females of childbearing potential (those who are not surgically sterilized or post-menopausal for at least 1 year) are excluded from participation in the study unless they agree to use highly effective contraception.
  • Females that are lactating.
  • History of hypersensitivity to study drug and/or any of its excipients.
  • Patients with hereditary galactose intolerance, total lactase deficiency or glucose-galactose malabsorption.
  • Required concomitant use of bardoxolone, rituximab, cyclo-phosphamide, abatacept, or sparsentan
  • Alport Syndrome Patients Exclusion Criteria (in addition):
  • Kidney disease apart from AS, e.g. diabetic nephropathy or lupus nephritis.
  • Use of Bardoxolone or sparsentan treatment in the 30 days prior to screening. SGLT2 inhibitors are allowed if the patient is on a stable dose for at least 3 months prior to enrollment and during the study.
  • Focal Segmental Glomerulosclerosis Patients Exclusion Criteria (in addition):
  • Patient has collapsing variant of FSGS on renal biopsy.
  • Patient has FSGS secondary to another condition (e.g. obesity, cardiovascular, infectious, or autoimmune disorder).
  • Use of Rituximab, cyclophosphamide or abatacept treatment in the 120 days prior to screening. If taking other chronic immune-modulatory medications that are small molecules, the dosage must be stable for 4 weeks prior to screening and during the study.
  • If previous Rituximab treatment is greater than 120 days from screening, CD20 cell count should be within normal limits.
  • If previous other antibody treatment on a stable dose is greater than 120 days from screening, the investigator must deem administration of study drug to be safe.
  • Use of sparsentan in the 30 days prior to screening. SGLT2 inhibitors are allowed if the patient is on a stable dose for at least 3 months prior to enrollment and during the study.

Trial locations and contacts

22 sites in 6 countries.

22 sites in 6 countries

United States14 sites

  • Investigative site

    Ann Arbor, Michigan, 48109-5718

  • Investigative site

    Atlanta, Georgia, 30322

  • Investigative site

    Boca Raton, Florida, 33431

  • Investigative site

    Boston, Massachusetts, 02111

  • Investigative Site

    Cary, North Carolina, 27511

  • Investigative site

    Cleveland, Ohio, 44195

  • Investigative site

    Columbus, Ohio, 43235

  • Investigative Site

    Dallas, Texas, 75204

  • Investigative site

    East Providence, Rhode Island, 02914

  • Investigative site

    Houston, Texas, 77054

  • Investigative Site

    Los Angeles, California, 90022

  • Investigative site

    Miami, Florida, 33136

  • Investigative site

    Minneapolis, Minnesota, 55454

  • Investigative site

    Riverview, Florida, 33578

Other trials in United States

Belgium2 sites

  • Investigative site

    Brussels, 1200

  • Investigative site

    Liège, 4000

Other trials in Belgium

Netherlands2 sites

  • Investigative site

    Amsterdam, 1105AZ

  • Investigative site

    Nijmegen, 6525 GA

Other trials in Netherlands

United Kingdom2 sites

  • Investigative site

    Leicester, LE5 4PW

  • Investigative site

    Nottingham, NG5 1PB

Other trials in United Kingdom

France1 site

  • Investigative site

    Paris, 75015

Other trials in France

Germany1 site

  • Investigative site

    Göttingen, 37075

Other trials in Germany

About this study

Official title: A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in AS Patients With Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients With Primary Steroid-Resistant FSGC

This is a Phase 2, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

Read the detailed description

R3R01 is investigational small molecule designed to decrease fat levels in certain cells in the kidney and therefore may improve kidney function and reduce damage in the kidney. This is a single arm open-label study enrolling patients in three cohorts. Cohort 1 will include 5 adult (≥18 y/o) patients from Cohorts 2 and 3 (including at least one patient from Cohort 2 and at least one patient from Cohort 3). Cohort 2 will include approximately 20 male and female patients from 12 years and older with X-linked Alport Syndrome (AS), and male and female patients with autosomal inherited AS. Cohort 3 will include approximately 30 male and female patients from age 12 to 75 years with a biopsy proven diagnosis who present with primary steroid-resistant focal segmental glomerulosclerosis (FSGS) with proteinuria.

All eligible patients will be enrolled to receive R3R01 over a treatment period of 12 weeks with a primary efficacy outcome as the percentage change in proteinuria from baseline to the end of treatment (Day 84) in each cohort as a whole

Being tested
R3R01
Keywords
FSGS, Kidney Disease, glomeruli
Medical subject headings
Nephritis, Hereditary, Glomerulosclerosis, Focal Segmental, Kidney Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • R3R01Drug

    R3R01 administered orally for 12 weeks

Study groups in NCT05267262
Study groupRoleWhat they receive
Cohort 2 (Alport Syndrome Patients)ExperimentalR3R01 administered orally as 200 mg tablets twice daily for 84 days.
Receives: Drug: R3R01
Cohort 3 (Focal Segmental Glomerulosclerosis Patients)ExperimentalR3R01 administered orally as 200 mg tablets twice daily for the 84 days.
Receives: Drug: R3R01

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of adverse events (Safety and Tolerability)

    Safety and tolerability as determined by the incidence of adverse events (AEs)

    Measured: 12 weeks

  2. Assess change in urine creatinine protein ratio

    Change from baseline in urine creatinine protein ratio for Cohort 1 (Alport Syndrome patient group) and Cohort 2(Focal Segmental Glomerulosclerosis patient group).

    Measured: 12 weeks

Secondary outcomes (2)

  1. 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 as measured by the Short Form SF-36 for adults

    Measured: 24 weeks

  2. 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

    Change in quality of life as measured by the pediatric quality of life inventory (PedsQL) for children

    Measured: 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Mar 2022
  2. Study start15 Jun 2022
  3. Primary completion21 May 2025
  4. Study completion19 Aug 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
43 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05267262
Sponsor's protocol ID
R3R01-ASFSGS-201
Status last verified
Sep 2025
First submitted
23 Feb 2022
Last updated
29 Sep 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Meliambro K, He JC, Campbell KN. Podocyte-targeted therapies - progress and future directions. Nat Rev Nephrol. 2024 Oct;20(10):643-658. doi: 10.1038/s41581-024-00843-z. Epub 2024 May 9.

    PubMed 38724717

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing R3R01 in people with Alport Syndrome and Focal Segmental Glomerulosclerosis, run by River 3 Renal Corp. at 22 sites in 6 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 24 inclusion and 31 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 29 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 22 sites in United States, Belgium, Netherlands, United Kingdom, France and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of adverse events (Safety and Tolerability) (time frame: 12 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05267262, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2025.

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